Trial Outcomes & Findings for A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients (NCT NCT03523728)

Last Updated: 2023-02-03

Results Overview

Total kidney volume is a measure for assessing disease progression in participants with ADPKD, a prognostic biomarker of renal function decline and progression to end-stage renal disease. Kidney volume was assessed using magnetic resonance imaging (MRI). The annualized slope of change in TKV (in percentage \[%\] per year) in each treatment group was obtained from the back-transformation of the mean slope of log10-transformed TKV obtained from the linear mixed effect model. The model included fix effects of treatment (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per randomization stratification factor: class 1C versus 1D versus 1E), time (as continuous variable in years), treatment \* time interaction and mayo imaging classification \* time interaction and included random intercept and slope.

Recruitment status

TERMINATED

Study phase

PHASE2/PHASE3

Target enrollment

478 participants

Primary outcome timeframe

From Baseline to Month 18

Results posted on

2023-02-03

Participant Flow

This study was conducted at 93 sites that enrolled participants in 23 countries. A total of 478 participants were enrolled from 04 October 2018 to 01 June 2021. Study was conducted in 2 stages: Stage 1 and Stage 2.

Participants who completed 24 months of treatment in either Stage 1 or Stage 2 of EFC15392 had option to enroll in an open-label long-term extension study LTS15823 (NCT ID: NCT04705051).

Participant milestones

Participant milestones
Measure	Stage 1- Placebo Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg Participants from Stage 1 were randomized to receive venglustat 8 milligrams (mg) (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 2- Placebo Participants from Stage 2 were randomized to receive 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 2- Venglustat 15 mg Participants from Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months.
Stage 1 (24 Months) STARTED	78	78	80	0	0
Stage 1 (24 Months) COMPLETED	12	10	12	0	0
Stage 1 (24 Months) NOT COMPLETED	66	68	68	0	0
Stage 2 (24 Months) STARTED	0	0	0	123	119
Stage 2 (24 Months) Treated	0	0	0	122	119
Stage 2 (24 Months) Estimated Glomerular Filtration Rate (eGFR) Between 45 and 89.9 mL/Min/1.73 m^2 at Screening	0	0	0	97	90
Stage 2 (24 Months) eGFR Between 30 and 44.9 mL/Min/1.73 m^2 at Screening	0	0	0	25	29
Stage 2 (24 Months) COMPLETED	0	0	0	0	0
Stage 2 (24 Months) NOT COMPLETED	0	0	0	123	119

Reasons for withdrawal

Reasons for withdrawal
Measure	Stage 1- Placebo Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg Participants from Stage 1 were randomized to receive venglustat 8 milligrams (mg) (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 2- Placebo Participants from Stage 2 were randomized to receive 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 2- Venglustat 15 mg Participants from Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months.
Stage 1 (24 Months) Adverse Event	2	2	4	0	0
Stage 1 (24 Months) Progressive disease	0	0	1	0	0
Stage 1 (24 Months) Lack of Efficacy	2	0	0	0	0
Stage 1 (24 Months) Poor compliance to protocol	1	0	0	0	0
Stage 1 (24 Months) Withdrawal by Subject	6	5	7	0	0
Stage 1 (24 Months) Study terminated by sponsor	54	59	56	0	0
Stage 1 (24 Months) Other-Unspecified	1	2	0	0	0
Stage 2 (24 Months) Adverse Event	0	0	0	1	2
Stage 2 (24 Months) Withdrawal by Subject	0	0	0	0	4
Stage 2 (24 Months) Study terminated by sponsor	0	0	0	119	110
Stage 2 (24 Months) Other-Unspecified	0	0	0	2	3
Stage 2 (24 Months) Randomized and not treated	0	0	0	1	0

Baseline Characteristics

A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Stage 1- Placebo n=78 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=80 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 2- Placebo n=123 Participants Participants from Stage 2 were randomized to receive 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 2- Venglustat 15 mg n=119 Participants Participants from Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months.	Total Title n=478 Participants
Age, Continuous	42.6 years STANDARD_DEVIATION 6.0 • n=93 Participants	41.7 years STANDARD_DEVIATION 6.9 • n=4 Participants	43.6 years STANDARD_DEVIATION 5.7 • n=27 Participants	41.7 years STANDARD_DEVIATION 6.9 • n=483 Participants	42.1 years STANDARD_DEVIATION 6.7 • n=36 Participants	42.2 years STANDARD_DEVIATION 6.5 • n=10 Participants
Sex: Female, Male Female	37 Participants n=93 Participants	31 Participants n=4 Participants	34 Participants n=27 Participants	46 Participants n=483 Participants	48 Participants n=36 Participants	196 Participants n=10 Participants
Sex: Female, Male Male	41 Participants n=93 Participants	47 Participants n=4 Participants	46 Participants n=27 Participants	77 Participants n=483 Participants	71 Participants n=36 Participants	282 Participants n=10 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	1 Participants n=483 Participants	0 Participants n=36 Participants	1 Participants n=10 Participants
Race (NIH/OMB) Asian	27 Participants n=93 Participants	27 Participants n=4 Participants	27 Participants n=27 Participants	52 Participants n=483 Participants	55 Participants n=36 Participants	188 Participants n=10 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants	0 Participants n=483 Participants	0 Participants n=36 Participants	1 Participants n=10 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants	1 Participants n=4 Participants	0 Participants n=27 Participants	2 Participants n=483 Participants	0 Participants n=36 Participants	3 Participants n=10 Participants
Race (NIH/OMB) White	48 Participants n=93 Participants	50 Participants n=4 Participants	52 Participants n=27 Participants	66 Participants n=483 Participants	64 Participants n=36 Participants	280 Participants n=10 Participants
Race (NIH/OMB) More than one race	1 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	0 Participants n=483 Participants	0 Participants n=36 Participants	1 Participants n=10 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants	2 Participants n=483 Participants	0 Participants n=36 Participants	4 Participants n=10 Participants

