HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in participants with ATTR amyloidosis with cardiomyopathy.

Study Overview

• Status: Active, not recruiting
• Conditions: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
• Intervention / Treatment: Drug: Vutrisiran; Drug: Sterile Normal Saline (0.9% NaCl)

• Study Type: Interventional
• Enrollment (Actual): 655
• Phase: Phase 3

Contacts and Locations

Study Locations

• Argentina
  • Buenos Aires, Argentina, C1199ABB
    • Clinical Trial Site
  • Buenos Aires, Argentina, C1428ART
    • Clinical Trial Site
  • Ciudad Autónoma de BuenosAires
    • Buenos Aires, Ciudad Autónoma de BuenosAires, Argentina, C1093AAS
      • Clinical Trial Site
  • Pilar
    • Buenos Aires, Pilar, Argentina, B1629ODT
      • Clinical Trial Site
• Australia
  • New South Wales
    • Darlinghurst, New South Wales, Australia, 2010
      • Clinical Trial Site
    • Northmead, New South Wales, Australia, 2152
      • Clinical Trial Site
  • Queensland
    • Woolloongabba, Queensland, Australia, 4102
      • Clinical Trial Site
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • Clinical Trial Site
  • Victoria
    • Melbourne, Victoria, Australia, 3004
      • Clinical Trial Site
• Austria
  • Graz, Austria, 8036
    • Clinical Trial Site
  • Vienna, Austria, 1090
    • Clinical Trial Site
  • Vienna, Austria, 1210
    • Clinical Trial Site
• Belgium
  • Brussels, Belgium, 1070
    • Clinical Trial Site
  • Leuven, Belgium, 3000
    • Clinical Trial Site
  • Oost-Vlaanderen
    • Dendermonde, Oost-Vlaanderen, Belgium, 9200
      • Clinical Trial Site
    • Ghent, Oost-Vlaanderen, Belgium, 9000
      • Clinical Trial Site
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 2C4
      • Clinical Trial Site
  • Quebec
    • Québec, Quebec, Canada, G1V4G5
      • Clinical Trial Site
• Croatia
  • Zagreb, Croatia, 10000
    • Clinical Trial Site
• Czechia
  • Olomoucký kraj
    • Olomouc, Olomoucký kraj, Czechia, 779 00
      • Clinical Trial Site
  • Praha, Hlavní Mesto
    • Prague, Praha, Hlavní Mesto, Czechia, 12000
      • Clinical Trial Site
• Denmark
  • Aarhus N, Denmark, 8200
    • Clinical Trial Site
  • Region Syddanmark
    • Odense, Region Syddanmark, Denmark, 5000
      • Clinical Trial Site
• France
  • Créteil, France, 94000
    • Clinical Trial Site
  • Marseille, France, 13385
    • Clinical Trial Site
  • Paris, France, 75877
    • Clinical Trial Site
  • Toulouse, France, 31059
    • Clinical Trial Site
• Germany
  • Essen, Germany, 45122
    • Clinical Trial Site
  • Göttingen, Germany, 37075
    • Clinical Trial Site
  • Heidelberg, Germany, 69120
    • Clinical Trial Site
  • Münster, Germany, 48149
    • Clinical Trial Site
  • Würzburg, Germany, 97078
    • Clinical Trial Site
  • Hesse
    • Frankfurt am Main, Hesse, Germany, 60313
      • Clinical Trial Site
  • Saxony
    • Leipzig, Saxony, Germany, 04103
      • Clinical Trial Site
• Hungary
  • Budapest, Hungary, 1134
    • Clinical Trial Site
  • Budapest, Hungary, 1088
    • Clinical Trial Site
  • Szeged, Hungary, 6725
    • Clinical Trial Site
• Ireland
  • Dublin, Ireland, D07 A8NN
    • Clinical Trial Site
  • Dublin, Ireland, Dublin 8
    • Clinical Trial Site
