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

JRCT ID: jRCT2031210402

Registered date:29/10/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Patients with ATTR amyloidosis (hATTR or wtATTR) with cardiomyopathy
• Date of first enrollment: 04/03/2021
• Target sample size: 24
• Countries of recruitment: USA,Japan,Canada,Japan,France,Japan,Germany,Japan,Mexico,Japan,Korea,Japan
• Study type: Interventional

Intervention(s)

Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) during the double-blind period.
Drug: Vutrisiran
Vutrisiran 25 mg will be administered by SC injection q3M.
Other Names: ALN-TTRSC02

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.

Outcome(s)

• Primary Outcome: Composite Endpoint of all-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits)

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: <= 85age old
• Gender: Both

Include 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

Exclude criteria

- 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.

Related Information

• Primary Sponsor: Ichida Masanori
• Secondary ID(s): NCT04153149