HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
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|ClinicalTrials.gov Identifier: NCT04153149|
Recruitment Status : Active, not recruiting
First Posted : November 6, 2019
Last Update Posted : May 23, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Transthyretin Amyloidosis (ATTR) With Cardiomyopathy||Drug: Vutrisiran Drug: Sterile Normal Saline (0.9% NaCl)||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||655 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||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)|
|Actual Study Start Date :||November 26, 2019|
|Estimated Primary Completion Date :||February 2024|
|Estimated Study Completion Date :||December 2026|
Experimental: Vutrisiran 25 mg
Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) during the double-blind period.
Vutrisiran 25 mg will be administered by SC injection q3M.
Other Name: ALN-TTRSC02
Placebo Comparator: Placebo
Participants will receive placebo during the double-blind period.
Drug: Sterile Normal Saline (0.9% NaCl)
Sterile normal saline (0.9% NaCl) will be administered by SC injection q3M.
- Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) [ Time Frame: 30-36 months ]All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) will be compared between treatment groups using an Andersen-Gill model.
- Change from Baseline in 6-Minute Walk Test (6-MWT) at Month 30 [ Time Frame: Baseline, Month 30 ]
- Change from Baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) at Month 30 [ Time Frame: Baseline, Month 30 ]The KCCQ is a 23-item self-administered questionnaire quantifying 6 domains (symptoms, physical function, quality of life, social limitation, self-efficacy, and symptom stability) and 2 summary scores (clinical and overall summary [OS]). Scores are transformed to a range of 0-100, in which higher scores reflect better health status.
- Change from Baseline in Mean Left Ventricular (LV) Wall Thickness by Echocardiographic Assessment at Month 30 [ Time Frame: Baseline, Month 30 ]
- Change from Baseline in Global Longitudinal Strain by Echocardiographic Assessment at Month 30 [ Time Frame: Baseline and Month 30 ]
- Composite Endpoint of All-Cause Mortality and Recurrent All-cause Hospitalizations and Urgent HF Visits [ Time Frame: 30-36 months ]All-cause mortality and recurrent all-cause hospitalizations and urgent HF visits will be compared between treatment groups using an Andersen-Gill model.
- All-cause Mortality [ Time Frame: 30-36 months ]
- Rate of Recurrent CV Events (CV Hospitalizations and Urgent HF Visits) [ Time Frame: 30-36 months ]
- Change from Baseline in N-terminal prohormone B-type Natriuretic Peptide (NTproBNP) at Month 30 [ Time Frame: Baseline, Month 30 ]
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|Ages Eligible for Study:||18 Years to 85 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04153149
|Study Director:||Medical Director||Alnylam Pharmaceuticals|
|Responsible Party:||Alnylam Pharmaceuticals|
|Other Study ID Numbers:||
2019-003153-28 ( EudraCT Number )
|First Posted:||November 6, 2019 Key Record Dates|
|Last Update Posted:||May 23, 2023|
|Last Verified:||May 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Transthyretin amyloid cardiomyopathy
Amyloidosis, Hereditary, Transthyretin-Related
Amyloidosis, Wild Type
Amyloid Neuropathies, Familial
Heredodegenerative Disorders, Nervous System
Nervous System Diseases
Peripheral Nervous System Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors