Trial Outcomes & Findings for An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1 (NCT NCT03350451)

Last Updated: 2024-04-25

Results Overview

AE is any untoward medical occurrence in a participant or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Safety analysis set included all participants who received any amount of study drug.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

20 participants

Primary outcome timeframe

Baseline (Day -1) up to 54 months

Results posted on

2024-04-25

Participant Flow

Participants took part in the study at 9 study centers in France, Germany, Israel, Netherlands and the United Kingdom from 04 April 2018 to 07 February 2023. A total of 20 participants previously treated in Study 001B (NCT02706886) were enrolled and treated in this study.

The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg once monthly (QM) \& 3 mg/kg once every 3 months (Q3M), these participants were pooled into one arm as recommended by the Safety Review Committee (SRC). As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.

Participant milestones

Participant milestones
Measure	Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M Participants enrolling from study 001B, received lumasiran, subcutaneous (SC) injection, at a starting dose of 1.0 milligrams per kilograms (mg/kg) QM or 3.0 mg/kg (Q3M) from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC.	Lumasiran (ALN-GO1): 3.0 mg/kg QM Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period.
Overall Study STARTED	13	7
Overall Study Safety Analysis Set	13	7
Overall Study Pharmacodynamic Analysis Set	13	7
Overall Study COMPLETED	13	7
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M n=13 Participants Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 1.0 mg/kg QM or 3.0 mg/kg Q3M from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC.	Lumasiran (ALN-GO1): 3.0 mg/kg QM n=7 Participants Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period.	Total n=20 Participants Total of all reporting groups
Age, Continuous	16.1 years STANDARD_DEVIATION 12.31 • n=5 Participants	12.6 years STANDARD_DEVIATION 3.95 • n=7 Participants	14.9 years STANDARD_DEVIATION 10.18 • n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants	4 Participants n=7 Participants	13 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=5 Participants	7 Participants n=7 Participants	20 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race/Ethnicity, Customized Asian	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Race/Ethnicity, Customized White	10 Participants n=5 Participants	5 Participants n=7 Participants	15 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline (Day -1) up to 54 months

Population: The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.

Outcome measures

Outcome measures
Measure	Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M n=13 Participants Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 1.0 mg/kg QM or 3.0 mg/kg Q3M from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC.	Lumasiran (ALN-GO1): 3.0 mg/kg QM n=7 Participants Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period.
Number of Participants With at Least One Adverse Event (AE)	13 Participants	7 Participants

SECONDARY outcome

Timeframe: Baseline (Day -1) up to 54 months

Oxalate produced by the liver is the key toxic metabolite that drives disease pathology in participants with primary hyperoxaluria type 1 (PH1). The risk of disease complications increase continuously as oxalate levels increase. 24-hour urinary oxalate (millimole \[mmol\]/ 24 hour \[h\]/1.73 meters squared \[m\^2\]) corrected for BSA at each visit per participant was calculated as follows: \[Urine oxalate concentration (micromole per liter \[umol/L\])/1000 (umol/mmol)\]\*\[24hour urine volume (mL)/1000 (mL/L)\]\* \[24 hours/actual collection hours\]\*1.73/(BSA). Baseline was the derived baseline value from the lumasiran treated period of Study ALN-GO1-001. A negative change from baseline indicated a favorable outcome. PD analysis set included all participants who received any amount of study drug and who had at least 1 post-dose urine sample for PD. Overall number of participants analyzed are the number of participants with data available for analysis.

Outcome measures

Outcome measures
Measure	Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M n=12 Participants Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 1.0 mg/kg QM or 3.0 mg/kg Q3M from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC.	Lumasiran (ALN-GO1): 3.0 mg/kg QM n=6 Participants Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period.
Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 54 Months	-1.425 mmol/24hr/1.73m^2 Standard Error 0.2188	-2.126 mmol/24hr/1.73m^2 Standard Error 0.5528

SECONDARY outcome

Timeframe: Baseline (Day -1) up to 54 months

Baseline is the derived baseline value from the lumasiran treated period of Study ALN-GO1-001. A negative change from baseline indicates a favorable outcome. PD analysis set included all participants who received any amount of study drug and who had at least 1 post-dose urine sample for PD.