PRIMARY outcome

Timeframe: From Baseline to Month 18

Population: Analysis was performed on Stage 1 intent-to-treat (ITT) population that includes all participants randomized in Stage 1 and were analyzed according to the intervention allocated by randomization (venglustat 15 mg, venglustat 8 mg or placebo). Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=78 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=79 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Annualized Slope of Change in Total Kidney Volume (TKV) From Baseline to Month 18: Stage 1	6.35 percent change in TKV/year Interval 5.1 to 7.62	7.71 percent change in TKV/year Interval 6.46 to 8.98	6.38 percent change in TKV/year Interval 5.11 to 7.66

PRIMARY outcome

Timeframe: From Baseline to Month 24

Population: Analysis was performed on Combined Stage 1 and Stage 2 ITT population that included all participants with an eGFR between 45 and 89.9 mL/min/1.73 m\^2 at screening who are randomized in Stage 1 or Stage 2 and were analyzed according to the intervention allocated by randomization (venglustat 15 mg, venglustat 8 mg or placebo). Data for this outcome measure (OM) was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol.

An eGFR was used to measure level of kidney function and determine the stage of kidney disease in ADPKD participants. Baseline eGFR was defined for each participant as the average of eGFR values assessed prior or equal to first dose of study drug or randomization for participants randomized and not exposed. Annualized rate of change in eGFR in each treatment group was obtained from the linear mixed effect model including the fixed categorical effects of treatment group (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per interactive response technology \[IRT\]: 1C, 1D, 1E), time (as continuous variable in years), treatment-by-time, mayo imaging classification-by-time, and included random intercept and slope.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=175 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=170 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) (Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI]) From Baseline to Month 24: Combined Stage 1 and Stage 2	-2.40 mL/min/1.73 m^2/year Interval -3.3 to -1.49	-4.82 mL/min/1.73 m^2/year Interval -5.82 to -3.83	-4.89 mL/min/1.73 m^2/year Interval -5.8 to -3.99

SECONDARY outcome

Timeframe: From Baseline to Month 24

Population: Analysis was performed on Stage 1 ITT population.

An eGFR was used to measure level of kidney function and determine the stage of kidney disease in ADPKD participants. Baseline eGFR was defined for each participant as the average of eGFR values assessed prior or equal to first dose of study drug or randomization for participants randomized and not exposed. Annualized rate of change in eGFR was obtained from the linear mixed effect model including the fixed categorical effects of treatment group, mayo imaging classification (as per IRT), time (as continuous variable in years), treatment-by-time, mayo imaging classification-by-time, and included random intercept and slope. Due to early termination of study for futility, the two-steps analysis initially planned was not applicable, then the annualized rate of change from baseline in eGFR in Stage 1 population were assessed using all data available up to database lock (i.e., including Month 24 assessment). As this is a slope, it allowed to have more data and to reduce variability.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=78 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=80 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Month 24: Stage 1	-3.14 mL/min/1.73 m^2/year Interval -4.09 to -2.18	-4.74 mL/min/1.73 m^2/year Interval -5.68 to -3.8	-4.59 mL/min/1.73 m^2/year Interval -5.53 to -3.65

SECONDARY outcome

Timeframe: From Baseline to Month 18

Population: As no post-baseline MRI data were collected in Stage 2 due to early study discontinuation, the analysis of TKV in Combined Stage 1 and Stage 2 was not done.

Total kidney volume is a measure for assessing disease progression in participants with ADPKD, a prognostic biomarker of renal function decline and progression to end-stage renal disease. Kidney volume was assessed using MRI. The annualized slope of change in TKV (in % per year) in each treatment group was obtained from the back-transformation of the mean slope of log10-transformed TKV obtained from the linear mixed effect model. The model included fix effects of treatment (venglustat 15 mg, venglustat 8 mg or placebo), mayo imaging classification (as per randomization stratification factor: class 1C versus 1D versus 1E), time (as continuous variable in years), treatment \* time interaction and mayo imaging classification \* time interaction and included random intercept and slope.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From Baseline to Month 18

Population: Analysis was performed on Stage 1 ITT population. Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure.

The BPI-SF is a validated, self-administered questionnaire designed to measure a participant's perceived level of pain. The BPI-SF consisted of 15 items that use a numeric rating scale (NRS) to assess pain severity and pain interference in the past 24 hours and the past week. BPI-SF Item 3 asks participants to "Please rate your pain by marking the box beside the number that best describes your pain at its worst in the past 24 hours." The NRS ranged from 0 (no pain) to 10 (worst imaginable pain), where higher scores indicates greater intensity of pain. Least-squares (LS) means, and standard errors (SE) were estimated from mixed-effect model with repeated measures (MMRM) analysis.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=69 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=65 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=65 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Change in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1	-0.09 score on a scale Standard Error 0.15	0.01 score on a scale Standard Error 0.17	-0.35 score on a scale Standard Error 0.16

SECONDARY outcome

Timeframe: From Baseline to Month 24

Population: Analysis was performed on Combined Stage 1 and Stage 2 ITT population. Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol.

The BPI-SF is a validated, self-administered questionnaire designed to measure a participant's perceived level of pain. The BPI-SF consisted of 15 items that use a NRS to assess pain severity and pain interference in the past 24 hours and the past week. BPI-SF Item 3 asks participants to "Please rate your pain by marking the box beside the number that best describes your pain at its worst in the past 24 hours." The NRS ranged from 0 (no pain) to 10 (worst imaginable pain), where higher scores indicate greater intensity of pain. LS means and SE were estimated from MMRM analysis.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=133 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=65 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=126 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Change in Pain Severity as Measured by Brief Pain Inventory-Short Form (BPI-SF)-Item 3 Scale Score From Baseline to Month 24: Combined Stage 1 and Stage 2	-0.20 score on a scale Standard Error 0.23	-0.31 score on a scale Standard Error 0.36	-0.36 score on a scale Standard Error 0.23

SECONDARY outcome

Timeframe: From Baseline to Month 18

Population: Analysis was performed on Stage 1 ITT population. Here, 'Overall number of participants analyzed' signifies participants who were evaluable for the outcome measure.

The BFI-SF is a 10-item, validated, self-administered questionnaire that was originally developed to assess fatigue severity. The 10-items were measured on a 0-10 scale, with 0 being 'does not interfere' and 10 being 'completely interferes.' BFI - Item 3 asks participants to 'Please rate your fatigue (weariness, tiredness) by circling the one number that best describes your worst level of fatigue during the past 24 hours. The NRS ranged from 0 (no fatigue) to 10 (worst imaginable fatigue). Higher global scores were associated with more severe fatigue. LS means and SE were estimated from MMRM analysis.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=69 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=65 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=65 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Change in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI-SF)-Item 3 Scale Score From Baseline to Month 18: Stage 1	-0.26 score on a scale Standard Error 0.23	-0.10 score on a scale Standard Error 0.25	-0.64 score on a scale Standard Error 0.24

SECONDARY outcome

Timeframe: From Baseline to Month 24

The BFI-SF is a 10-item, validated, self-administered questionnaire that was originally developed to assess fatigue severity. The 10-items were measured on a 0-10 scale, with 0 being 'does not interfere' and 10 being 'completely interferes.' BFI - Item 3 asks participants to "Please rate your fatigue (weariness, tiredness) by circling the one number that best describes your worst level of fatigue during the past 24 hours. The NRS ranged from 0 (no fatigue) to 10 (worst imaginable fatigue). Higher global scores were associated with more severe fatigue. LS means and SE were estimated from MMRM analysis.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=133 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=65 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=126 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Change in Fatigue Severity as Measured by Brief Fatigue Inventory (BFI-SF)-Item 3 Scale Score From Baseline to Month 24: Combined Stage 1 and Stage 2	0.02 score on a scale Standard Error 0.38	-0.85 score on a scale Standard Error 0.55	-0.51 score on a scale Standard Error 0.38

SECONDARY outcome

Timeframe: Day 1: 3 hours Post-Dose, Month 1: Pre-Dose and 3 hours Post-Dose, Month 6: Pre-Dose, Month 18: Pre-Dose

Population: Analysed on Stage 1 pharmacokinetic (PK) population that included all participants randomized in Stage 1 who took at least one dose of study drug and had at least one post-baseline PK assessment; analyzed according to the intervention they actually received. Here, "number analyzed" signifies participants with available data for each specified category.

Venglustat plasma concentrations was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method. Data for this outcome measure was not planned to be collected and analyzed for placebo arm.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=78 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=80 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Pharmacokinetics: Plasma Concentration of Venglustat: Stage 1 Day 1: 3 hours Post-Dose	27.8 nanograms per milliliter Standard Deviation 11.9	50.9 nanograms per milliliter Standard Deviation 22.5	—
Pharmacokinetics: Plasma Concentration of Venglustat: Stage 1 Month 1: Pre-Dose	54.7 nanograms per milliliter Standard Deviation 19.2	103.9 nanograms per milliliter Standard Deviation 50.2	—
Pharmacokinetics: Plasma Concentration of Venglustat: Stage 1 Month 1: 3 hours Post-Dose	82.1 nanograms per milliliter Standard Deviation 24.7	154.6 nanograms per milliliter Standard Deviation 61.1	—
Pharmacokinetics: Plasma Concentration of Venglustat: Stage 1 Months 6: Pre-Dose	57.5 nanograms per milliliter Standard Deviation 23.5	106.2 nanograms per milliliter Standard Deviation 56.3	—
Pharmacokinetics: Plasma Concentration of Venglustat: Stage 1 Months 18: Pre-Dose	58.3 nanograms per milliliter Standard Deviation 22.0	120.3 nanograms per milliliter Standard Deviation 73.1	—

SECONDARY outcome

Timeframe: Month 1: Pre-dose and 3 hours Post-dose

Population: Analysis was performed on Stage 2 PK population that included all participants with an eGFR between 45 and 89.9 mL/min/1.73 m\^2 at screening who were randomized in Stage 2, who took at least one dose of study drug and who had at least one post-baseline PK assessment. Participants were analyzed according to study drug they actually received. Here, "number analyzed" signifies participants with available data for each specified category.

Venglustat plasma concentrations was determined using a validated LC-MS/MS method. Data for this outcome measure was not planned to be collected and analyzed for placebo arm.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=77 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Pharmacokinetics: Plasma Concentration of Venglustat: Stage 2 Month 1: Pre-Dose	109.3 nanograms per milliliter Standard Deviation 60.7	—	—
Pharmacokinetics: Plasma Concentration of Venglustat: Stage 2 Month 1: 3 hours Post-Dose	163.5 nanograms per milliliter Standard Deviation 79.7	—	—

SECONDARY outcome

Timeframe: From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier

Population: Analysis was performed on Stage 1 safety population that included all participants randomized in Stage 1 who took at least one dose or part of a dose of the double-blind IMP, analyzed according to the treatment actually received (venglustat 15 mg, venglustat 8 mg, or placebo)

An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. A serious adverse event (SAE) was any untoward medical occurrence that at any dose: results in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or was a medically important event. TEAEs were AEs that developed, worsened or became serious during the treatment-emergent (TE) period (defined as the time from the first investigational medicinal product \[IMP\] administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier).

