Trial Outcomes & Findings for Extension Study of Efficacy and Safety of LTP001 in Pulmonary Arterial Hypertension Participants (NCT NCT05764265)

Results Overview

Incidence and severity of adverse events (AEs) by treatment group, including changes in the vital signs, electrocardiogram and laboratory results qualifying and reported as AEs. Due to the study termination, no patient reached Week 52. At the end of treatment visit, final safety assessments were performed.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

31 participants

Primary outcome timeframe

Up to approximately 45 weeks

Results posted on

2025-12-26

Participant Flow

A total of 31 participants who completed the parent study up to the end of treatment were screened for the extension study.

Participant milestones

Participant milestones
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Overall Study STARTED	23	8
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	23	8

Reasons for withdrawal

Reasons for withdrawal
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Overall Study Adverse Event	1	0
Overall Study Physician Decision	1	8
Overall Study Study Terminated By Sponsor	20	0
Overall Study Participant Decision	1	0

Baseline Characteristics

Extension Study of Efficacy and Safety of LTP001 in Pulmonary Arterial Hypertension Participants

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=23 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=8 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	Total n=31 Participants Total of all reporting groups
Age, Continuous	47.0 Years STANDARD_DEVIATION 11.76 • n=30 Participants	48.4 Years STANDARD_DEVIATION 13.35 • n=30 Participants	47.4 Years STANDARD_DEVIATION 11.97 • n=60 Participants
Age, Customized 18 - <65	20 Participants n=30 Participants	7 Participants n=30 Participants	27 Participants n=60 Participants
Age, Customized 65 - <85	3 Participants n=30 Participants	1 Participants n=30 Participants	4 Participants n=60 Participants
Sex: Female, Male Female	21 Participants n=30 Participants	6 Participants n=30 Participants	27 Participants n=60 Participants
Sex: Female, Male Male	2 Participants n=30 Participants	2 Participants n=30 Participants	4 Participants n=60 Participants
Race/Ethnicity, Customized White	21 Participants n=30 Participants	7 Participants n=30 Participants	28 Participants n=60 Participants
Race/Ethnicity, Customized American Indian or Alaskan	2 Participants n=30 Participants	0 Participants n=30 Participants	2 Participants n=60 Participants
Race/Ethnicity, Customized Asian	0 Participants n=30 Participants	1 Participants n=30 Participants	1 Participants n=60 Participants

PRIMARY outcome

Timeframe: Up to approximately 45 weeks

Population: The safety analysis set included all participants who received any study treatment.

Incidence and severity of adverse events (AEs) by treatment group, including changes in the vital signs, electrocardiogram and laboratory results qualifying and reported as AEs. Due to the study termination, no patient reached Week 52. At the end of treatment visit, final safety assessments were performed.

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=23 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=8 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) AEs	52.2 Percentage of participants	62.5 Percentage of participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Serious AEs	17.4 Percentage of participants	0 Percentage of participants

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data.

Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including CO.

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=6 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=2 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Average Cardiac Output (CO) at Week 26	-0.111 liters per minute Standard Deviation 0.2153	-0.065 liters per minute Standard Deviation 0.5916

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data.

Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including PA pressure.

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=6 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=2 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Mean Pulmonary Artery (PA) Pressure at Week 26	3.8 mmHg Standard Deviation 8.18	-1.5 mmHg Standard Deviation 7.78

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data.

Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including pulmonary capillary wedge pressure (PCWP).

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=6 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=2 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Pulmonary Capillary Wedge Pressure (PCWP) at Week 26	-1.0 mmHg Standard Deviation 1.79	-0.5 mmHg Standard Deviation 0.71

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data.

PVR was defined as the resistance against blood flow from the pulmonary artery to the left atrium measured in dynes.sec.cm-5.

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=6 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=2 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Right Heart Catheterization Pulmonary Vascular Resistance (PVR) at Week 26	100.058 dynes.sec.cm-5 Standard Deviation 95.5879	-7.445 dynes.sec.cm-5 Standard Deviation 34.8250

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data.

The Right Heart Catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including RA pressures.

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=6 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=2 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Right Atrium (RA) Pressures at Week 26	-1.5 mmHg Standard Deviation 6.22	0.0 mmHg Standard Deviation 2.83

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data.

The Right Heart Catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including SVR.