• Israel
  • Haifa, Israel, 3339419
    • Clinical Trial Site
  • Jerusalem, Israel, 9112000
    • Clinical Trial Site
  • Ramat Gan, Israel, 5262000
    • Clinical Trial Site
• Japan
  • Fukuoka, Japan, 812-8582
    • Clinical Trial Site
  • Kumamoto, Japan, 860-8556
    • Clinical Trial Site
  • Aichi-ken
    • Tsurumai-cho, Aichi-ken, Japan, 466-8560
      • Clinical Trial Site
  • Fukuoka
    • Fukuoka, Fukuoka, Japan, 810-0001
      • Clinical Trial Site
    • Kurume, Fukuoka, Japan, 830-0011
      • Clinical Trial Site
  • Kagawa-ken
    • Kita-gun, Kagawa-ken, Japan, 761-0793
      • Clinical Trial Site
  • Kochi
    • Nankoku, Kochi, Japan, 783-8505
      • Clinical Trial Site
  • Nagano
    • Matsumoto, Nagano, Japan, 390-8621
      • Clinical Trial Site
  • Nara
    • Kashihara, Nara, Japan, 634-8522
      • Clinical Trial Site
  • Osaka
    • Suita, Osaka, Japan, 564-8565
      • Clinical Trial Site
    • Suita, Osaka, Japan, 565-0871
      • Clinical Trial Site
  • Tokyo
    • Bunkyo-ku, Tokyo, Japan, 113-8655
      • Clinical Trial Site
    • Bunkyo-ku, Tokyo, Japan, 113-8431
      • Clinical Trial Site
    • Shinjuku-Ku, Tokyo, Japan, 160-8582
      • Clinical Trial Site
• Latvia
  • Riga, Latvia, LV-1002
    • Clinical Trial Site
• Lebanon
  • Beyrouth
    • Hamra, Beyrouth, Lebanon, 1111
      • Clinical Trial Site
• Lithuania
  • Kaunas, Lithuania, LT-50161
    • Clinical Trial Site
• Malaysia
  • Pahang
    • Kuantan, Pahang, Malaysia, 25100
      • Clinical Trial Site
  • Selangor
    • Sungai Buloh, Selangor, Malaysia, 47000
      • Clinical Trial Site
• Moldova
  • Chisinau, Moldova, MD2025
    • Clinical Trial Site
• Netherlands
  • Eindhoven, Netherlands, 5623 EJ
    • Clinical Trial Site
  • Groningen, Netherlands, 9713 GZ
    • Clinical Trial Site
  • Utrecht, Netherlands, 3584 CW
    • Clinical Trial Site
• Norway
  • Oslo, Norway, 0424
    • Clinical Trial Site
• Peru
  • Lima, Peru, 15088
    • Clinical Trial Site
  • Lima region
    • San Isidro, Lima region, Peru, 15076
      • Clinical Trial Site
• Poland
  • Katowice, Poland, 40-555
    • Clinical Trial Site
  • Lower Silesian Voivodeship
    • Wroclaw, Lower Silesian Voivodeship, Poland, 50-556
      • Clinical Trial Site
  • Lódzkie
    • Lodz, Lódzkie, Poland, 90-127
      • Clinical Trial Site
  • Masovian Voivodeship
    • Warsaw, Masovian Voivodeship, Poland, 01-192
      • Clinical Trial Site
  • Pomeranian Voivodeship
    • Gdansk, Pomeranian Voivodeship, Poland, 80-382
      • Clinical Trial Site
• Portugal
  • Coimbra, Portugal, 3000-075
    • Clinical Trial Site
  • Faro, Portugal, 8000-386
    • Clinical Trial Site
  • Guimarães, Portugal, 4835-044
    • Clinical Trial Site
  • Porto, Portugal, 4099-001
    • Clinical Trial Site
  • Lisbon District
    • Lisbon, Lisbon District, Portugal, 1649-035
      • Clinical Trial Site
• Saudi Arabia
  • Riyadh, Saudi Arabia, 12372
    • Clinical Trial Site
  • Mecca Region
    • Mecca, Mecca Region, Saudi Arabia, 21955
      • Clinical Trial Site
• Slovenia
  • Ljubljana, Slovenia, 1000
    • Clinical Trial Site
• South Korea
  • Seoul, South Korea, 03080
    • Clinical Trial Site
  • Seoul, South Korea, 03722
    • Clinical Trial Site