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=78 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=80 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs): Stage 1 TEAEs	62 Participants	65 Participants	70 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs): Stage 1 TESAEs	8 Participants	15 Participants	19 Participants

SECONDARY outcome

Timeframe: From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier

Population: Analysis was performed on extended Combined Stage 1 and Stage 2 safety population that included all participants with an eGFR between 45 and 89.9 mL/min/1.73 m\^2 at screening who were randomized in Stage 1 or Stage 2 and who took at least one dose or part of a dose of IMP, analyzed according to treatment actually received. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol.

An AE was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. SAE was any untoward medical occurrence that at any dose: results in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or was a medically important event. TEAEs were AEs that developed, worsened or became serious during the TE period (defined as the time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier).

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=200 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=199 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs): Combined Stage 1 and Stage 2 TEAEs	128 Participants	65 Participants	143 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs): Combined Stage 1 and Stage 2 TESAEs	14 Participants	15 Participants	26 Participants

SECONDARY outcome

Timeframe: From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier

Population: Analysis was performed on extended Combined Stage 1 and Stage 2 safety population. Here, "number analyzed" signifies participants with available data for specified category. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol.

Criteria for potentially clinically significant abnormalities: Hemoglobin: less than or equal to (\<=) 115 grams per liter (g/L) \[Male\]; \<=95 g/L \[Female\]; greater than or equal to (\>=) 185 g/L \[Male\]; \>=165 g/L \[Female\]; Decrease from baseline \>=20 g/L; Hematocrit: \<=0.37 volume/volume (v/v) \[Male\]; \<=0.32 v/v \[Female\]; \>=0.55 v/v \[Male\]; \>=0.5 v/v \[Female\]; Erythrocyte (red blood cells \[RBC\]): \>=6\*10\^12 per liter (/L); Platelet: less than (\<) 100\*10\^9/L; \>=700\*10\^9/L; Leukocyte (white blood cells \[WBC\]): \<3\*10\^9/L \[Non-Black\]; \<2\*10\^9/L \[Black\], \>=16\*10\^9/L; Neutrophils: \<1.5\*10\^9/L \[Non-Black\]; \<1\*10\^9/L \[Black\]; Lymphocytes: greater than (\>) 4\*10\^9/L, Monocytes: \>0.7\*10\^9/L; Basophils: \>0.1\*10\^9/L; and Eosinophils: \>0.5\*10\^9/L or \>upper limit of normal (ULN) (if ULN \>=0.5\*10\^9/L).

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=200 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=199 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Hemoglobin: <=115 g/L (Male); <=95 g/L (Female)	7 Participants	3 Participants	5 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Hemoglobin: >=185 g/L (Male); >=165 g/L (Female)	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Hemoglobin: Decrease from baseline >=20 g/L	4 Participants	7 Participants	5 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Hematocrit: <=0.37 v/v (Male); <=0.32 v/v (Female)	40 Participants	20 Participants	42 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Hematocrit: >=0.55 v/v (Male); >=0.5 v/v (Female)	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Erythrocyte (RBC): >=6 * 10^12/L	1 Participants	0 Participants	1 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Platelet: <100*10^9/L	0 Participants	2 Participants	1 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Platelet: >=700*10^9/L	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Leukocyte (WBC): <310^9/L (Non-Black); <210^9/L (Black)	1 Participants	2 Participants	4 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Leukocyte (WBC): >=16*10^9/L	0 Participants	2 Participants	1 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Neutrophils: <1.510^9/L (Non-Black); <110^9/L (Black)	1 Participants	1 Participants	3 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Lymphocytes: >4*10^9/L	0 Participants	1 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Monocytes >0.7*10^9/L	6 Participants	3 Participants	7 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Basophils: >0.1*10^9/L	33 Participants	5 Participants	8 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Hematology: Combined Stage 1 and Stage 2 Eosinophils: >0.5*10^9/L or >ULN	7 Participants	2 Participants	1 Participants

SECONDARY outcome

Timeframe: From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier

Criteria for potentially clinically significant abnormalities: Glucose: \<=3.9 millimoles per liter (mmol/L) and \<lower limit of normal (LLN): \>=11.1 mmol/L (unfasted); \>=7 mmol/L (fasted); Albumin:\<=25 g/L; Sodium: \<=129 mmol/L; \>=160 mmol/L; Potassium: \<3 mmol/L; \>=5.5 mmol/L; Chloride: \<80 mmol/L, \>115 mmol/L; Creatinine: \>=150 micro millimoles per liter (mcmol/L) (Adults); \>=30% change from Baseline; \>=100% change from Baseline, Urea Nitrogen: \>=17 mmol/L; Alanine Aminotransferase (ALT): \>3 ULN; Aspartate Aminotransferase (AST): \>3 ULN; Alkaline Phosphatase: \>1.5 ULN; Total Bilirubin: \>1.5 ULN, \>2 ULN; ALT \>3 ULN and Bilirubin \>2 ULN; and Direct Bilirubin \>35% Bilirubin and Bilirubin \>1.5 ULN.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=200 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=199 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Glucose: <=3.9 mmol/L and <LLN	17 Participants	5 Participants	12 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Glucose: >=11.1 mmol/L (unfasted); >=7 mmol/L (fasted)	10 Participants	5 Participants	19 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Albumin: <=25 g/L	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Sodium: <=129 mmol/L	1 Participants	0 Participants	3 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Sodium: >=160 mmol/L	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Potassium: <3 mmol/L	0 Participants	1 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Potassium: >=5.5 mmol/L	5 Participants	5 Participants	11 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Chloride: <80 mmol/L	0 Participants	1 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Chloride: >115 mmol/L	1 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Creatinine: >=150 mcmol/L (Adults)	50 Participants	21 Participants	70 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Creatinine: >=30% change from Baseline	12 Participants	18 Participants	25 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Creatinine: >=100% change from Baseline	0 Participants	1 Participants	4 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Urea Nitrogen: >=17 mmol/L	1 Participants	0 Participants	6 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 ALT: > 3 ULN	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 AST: >3 ULN	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Alkaline Phosphatase: >1.5 ULN	3 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Total Bilirubin: >1.5 ULN	2 Participants	0 Participants	1 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Total Bilirubin: >2 ULN	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 ALT >3 ULN and Bilirubin >2 ULN	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Clinical Chemistry: Combined Stage 1 and Stage 2 Direct Bilirubin >35% Bilirubin and Bilirubin >1.5 ULN	2 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier

Criteria for potentially clinically significant abnormalities: Urine pH: \<=4.6 and \>=8.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=200 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=199 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Number of Participants With Potentially and Clinically Significant Abnormalities: Urinalysis: Combined Stage 1 and Stage 2 Urine pH: <=4.6	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially and Clinically Significant Abnormalities: Urinalysis: Combined Stage 1 and Stage 2 Urine pH: >=8	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier

Criteria for potentially clinically significant abnormalities: Sitting Systolic Blood Pressure: \<=95 millimeters of Mercury (mmHg) and decrease from Baseline \>=20 mmHg; \>=160 mmHg and increase from Baseline \>=20 mmHg; Sitting Diastolic Blood Pressure: \<=45 mmHg and decrease from Baseline \>=10 mmHg, \>=110 mmHg and increase from Baseline \>=10 mmHg; Sitting Heart Rate: \<=50 beats/minute and decrease from Baseline \>=20 beats/minute; \>=120 beats/minute and increase from Baseline \>=20 beats/minute; and Weight: \>=5% decrease from Baseline; \>=5% increase from Baseline.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=200 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=199 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Number of Participants With Potentially and Clinically Significant Abnormalities: Vital Signs: Combined Stage 1 and Stage 2 Sitting Systolic Blood Pressure: <=95 mmHg and decrease from Baseline >=20 mmHg	0 Participants	0 Participants	1 Participants
Number of Participants With Potentially and Clinically Significant Abnormalities: Vital Signs: Combined Stage 1 and Stage 2 Sitting Systolic Blood Pressure: >=160 mmHg and increase from Baseline >=20 mmHg	2 Participants	3 Participants	10 Participants
Number of Participants With Potentially and Clinically Significant Abnormalities: Vital Signs: Combined Stage 1 and Stage 2 Sitting Diastolic Blood Pressure: <= 45 mmHg and decrease from Baseline >=10 mmHg	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially and Clinically Significant Abnormalities: Vital Signs: Combined Stage 1 and Stage 2 Sitting Diastolic Blood Pressure: >=110 mmHg and increase from Baseline >=10 mmHg	2 Participants	4 Participants	7 Participants
Number of Participants With Potentially and Clinically Significant Abnormalities: Vital Signs: Combined Stage 1 and Stage 2 Sitting Heart Rate: <=50 beats/min and decrease from Baseline >=20 beats/min	0 Participants	2 Participants	2 Participants
Number of Participants With Potentially and Clinically Significant Abnormalities: Vital Signs: Combined Stage 1 and Stage 2 Sitting Heart Rate: >=120 beats/min and increase from Baseline >=20 beats/min	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially and Clinically Significant Abnormalities: Vital Signs: Combined Stage 1 and Stage 2 Weight: >=5% decrease from Baseline	11 Participants	4 Participants	13 Participants
Number of Participants With Potentially and Clinically Significant Abnormalities: Vital Signs: Combined Stage 1 and Stage 2 Weight: >=5% increase from Baseline	15 Participants	9 Participants	15 Participants

SECONDARY outcome

Timeframe: From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier

Criteria for potentially clinically significant abnormalities: Heart Rate: \<50 beats/minute; \<50 beats/minute and decrease from Baseline \>=20 beats/minute; \<40 beats/minute; \<40 beats/minute and decrease from Baseline \>=20 beats/min; \<30 beats/minute; \>90 beats/minute; \>90 beats/minute and increase from Baseline \>=20 beats/minute; \>100 beats/minute; \>100 beats/minute and increase from Baseline \>=20 beats/minute; \>120 beats/minute; \>120 beats/minute, increase from Baseline \>=20 beats/minute; PR Interval: \>200 milliseconds (msec); \>200 msec and increase from Baseline \>=25%; \>220 msec, \>240 msec; QRS Interval: \>110 msec; \>110 msec and increase from Baseline \>=25%; \>120 msec; \>120 msec and increase from Baseline \>=25%; QT Interval: \>500 msec; QT corrected for heart rate (QTc) Bazett: \>450 msec; \>480 msec; increase from Baseline (30-60) msec; increase from Baseline \>60 msec; QTc Fridericia: \>450 msec; \>480 msec; increase from Baseline (30-60) msec and increase from Baseline \> 60 msec.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=200 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=199 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 Heart Rate: <50 beats/min	7 Participants	6 Participants	13 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 Heart Rate: <50 beats/min and decrease from Baseline >=20 beats/min	1 Participants	0 Participants	1 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 Heart Rate: <40 beats/min	0 Participants	1 Participants	1 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 Heart Rate: <40 beats/min and decrease from Baseline >=20 beats/min	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 Heart Rate: <30 beats/min	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 Heart Rate: >90 beats/min	4 Participants	2 Participants	3 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 Heart Rate: >90 beats/min and increase from Baseline >=20 beats/min	2 Participants	2 Participants	2 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 Heart Rate: >100 beats/min	1 Participants	0 Participants	2 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 Heart Rate: >100 beats/min and increase from Baseline >=20 beats/min	1 Participants	0 Participants	1 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 Heart Rate: >120 beats/min	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 Heart Rate: >120 beats/min and increase from Baseline >=20 beats/min	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 PR Interval: >200 msec	8 Participants	2 Participants	10 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 PR Interval: >200 msec and increase from Baseline >=25%	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 PR Interval: >220 msec	1 Participants	0 Participants	2 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 PR Interval: >240 msec	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QRS Interval: >110 msec	11 Participants	4 Participants	10 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QRS Interval: > 110 msec and increase from Baseline >=25%	0 Participants	0 Participants	1 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QRS Interval: >120 msec	2 Participants	1 Participants	3 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QRS Interval: >120 msec and increase from Baseline >=25%	0 Participants	0 Participants	1 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QT Interval: >500 msec	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QTc Bazett: >450 msec	5 Participants	2 Participants	3 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QTc Bazett: >480 msec	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QTc Bazett: Increase from Baseline [30-60] msec	13 Participants	1 Participants	5 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QTc Bazett: Increase from Baseline >60 msec	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QTc Fridericia: >450 msec	4 Participants	1 Participants	2 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QTc Fridericia: >480 msec	0 Participants	0 Participants	0 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QTc Fridericia: Increase from Baseline [30-60] msec	5 Participants	1 Participants	3 Participants
Number of Participants With Potentially Clinically Significant Abnormalities: Electrocardiogram: Combined Stage 1 and Stage 2 QTc Fridericia: Increase from Baseline >60 msec	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, Month 18, Month 24

