Active — Not RecruitingPhase 3

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

United States655 participantsStarted 2019-11-26

Plain-language summary

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.

Who can participate

Age range18 Years – 85 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population

Timeframe: 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

Timeframe: Up to Month 36

Trial details

NCT IDNCT04153149

SponsorAlnylam Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-05-08

Results submitted2025-06-18

View on ClinicalTrials.gov More Transthyretin Amyloidosis (ATTR) With Cardiomyopathy trials

Who can participate

Age range18 Years – 85 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits) in the Overall Population

Timeframe: 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

Timeframe: Up to Month 36