  • Seoul, South Korea, 06351
    • Clinical Trial Site
  • Seoul, South Korea, 06591
    • Clinical Trial Site
• Spain
  • Barcelona, Spain, 08035
    • Clinical Trial Site
  • Bilbao, Spain, 48013
    • Clinical Trial Site
  • Huelva, Spain, 21005
    • Clinical Trial Site
  • Málaga, Spain, 29010
    • Clinical Trial Site
  • Barcelona
    • L'Hospitalet de Llobregat, Barcelona, Spain, 08907
      • Clinical Trial Site
  • Madrid
    • Majadahonda, Madrid, Spain, 28222
      • Clinical Trial Site
• Sweden
  • Gothenburg, Sweden, 422 46
    • Clinical Trial Site
  • Västerbotten County
    • Göteborg, Västerbotten County, Sweden, 422 46
      • Clinical Trial Site
• Thailand
  • Chiang Mai, Thailand, 50200
    • Clinical Trial Site
• United Kingdom
  • Birmingham, United Kingdom, B15 2SQ
    • Clinical Trial Site
  • Cardiff, United Kingdom, CF15 9SS
    • Clinical Trial Site
  • London, United Kingdom, NW3 2QG
    • Clinical Trial Site
  • Manchester, United Kingdom, M15 6SE
    • Clinical Trial Site
  • Lanarkshire
    • Bellshill, Lanarkshire, United Kingdom, ML4 3NJ
      • Clinical Trial Site
  • London, City of
    • London, London, City of, United Kingdom, SE1 1YR
      • Clinical Trial Site
  • Londonderry
    • Londonderry, Londonderry, United Kingdom, BT47 6SB
      • Clinical Trial Site
  • Northumberland
    • Hexham, Northumberland, United Kingdom, NE46 1QJ
      • Clinical Trial Site
• United States
  • California
    • La Mesa, California, United States, 91942
      • Clinical Trial Site
    • Los Angeles, California, United States, 90033
      • Clinical Trial Site
    • Stanford, California, United States, 94305
      • Clinical Trial Site
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • Clinical Trial Site
  • Georgia
    • Gainesville, Georgia, United States, 30501
      • Clinical Trial Site
  • Illinois
    • Chicago, Illinois, United States, 60637
      • Clinical Trial Site
    • Evanston, Illinois, United States, 60201
      • Clinical Trial Site
  • Massachusetts
    • Boston, Massachusetts, United States, 02118
      • Clinical Trial Site
    • Boston, Massachusetts, United States, 02115
      • Clinical Trial Site
  • Minnesota
    • Rochester, Minnesota, United States, 55902
      • Clinical Trial Site
  • New York
    • Manhasset, New York, United States, 11030
      • Clinical Trial Site
    • New York, New York, United States, 10032
      • Clinical Trial Site
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Clinical Trial Site
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Clinical Trial Site
  • Pennsylvania
    • Allentown, Pennsylvania, United States, 18103
      • Clinical Trial Site
    • Philadelphia, Pennsylvania, United States, 19104
      • Clinical Trial Site
  • Texas
    • Houston, Texas, United States, 77030
      • Clinical Trial Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Has a documented diagnosis of transthyretin (ATTR) amyloidosis with cardiomyopathy, classified as either hereditary ATTR (hATTR) amyloidosis with cardiomyopathy or wild-type ATTR (wtATTR) amyloidosis with cardiomyopathy meeting pre-specified diagnostic criteria
• Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF OR clinical evidence of HF

Exclusion Criteria:

• Has known primary amyloidosis or leptomeningeal amyloidosis
• Has New York Heart Association (NYHA) Class IV heart failure
• Has NYHA Class III heart failure AND is at high risk based on pre-specified criteria
• Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV at the Screening visit
• Has estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m^2
• Has received prior TTR-lowering treatment
• Has other non-TTR cardiomyopathy, hypertensive cardiomyopathy, cardiomyopathy due to valvular heart disease, or cardiomyopathy due to ischemic heart disease

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Vutrisiran 25 mg; Participants received vutrisiran 25 milligrams (mg) administered subcutaneously (SC) once every 3 months (q3M) during the 36-month double-blind (DB) period. After the DB period, participants continue receiving vutrisiran, 25 mg, SC injection, q3M for up to 24 months in the open-label treatment extension (OLE) period.	Drug: Vutrisiran; Vutrisiran will be administered by SC injection.; Other Names: ALN-TTRSC02
Placebo Comparator: Placebo; Participants received vutrisiran matching placebo administered SC q3M during the 36-month DB period. After the DB period, participants receive vutrisiran, 25 mg, SC injection, q3M for up to 24 months in the OLE period.	Drug: Vutrisiran; Vutrisiran will be administered by SC injection.; Other Names: ALN-TTRSC02; Drug: Sterile Normal Saline (0.9% NaCl); Sterile normal saline (0.9% NaCl) will be administered by SC injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population	All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) were compared between treatment groups using a modified Andersen-Gill model with a robust variance. All-cause mortality included heart transplantation and LVAD placement events along with deaths; recurrent CV events included CV hospitalizations and urgent HF visits. The number of participants with at least one CV event or all-cause mortality event have been reported here. No imputation was done for participants who dropped out early for the primary analysis of this composite outcome endpoint.	Up to Month 36
Composite Endpoint of All-Cause Mortality and Recurrent CV Events (CV Hospitalizations and Urgent HF Visits) in the Vutrisiran Monotherapy Subgroup	All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) were compared between treatment groups using a modified Andersen-Gill model with a robust variance. All-cause mortality included heart transplantation and LVAD placement events along with deaths; recurrent CV events included CV hospitalizations and urgent HF visits. The number of participants with at least one CV event or all-cause mortality event have been reported here. No imputation was done for participants who dropped out early for the primary analysis of this composite outcome endpoint.	Up to Month 36