Physical examination included: Head, Heart, Lung, Abdomen, Musculo/Skeletal, Skin, and Mental Status. Abnormality in physical examination was based on investigator's discretion. Results are based on the treatment emergent period which was defined as time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=200 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=199 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Baseline: Head	1 Participants	1 Participants	4 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Baseline: Heart	2 Participants	1 Participants	1 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Baseline: Lung	0 Participants	0 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Baseline: Abdomen	15 Participants	12 Participants	24 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Baseline: Musculo/Skeletal	5 Participants	1 Participants	4 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Baseline: Skin	7 Participants	2 Participants	3 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Baseline: Mental Status	0 Participants	0 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 18: Head	0 Participants	0 Participants	1 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 18: Heart	0 Participants	0 Participants	1 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 18: Lung	0 Participants	0 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 18: Abdomen	2 Participants	1 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 18: Musculo/Skeletal	0 Participants	0 Participants	1 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 18: Skin	1 Participants	1 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 18: Mental Status	1 Participants	0 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 24: Head	0 Participants	0 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 24: Heart	0 Participants	0 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 24: Lung	0 Participants	0 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 24: Abdomen	0 Participants	0 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 24: Musculo/Skeletal	0 Participants	0 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 24: Skin	0 Participants	0 Participants	0 Participants
Number of Participants With Physical Examination Abnormalities: Combined Stage 1 and Stage 2 Month 24: Mental Status	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, Months 3, 6, 9, 12, 15, 18, 21, and 24, Last on-treatment value up to last IMP + 1 day (anytime during the maximum duration of 25 months)

The Beck Depression Inventory-II (BDI-II) is a 21-item questionnaire used to assess depression. Most items are rated on a 4-point scale from 0 to 3, and a few items are rated on a 7-point scale. Individual item scores are added to get a total BDI-II score from 0 to 63. The higher the total score, the more severe the depression, and the lower the total score, the less severe the depression.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=200 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=199 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 Change at Last on-treatment value	-0.5 score on a scale Standard Deviation 5.1	0.1 score on a scale Standard Deviation 4.0	0.3 score on a scale Standard Deviation 4.6
Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 Baseline	4.0 score on a scale Standard Deviation 4.7	3.0 score on a scale Standard Deviation 4.1	3.4 score on a scale Standard Deviation 4.1
Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 Change at Month 3	-0.1 score on a scale Standard Deviation 4.0	0.7 score on a scale Standard Deviation 4.0	0.7 score on a scale Standard Deviation 4.3
Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 Change at Month 6	-0.6 score on a scale Standard Deviation 4.2	0.9 score on a scale Standard Deviation 4.2	0.3 score on a scale Standard Deviation 3.9
Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 Change at Month 9	-0.3 score on a scale Standard Deviation 4.8	0.1 score on a scale Standard Deviation 3.7	0.7 score on a scale Standard Deviation 4.9
Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 Change at Month 12	-1.9 score on a scale Standard Deviation 3.9	0.2 score on a scale Standard Deviation 4.7	1.5 score on a scale Standard Deviation 5.3
Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 Change at Month 15	-1.5 score on a scale Standard Deviation 4.4	-0.1 score on a scale Standard Deviation 4.4	-0.3 score on a scale Standard Deviation 4.0
Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 Change at Month 18	-1.0 score on a scale Standard Deviation 4.9	0.5 score on a scale Standard Deviation 3.0	0.0 score on a scale Standard Deviation 3.5
Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 Change at Month 21	1.0 score on a scale Standard Deviation 9.5	-0.1 score on a scale Standard Deviation 1.3	1.1 score on a scale Standard Deviation 2.8
Change From Baseline in Beck Depression Inventory-II (BDI-II) Score: Combined Stage 1 and Stage 2 Change at Month 24	-0.3 score on a scale Standard Deviation 0.5	0.8 score on a scale Standard Deviation 2.2	0.3 score on a scale Standard Deviation 1.7

SECONDARY outcome

Timeframe: From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier

Population: Analysis was performed on extended Combined Stage 1 and Stage 2 safety population. Here, "number analyzed" signifies participants with available data for each specified category. Data for this OM was planned to be collected and analyzed for combined Stage 1 and Stage 2 population as pre-specified in protocol. TE period: time from first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier.

Worsening of lens opacity classification system (LOCS) III score or World Health Organization (WHO) grade in nuclear opacification, cortical opacification and posterior subcapsular opacification were assessed for 'Any eye', 'Unilateral' and 'Bilateral' separately. A participant could be counted in all the 3 categories. In each category, the worst case was taken into account. To be evaluable for 'Any', a participant had to have at least one eye evaluable, whereas, for 'Unilateral' and 'Bilateral', a participant had to have both eyes evaluable. Therefore, the sum of 'Unilateral' + 'Bilateral' is not necessarily equal to 'Any' in the below table. The difference observed in 'Nuclear Opacification' in 15 mg group, comes from that 1 participant who had Unilateral worsening at a given visit and a Bilateral worsening at another visit. Therefore, this participant was counted as 'Unilateral', 'Bilateral' and counted only once in 'Any'. Results are based on the TE period.

Outcome measures

Outcome measures
Measure	Stage 1- Placebo n=200 Participants Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 Participants Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=199 Participants Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.
Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2 Worsening of LOCS III score >= 0.5 or WHO grade >= 1.0 in nuclear opacification: Any eye	8 Participants	3 Participants	9 Participants
Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2 Worsening of LOCS III score >= 0.5 or WHO grade >= 1.0 in nuclear opacification: Unilateral	0 Participants	0 Participants	4 Participants
Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2 Worsening of LOCS III score >= 0.5 or WHO grade >= 1.0 in nuclear opacification: Bilateral	8 Participants	3 Participants	6 Participants
Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2 Worsening of LOCS III score >= 0.8 or WHO grade >= 1.0 in cortical opacification: Any eye	4 Participants	4 Participants	4 Participants
Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2 Worsening of LOCS III score >= 0.8 or WHO grade >= 1.0 in cortical opacification: Unilateral	1 Participants	0 Participants	1 Participants
Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2 Worsening of LOCS III score >= 0.8 or WHO grade >= 1.0 in cortical opacification: Bilateral	2 Participants	4 Participants	3 Participants
Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2 Worsening of LOCS III score>=0.5 or WHO grade >= 1.0 in posterior subcapsular opacification:Any eye	2 Participants	2 Participants	3 Participants
Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2 Worsening of LOCS III score>=0.5 or WHO grade >=1.0 in posterior subcapsularopacification:Unilateral	0 Participants	1 Participants	1 Participants
Number of Participants With Worsening Lens Opacity From Baseline During the Treatment-emergent Period: Combined Stage 1 and Stage 2 Worsening of LOCS III score>=0.5 or WHO grade >=1.0 in posterior subcapsular opacification:Bilateral	2 Participants	1 Participants	2 Participants

Adverse Events

Stage 1- Placebo

Serious events: 8 serious events

Other events: 42 other events

Deaths: 0 deaths

Stage 1- Venglustat 8 mg

Serious events: 15 serious events

Other events: 46 other events

Deaths: 0 deaths

Stage 1- Venglustat 15 mg

Serious events: 19 serious events

Other events: 48 other events

Deaths: 0 deaths

Stage 2- Placebo

Serious events: 6 serious events

Other events: 32 other events

Deaths: 0 deaths

Stage 2- Venglustat 15 mg

Serious events: 7 serious events

Other events: 48 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Stage 1- Placebo n=78 participants at risk Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 participants at risk Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=80 participants at risk Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 2- Placebo n=122 participants at risk Participants from Stage 2 were randomized to receive 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 2- Venglustat 15 mg n=119 participants at risk Participants from Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months.
Infections and infestations Appendicitis	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.5% 2/80 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Covid-19	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.6% 2/122 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Diverticulitis	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Gastroenteritis Viral	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Infected Cyst	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Influenza	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Kidney Infection	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Pneumonia	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Renal Cyst Infection	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	3.8% 3/78 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.84% 1/119 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Urinary Tract Infection	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.5% 2/80 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Basal Cell Carcinoma	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Breast Cancer	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Intraductal Proliferative Breast Lesion	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.84% 1/119 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Invasive Ductal Breast Carcinoma	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.84% 1/119 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Mediastinum Neoplasm	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.84% 1/119 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Metabolism and nutrition disorders Fluid Overload	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Metabolism and nutrition disorders Hyperkalaemia	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Psychiatric disorders Depression	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Psychiatric disorders Suicidal Ideation	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Nervous system disorders Carpal Tunnel Syndrome	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Nervous system disorders Intracranial Aneurysm	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Nervous system disorders Loss Of Consciousness	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.84% 1/119 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Nervous system disorders Polyneuropathy	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Nervous system disorders Syncope	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Cardiac disorders Arteriosclerosis Coronary Artery	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Cardiac disorders Coronary Artery Stenosis	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Cardiac disorders Mitral Valve Incompetence	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Cardiac disorders Supraventricular Tachycardia	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Gastrointestinal disorders Dental Cyst	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Gastrointestinal disorders Inguinal Hernia	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Gastrointestinal disorders Large Intestinal Stenosis	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Gastrointestinal disorders Pancreatitis Acute	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.84% 1/119 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Gastrointestinal disorders Rectal Haemorrhage	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Hepatobiliary disorders Hepatic Pain	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Skin and subcutaneous tissue disorders Rash	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Musculoskeletal and connective tissue disorders Flank Pain	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Musculoskeletal and connective tissue disorders Osteoarthritis	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Musculoskeletal and connective tissue disorders Rotator Cuff Syndrome	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Renal and urinary disorders Acute Kidney Injury	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Renal and urinary disorders Calculus Urinary	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.84% 1/119 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Renal and urinary disorders Chronic Kidney Disease	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Renal and urinary disorders Haematuria	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	3.8% 3/78 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Renal and urinary disorders Renal Cyst Haemorrhage	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.7% 2/119 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Renal and urinary disorders Renal Cyst Ruptured	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.6% 2/78 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Renal and urinary disorders Renal Haemorrhage	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Renal and urinary disorders Ureterolithiasis	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Reproductive system and breast disorders Adenomyosis	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
General disorders Haemorrhagic Cyst	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
General disorders Non-Cardiac Chest Pain	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Injury, poisoning and procedural complications Back Injury	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Injury, poisoning and procedural complications Hand Fracture	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Injury, poisoning and procedural complications Jaw Fracture	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Injury, poisoning and procedural complications Subdural Haematoma	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Injury, poisoning and procedural complications Tibia Fracture	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.

