JRCT ID: jRCT2031210402
Registered date:29/10/2021
HELIOS-B: A Study to Evaluate Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy
Basic Information
Recruitment status | Not Recruiting |
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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) |
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Secondary Outcome |
Key inclusion & exclusion criteria
Age minimum | >= 18age old |
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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 |
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Secondary Sponsor | |
Source(s) of Monetary Support | |
Secondary ID(s) | NCT04153149 |
Contact
Public contact | |
Name | Masanori Ichida |
Address | St Luke's Tover 12F, 8-1 Akashi-cho, Chuo-ku, Tokyo, Japan Tokyo Japan 104-0044 |
Telephone | +81-9063094091 |
Masanori.Ichida@ppd.com | |
Affiliation | PPDSNBL K.K |
Scientific contact | |
Name | Masanori Ichida |
Address | St Luke's Tover 12F, 8-1 Akashi-cho, Chuo-ku, Tokyo, Japan Tokyo Japan 104-0044 |
Telephone | +81-9063094091 |
Masanori.Ichida@ppd.com | |
Affiliation | PPDSNBL K.K |