Helios-B : A Study to Evaluate Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy
- Conditions
- Transthyretin Amyloidosis (ATTR) With CardiomyopathyCardiomyopathy
- Registration Number
- LBCTR2020104517
- Lead Sponsor
- Alnylam Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 600
1. 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.
2. Has medical history of heart failure (HF) with at least 1 prior hospitalization for HF OR clinical evidence of HF.
1. Has known primary amyloidosis or leptomeningeal amyloidosis
2. Has New York Heart Association (NYHA) Class IV heart failure
3. Has NYHA Class III heart failure AND is at high risk based on pre-specified criteria
4. Has a polyneuropathy disability (PND) Score IIIa, IIIb, or IV at the Screening visit
5. Has estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m^2
6. Has received prior TTR-lowering treatment
7. Has other non-TTR cardiomyopathy, hypertensive cardiomyopathy, cardiomyopathy due to valvular heart disease, or cardiomyopathy due to
ischemic heart disease
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method ame: Composite Endpoint of All-Cause Mortality and Recurrent Cardiovascular (CV) Events (CV Hospitalizations and Urgent Heart Failure [HF] Visits);Timepoints: [ Time Frame: 30-36 months];Measure: All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) will be compared between treatment groups using an Andersen-Gill model.
- Secondary Outcome Measures
Name Time Method