Other adverse events

Other adverse events
Measure	Stage 1- Placebo n=78 participants at risk Participants from Stage 1 were randomized to receive 2 capsules of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 1- Venglustat 8 mg n=78 participants at risk Participants from Stage 1 were randomized to receive venglustat 8 mg (i.e., 2 capsules of 4 mg) once daily for treatment period of 24 months.	Stage 1- Venglustat 15 mg n=80 participants at risk Participants from Stage 1 were randomized to receive 1 capsule of venglustat 15 mg and 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 2- Placebo n=122 participants at risk Participants from Stage 2 were randomized to receive 1 capsule of placebo matched to venglustat once daily for treatment period of 24 months.	Stage 2- Venglustat 15 mg n=119 participants at risk Participants from Stage 2 were randomized to receive 1 capsule of venglustat 15 mg once daily for treatment period of 24 months.
Infections and infestations Covid-19	6.4% 5/78 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	7.7% 6/78 • Number of events 6 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.5% 3/122 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.9% 7/119 • Number of events 7 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Conjunctivitis	5.1% 4/78 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.84% 1/119 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Influenza	5.1% 4/78 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	3.8% 3/78 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Nasopharyngitis	12.8% 10/78 • Number of events 13 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	6.4% 5/78 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	7.5% 6/80 • Number of events 9 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.6% 2/122 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Upper Respiratory Tract Infection	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.1% 4/78 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	7.5% 6/80 • Number of events 8 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	3.3% 4/122 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.0% 6/119 • Number of events 7 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Infections and infestations Urinary Tract Infection	7.7% 6/78 • Number of events 6 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	6.4% 5/78 • Number of events 6 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	8.8% 7/80 • Number of events 15 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.5% 3/122 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.84% 1/119 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Nervous system disorders Dizziness	2.6% 2/78 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.1% 4/78 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.0% 4/80 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.6% 2/122 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.5% 3/119 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Nervous system disorders Headache	7.7% 6/78 • Number of events 6 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	10.3% 8/78 • Number of events 9 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	13.8% 11/80 • Number of events 14 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.7% 7/122 • Number of events 8 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	6.7% 8/119 • Number of events 8 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Eye disorders Cataract Cortical	6.4% 5/78 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.6% 2/78 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	3.8% 3/80 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.7% 2/119 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Eye disorders Cataract Nuclear	6.4% 5/78 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.6% 2/78 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	3.8% 3/80 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.7% 2/119 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Eye disorders Dry Eye	6.4% 5/78 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.5% 2/80 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Vascular disorders Hypertension	2.6% 2/78 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	6.4% 5/78 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	8.8% 7/80 • Number of events 7 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	3.3% 4/122 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	3.4% 4/119 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Respiratory, thoracic and mediastinal disorders Cough	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	6.4% 5/78 • Number of events 8 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	10.0% 8/80 • Number of events 8 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.9% 7/119 • Number of events 8 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Gastrointestinal disorders Constipation	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.1% 4/78 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	3.8% 3/80 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	6.7% 8/119 • Number of events 8 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Gastrointestinal disorders Diarrhoea	2.6% 2/78 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	7.5% 6/80 • Number of events 6 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.5% 3/122 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.7% 2/119 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Gastrointestinal disorders Dyspepsia	5.1% 4/78 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.7% 2/119 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Gastrointestinal disorders Nausea	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	7.7% 6/78 • Number of events 6 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	8.8% 7/80 • Number of events 9 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/122 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.0% 6/119 • Number of events 6 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Musculoskeletal and connective tissue disorders Arthralgia	5.1% 4/78 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	3.8% 3/78 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.5% 2/80 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.6% 2/122 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.7% 2/119 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Musculoskeletal and connective tissue disorders Back Pain	10.3% 8/78 • Number of events 9 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	7.7% 6/78 • Number of events 7 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	10.0% 8/80 • Number of events 8 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	4.1% 5/122 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.7% 2/119 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Musculoskeletal and connective tissue disorders Flank Pain	3.8% 3/78 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	6.2% 5/80 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Musculoskeletal and connective tissue disorders Pain In Extremity	0.00% 0/78 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.1% 4/78 • Number of events 4 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
Renal and urinary disorders Haematuria	3.8% 3/78 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.1% 4/78 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/80 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	3.3% 4/122 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.7% 2/119 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
General disorders Fatigue	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	5.1% 4/78 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	11.2% 9/80 • Number of events 9 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	2.5% 3/122 • Number of events 3 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.7% 2/119 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.
General disorders Influenza Like Illness	1.3% 1/78 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	6.4% 5/78 • Number of events 5 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	1.2% 1/80 • Number of events 1 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.82% 1/122 • Number of events 2 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.	0.00% 0/119 • From the first IMP administration up to the last IMP administration in EFC15392 study + 30 days (i.e., up to 25 months) or up to the first visit in LTS15823 study, whichever comes earlier Reported AEs were TEAEs that developed/worsened in grade or became serious during TE period (time from the first IMP administration up to the last IMP administration in EFC15392 study + 30 days or up to the first visit in LTS15823 study, whichever comes earlier). Analysis was performed on the Stage 1 and Stage 2 safety population.

Additional Information

Trial Transparency Team

Sanofi aventis recherche & développement

Phone: 800-633-1610

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
Publication restrictions are in place

Restriction type: OTHER