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=6 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=2 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Systemic Vascular Resistance (SVR) at Week 26	166.748 dynes.sec.cm-5 Standard Deviation 128.6202	-49.710 dynes.sec.cm-5 Standard Deviation 28.5388

SECONDARY outcome

Timeframe: Baseline, Week 26, up to 39 weeks (EOT)

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data.

6MWD test measures the distance that a participant can walk on a flat, hard surface in a period of 6 minutes. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures.

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=16 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=2 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Six Minute Walk Distance (6MWD) Week 26 n=6,2	-33.7 meters Standard Deviation 55.60	-9.0 meters Standard Deviation 25.46
Change From Baseline in Six Minute Walk Distance (6MWD) EOT n=16,2	-6.3 meters Standard Deviation 50.21	-1.0 meters Standard Deviation 1.41

SECONDARY outcome

Timeframe: Baseline, Week 26, up to 39 weeks (EOT)

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data. Number analyzed is the number of participants with data available at the specified time points.

Key right ventricular (RV) function endpoints such as tricuspid annular plane systolic excursion (TAPSE) were assessed with echocardiography. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures. Only a minimal number of patients completed an echocardiogram (Echo).

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=7 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=2 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE) Week 26 n=6,2	0.03 centimeters Standard Deviation 0.175	0.00 centimeters Standard Deviation 0.283
Change From Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE) EOT n=7,0	0.01 centimeters Standard Deviation 0.682	—

SECONDARY outcome

Timeframe: Baseline, Week 26, up to 39 weeks (EOT)

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data. Number analyzed is the number of participants with data available at the specified time points.

Key right ventricular (RV) function endpoints such as tricuspid annular systolic velocity (TASV) were assessed with echocardiography. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures. Only a minimal number of patients completed an echocardiogram (Echo).

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=7 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=2 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Tricuspid Annular Plane Systolic Velocity (TASV) Week 26 n=6,2	-2.2 centimeters per second Standard Deviation 3.43	-0.5 centimeters per second Standard Deviation 0.71
Change From Baseline in Tricuspid Annular Plane Systolic Velocity (TASV) EOT n=7,0	-2.6 centimeters per second Standard Deviation 1.90	—

SECONDARY outcome

Timeframe: Baseline, Week 26, up to 39 weeks (EOT)

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data. Number analyzed is the number of participants with data available at the specified time points.

Key right ventricular (RV) function per echocardiography. The terms Tricuspid Annular Systolic Velocity (TASV) and Peak Velocity of Excursion (RV S') are synonymous in echocardiography to describe the peak systolic velocity of the lateral tricuspid annulus. Including both TASV and RV S' as separate secondary endpoints was an oversight in the protocol as the data, calculation, and analyses for both (TASV and RV S') are identical. Therefore, the TASV and RV S' data in this results disclosure are the same. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures. Only a minimal number of patients completed an echocardiogram (Echo).

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=7 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=2 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Peak Velocity of Excursion (RV S') Week 26 n=6,2	-2.2 centimeters per second Standard Deviation 3.43	-0.5 centimeters per second Standard Deviation 0.71
Change From Baseline in Peak Velocity of Excursion (RV S') EOT n=7,0	-2.6 centimeters per second Standard Deviation 1.90	—

SECONDARY outcome

Timeframe: Baseline, Week 26, up to 39 weeks (EOT)

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data. Number analyzed is the number of participants with data available at the specified time points.

Key right ventricular (RV) function endpoints such as RV fractional area change (RV FAC) were assessed with echocardiography. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures. Only a minimal number of patients completed an echocardiogram (Echo).

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=6 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=1 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Fractional Area Change (FAC) Week 26 n=6,1	-0.87 percent Standard Deviation 6.162	7.30 percent Standard Deviation NA Standard deviation was not calculable due to the single data point.
Change From Baseline in Fractional Area Change (FAC) EOT n=6,0	0.97 percent Standard Deviation 5.290	—

SECONDARY outcome

Timeframe: Baseline up to 39 weeks (EOT)

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data.

emPHasis-10 is a questionnaire with 10 questions designed to determine how pulmonary hypertension affects a participant's life. Each item is scored on a scale of 0 to 5, with a total score ranging from 0 to 50. A higher score indicates worse quality of life. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures.

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=3 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=1 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Quality of Life Measured by the emPHasis-10 Questionnaire	1.476 score Standard Deviation 2.3226	1.000 score Standard Deviation NA Standard deviation was not calculable due to the single data point.

SECONDARY outcome

Timeframe: Baseline up to 39 weeks (EOT)

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data.

PAH-SYMPACT is a questionnaire used to assess pulmonary arterial hypertension symptoms and their impact. Individual item scores range from 0 to 4. Total score is calculated as the sum of the scores for the individual items divided by the number of items. A higher score indicates more severe symptoms/impacts. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures.

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=2 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=1 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in Quality of Life Measured by the PAH-SYMPACT Questionnaire	2.929 score Standard Deviation 2.3234	-0.833 score Standard Deviation NA Standard deviation was not calculable due to the single data point.

SECONDARY outcome

Timeframe: Baseline up to 39 weeks (EOT)

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data. Number analyzed is the number of participants with an event up to and including the end time of the interval.

Time to any of the following: * Death * Hospital stay greater than 24 hours due to worsening of pulmonary arterial hypertension * Worsening of PAH resulting in need for lung transplantation or balloon atrial septostomy * Initiation of parenteral prostanoid therapy, initiation of oxygen therapy, initiation of any other pulmonary arterial hypertension-specific therapies or need for increase of diuretics for more than 4 weeks due to worsening of pulmonary arterial hypertension * Significant drop in six-minute walk distance Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures.

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=6 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Time to Clinical Worsening	346.0 days Interval 187.0 to The upper limit of 95% CI was not calculable due to an insufficient number of participants with events.	—

SECONDARY outcome

Timeframe: Baseline up to 39 weeks (EOT)

Population: The pharmacodynamic (PD) analysis set included all participants who received study treatment and had no protocol deviations with a relevant impact on PD data.

NT-proBNP is a blood biomarker to assess right ventricular distress. Due to the study termination, no patient reached Week 52. At the end of treatment (EOT) visit, final safety assessments were performed based on investigator judgement and patient willingness to undergo procedures.

Outcome measures

Outcome measures
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=19 Participants Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=6 Participants Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study
Change From Baseline in N-terminal Fragment of the Prohormone B-type Natriuretic Peptide (NT-ProBNP)	3.832 picomoles per liter Standard Deviation 31.2255	7.250 picomoles per liter Standard Deviation 16.6033

Adverse Events

LTP001 6 mg (Actual Treatment in CLTP001A12201)

Serious events: 4 serious events

Other events: 5 other events

Deaths: 0 deaths

LTP001 6 mg (Placebo in CLTP001A12201)

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Total

Serious events: 4 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=23 participants at risk Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=8 participants at risk Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	Total n=31 participants at risk Total
General disorders Medical device site haemorrhage	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	0.00% 0/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Infections and infestations Device related infection	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	0.00% 0/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Infections and infestations Disseminated gonococcal infection	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	0.00% 0/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Respiratory, thoracic and mediastinal disorders Pulmonary arterial hypertension	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	0.00% 0/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	6.5% 2/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.

Other adverse events

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: + 1 862 778 8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of pooled data (i.e.,data from all sites) in clinical trial or disclosure of trial results in their entirety.
Publication restrictions are in place

Restriction type: OTHER

Measure	LTP001 6 mg (Actual Treatment in CLTP001A12201) n=23 participants at risk Participants had received LTP001, 6 mg, in Study CLTP001A12201, and continued to receive LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	LTP001 6 mg (Placebo in CLTP001A12201) n=8 participants at risk Participants had received placebo in Study CLTP001A12201, followed by LTP001, 6 mg, orally once daily in the morning for approximately 39 weeks in this extension study	Total n=31 participants at risk Total
Blood and lymphatic system disorders Lymphadenopathy	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Eye disorders Eczema eyelids	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
General disorders Fatigue	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Infections and infestations Bartholinitis	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Infections and infestations Bronchitis	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Infections and infestations Nasopharyngitis	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	9.7% 3/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Infections and infestations Tooth abscess	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Musculoskeletal and connective tissue disorders Arthralgia	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	0.00% 0/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	6.5% 2/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Musculoskeletal and connective tissue disorders Musculoskeletal stiffness	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Nervous system disorders Dizziness	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Nervous system disorders Headache	4.3% 1/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	6.5% 2/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Psychiatric disorders Insomnia	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	12.5% 1/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	3.2% 1/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.
Respiratory, thoracic and mediastinal disorders Nasal congestion	8.7% 2/23 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	0.00% 0/8 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.	6.5% 2/31 • Adverse events were reported from first dose of study treatment until end of study treatment plus 6 weeks post treatment, up to approximately 45 weeks.