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in 6-Minute Walk Test (6-MWT) in the Overall Population	The 6-MWT is an assessment of functional exercise capacity. Participants are instructed to walk back and forth along a flat, straight path, typically 30 meters in length, for a duration of 6 minutes. The total distance covered in meters is recorded at the end of 6 minutes. A longer distance reflects a better outcome. Analysis was based on the mixed-effect model of repeated measures (MMRM). Missing change values due to amyloidosis disease progression and death were imputed using sampling with replacement from the worst 10% of observed values, as specified in the statistical analysis plan (SAP).	Baseline to Month 30
Change From Baseline in 6-MWT in the Vutrisiran Monotherapy Subgroup	The 6-MWT is an assessment of functional exercise capacity. Participants are instructed to walk back and forth along a flat, straight path, typically 30 meters in length, for a duration of 6 minutes. The total distance covered in meters is recorded at the end of 6 minutes. A longer distance reflects a better outcome. Analysis was based on the MMRM. Missing change values due to amyloidosis disease progression and death were imputed using sampling with replacement from the worst 10% of observed values, as specified in the SAP.	Baseline to Month 30
Change From Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) Score in the Overall Population	The KCCQ is a 23-item self-administered questionnaire quantifying 6 domains (symptoms [frequency and burden], physical function, quality of life (QoL), social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and overall summary [OS]). Scores were generated for each domain and transformed to a range of 0-100, in which higher scores reflect better health status. KCCQ- overall summary score was average of domains- physical function, total symptoms (average of symptom frequency and burden), QoL, and social limitation, and transformed to a single score which ranged from 0 (worst) - 100 (the best possible status), where the higher score reflected better health status. Analysis was based on the MMRM.	Baseline to Month 30
Change From Baseline in the KCCQ-OS Score in the Vutrisiran Monotherapy Subgroup	The KCCQ is a 23-item self-administered questionnaire quantifying 6 domains (symptoms [frequency and burden], physical function, QoL, social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and OS). Scores were generated for each domain and transformed to a range of 0-100, in which higher scores reflect better health status. KCCQ- overall summary score was average of domains- physical function, total symptoms (average of symptom frequency and burden), QoL, and social limitation, and transformed to a single score which ranged from 0 (worst) - 100 (the best possible status), where the higher score reflected better health status. Analysis was based on the MMRM.	Baseline to Month 30
All-cause Mortality in the Overall Population and Vutrisiran Monotherapy Subgroup	All-cause mortality also included heart transplantation and LVAD placement events along with deaths; recurrent CV events included CV hospitalizations and urgent HF visits.	Up to 42 months
Percentage of Participants With Change in NYHA Class at Month 30 in the Overall Population	NYHA class is a clinical assessment of symptoms resulting from heart failure. In NYHA functional classification system places participants in 1 of 4 categories based on limitations of physical activity. Class I denotes no symptoms and no limitation of physical activity; II, slight limitation, resulting in symptoms with ordinary physical activity; III, marked limitation, resulting in symptoms with less than ordinary physical activity; and IV, symptoms at rest. Here, the participants with a change in NYHA class have been reported in two categories: 1) participants who had no change or had improvement in the NYHA class (lower class) from baseline and 2) participants who had a worsened NYHA class from baseline (higher class). Values have been rounded off to a single decimal.	Baseline to Month 30
Percentage of Participants With Change in NYHA Class at Month 30 in the Vutrisiran Monotherapy Subgroup	NYHA class is a clinical assessment of symptoms resulting from heart failure. In NYHA functional classification system places participants in 1 of 4 categories based on limitations of physical activity. Class I denotes no symptoms and no limitation of physical activity; II, slight limitation, resulting in symptoms with ordinary physical activity; III, marked limitation, resulting in symptoms with less than ordinary physical activity; and IV, symptoms at rest. Here, the participants with a change in NYHA class have been reported in two categories: 1) participants who had no change or had improvement in the NYHA class (lower class) from baseline and 2) participants who had a worsened NYHA class from baseline (higher class). Values have been rounded off to a single decimal.	Baseline to Month 30

Collaborators and Investigators

• Sponsor: Alnylam Pharmaceuticals
• Investigators: Study Director: Medical Director, Alnylam Pharmaceuticals