Outcome measures

Outcome measures
Measure	Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M n=13 Participants Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 1.0 mg/kg QM or 3.0 mg/kg Q3M from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC.	Lumasiran (ALN-GO1): 3.0 mg/kg QM n=7 Participants Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period.
Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 54 Months	-0.2175 mmol/mmol Standard Deviation 0.03880	-0.2480 mmol/mmol Standard Deviation 0.04047

SECONDARY outcome

Timeframe: Baseline (Day -1) up to 54 months

Baseline was defined as the last measurement prior to the first dose of lumasiran in the ALN-GO1-001 study. eGFR was calculated based on the Modification of Diet in Renal Disease (MDRD) formula for participants \>=18 years of age at enrollment and the Schwartz Bedside formula for participants \<18 years of age at enrollment. eGFR based on MDRD formula was calculated as follows: eGFR (mL/min/1.73 m\^2) = 175 × (serum creatinine {SCr} \[μmol/deciliter(dL)\]/88.4)-1.154 × (age)-0.203 × (0.742, if female), or × (1.212, if African American) and based on Schwartz formula: eGFR (mL/min/1.73m2) = (36.2 × height \[cm\])/ SCr (μmol /dL). Safety analysis set included all participants who received any amount of study drug.

Outcome measures

Outcome measures
Measure	Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M n=13 Participants Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 1.0 mg/kg QM or 3.0 mg/kg Q3M from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC.	Lumasiran (ALN-GO1): 3.0 mg/kg QM n=7 Participants Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period.
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 54 Months	0.674 mL/min/1.73m^2 Standard Error 3.7970	-12.026 mL/min/1.73m^2 Standard Error 3.6369

Adverse Events

Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M

Serious events: 4 serious events

Other events: 13 other events

Deaths: 0 deaths

Lumasiran (ALN-GO1): 3.0 mg/kg QM

Serious events: 3 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Lumasiran (ALN-GO1): 1.0 mg/kg QM or 3.0 mg/kg Q3M n=13 participants at risk Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 1.0 mg/kg QM or 3.0 mg/kg Q3M from Day 1 up to a maximum of Month 6. By Month 6, all participants were approved to change dose and/or dosing regimen to receive lumasiran, SC injection at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6. As the cumulative dose administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC.	Lumasiran (ALN-GO1): 3.0 mg/kg QM n=7 participants at risk Participants enrolling from study 001B, received lumasiran, SC injection, at a starting dose of 3.0 mg/kg, QM, from Day 1 up to a maximum of Month 21. By Month 21, all participants were approved to change dosing regimen to receive lumasiran, SC injection, at a dose of 3.0 mg/kg, Q3M, up to Month 51 of the treatment period.
Endocrine disorders Thyroid Mass	0.00% 0/13 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.	14.3% 1/7 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.
Infections and infestations Pyelonephritis	0.00% 0/13 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.	14.3% 1/7 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.
Injury, poisoning and procedural complications Bone Contusion	7.7% 1/13 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.	0.00% 0/7 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.
Injury, poisoning and procedural complications Craniocerebral Injury	7.7% 1/13 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.	0.00% 0/7 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.
Investigations Glomerular Filtration Rate Decreased	0.00% 0/13 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.	14.3% 1/7 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.
Renal and urinary disorders Nephrolithiasis	7.7% 1/13 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.	0.00% 0/7 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.
Renal and urinary disorders Renal Colic	7.7% 1/13 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.	0.00% 0/7 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.
Renal and urinary disorders Ureterolithiasis	15.4% 2/13 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.	14.3% 1/7 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.
Surgical and medical procedures Renal Stone Removal	7.7% 1/13 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.	0.00% 0/7 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.
Surgical and medical procedures Thyroid Operation	0.00% 0/13 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.	14.3% 1/7 • Baseline (Day -1) up to 54 months Safety analysis set. The cumulative dose of lumasiran administered over 6 months was the same for both 1 mg/kg QM \& 3 mg/kg Q3M, these participants were pooled into one arm as recommended by the SRC. As pre-specified in the SAP, participants who began treatment at 1 mg/kg QM, then transitioned to 3 mg/kg Q3M were to be summarized as a single group. All 3 participants who began treatment at 1 mg/kg QM transitioned to 3 mg/kg Q3M regimen by Month 6.

Other adverse events

Additional Information

Chief Medical Officer

Alnylam Pharmaceuticals Inc.

Phone: 866-330-0326

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER