HELIOS-B: A Study to Evaluate Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy
- Conditions
- Patients with ATTR amyloidosis (hATTR or wtATTR) with cardiomyopathy
- Registration Number
- JPRN-jRCT2031210402
- Lead Sponsor
- Yamaji Hideomi
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 24
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 (AL amyloidosis) or leptomeningeal amyloidosis.
- Has NYHA Class IV heart failureNYHA 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 eGFR <30 mL/min/1.73 m2.
- 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 & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method