Publications and helpful links

General Publications

• Maurer MS, Berk JL, Damy T, Sheikh FH, Gonzalez-Costello J, Morbach C, Delgado D, Bondue A, Azevedo O, Poulsen SH, Jankowska EA, Yang L, Bender S, Eraly SA, Jay PY, Vest J, Fontana M. Impact of Vutrisiran on Cardiac Biomarkers in Patients With Transthyretin Amyloidosis With Cardiomyopathy From HELIOS-B. J Am Coll Cardiol. 2025 Aug 12;86(6):459-475. doi: 10.1016/j.jacc.2025.04.055.
• Witteles RM, Garcia-Pavia P, Damy T, Grogan M, Sheikh FH, Morbach C, Bender S, Exter J, Eraly SA, Fontana M. Vutrisiran Improves Survival and Reduces Cardiovascular Events in ATTR Amyloid Cardiomyopathy: HELIOS-B. J Am Coll Cardiol. 2025 Apr 30:S0735-1097(25)06170-4. doi: 10.1016/j.jacc.2025.04.008. Online ahead of print.
• Maurer MS, Witteles RM, Garcia-Pavia P, Sheikh FH, Morbach C, Rodriguez Duque D, Aldinc E, Eraly SA, Gillmore JD. Impact of Heart Failure Severity on Vutrisiran Efficacy in Transthyretin Amyloidosis With Cardiomyopathy. J Am Coll Cardiol. 2025 May 27;85(20):1927-1939. doi: 10.1016/j.jacc.2025.03.477. Epub 2025 Mar 17.
• Fontana M, Maurer MS, Gillmore JD, Bender S, Aldinc E, Eraly SA, Jay PY, Solomon SD. Outpatient Worsening Heart Failure in Patients With Transthyretin Amyloidosis With Cardiomyopathy in the HELIOS-B Trial. J Am Coll Cardiol. 2025 Feb 25;85(7):753-761. doi: 10.1016/j.jacc.2024.11.015. Epub 2024 Nov 18.
• Fontana M, Berk JL, Gillmore JD, Witteles RM, Grogan M, Drachman B, Damy T, Garcia-Pavia P, Taubel J, Solomon SD, Sheikh FH, Tahara N, Gonzalez-Costello J, Tsujita K, Morbach C, Pozsonyi Z, Petrie MC, Delgado D, Van der Meer P, Jabbour A, Bondue A, Kim D, Azevedo O, Hvitfeldt Poulsen S, Yilmaz A, Jankowska EA, Algalarrondo V, Slugg A, Garg PP, Boyle KL, Yureneva E, Silliman N, Yang L, Chen J, Eraly SA, Vest J, Maurer MS; HELIOS-B Trial Investigators. Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy. N Engl J Med. 2025 Jan 2;392(1):33-44. doi: 10.1056/NEJMoa2409134. Epub 2024 Aug 30.
• Jering KS, Fontana M, Lairez O, Longhi S, Azevedo O, Morbach C, Bender S, Jay PY, Vest J, Bulwer BE, Prasad N, Solomon SD, Skali H. Effects of vutrisiran on cardiac structure and function in patients with transthyretin amyloidosis with cardiomyopathy: secondary outcomes of the HELIOS-B trial. Nat Med. 2025 Oct;31(10):3560-3568. doi: 10.1038/s41591-025-03851-z. Epub 2025 Aug 6.

Study record dates

Study Major Dates

• Study Start (Actual): November 26, 2019
• Primary Completion (Actual): May 8, 2024
• Study Completion (Estimated): December 2, 2026

Study Registration Dates

• First Submitted: November 4, 2019
• First Submitted That Met QC Criteria: November 4, 2019
• First Posted (Actual): November 6, 2019

Study Record Updates

• Last Update Posted (Estimated): January 12, 2026
• Last Update Submitted That Met QC Criteria: December 18, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: TTR; Amyloidosis; Cardiomyopathy; RNAi therapeutic; ATTR; Transthyretin; TTR-mediated amyloidosis; Amyloidosis, Hereditary; Amyloidosis, Hereditary, Transthyretin-Related; Familial Amyloidosis; Transthyretin amyloid cardiomyopathy; TTR cardiomyopathy; ATTR-CM; Wild-type TTR; V122I; TTR amyloidosis; Amyloidosis, Wild Type
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Heart Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Proteostasis Deficiencies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Cardiomyopathies; Amyloidosis; Amyloidosis, Familial; Amyloidosis, Hereditary, Transthyretin-Related
• Other Study ID Numbers: ALN-TTRSC02-003; 2023-508366-15-00 (Ctis)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU.

Access to data may be declined where there is likelihood a patient could be identified or other feasibility issue, where there is a potential conflict of interest, a planned business activities or an actual or potential competitive risk. Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Timeframes for data access may vary and can take up to 6 months or more.

Requests for access to data can be submitted via the website www.vivli.org. Questions can also be directed to datasharing@alnylam.com.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No