Status:
COMPLETED
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Lead Sponsor:
Alnylam Pharmaceuticals
Conditions:
Amyloidosis, Hereditary
Transthyretin Amyloidosis
Eligibility:
All Genders
18-85 years
Phase:
PHASE3
Brief Summary
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in participants with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vu...
Eligibility Criteria
Inclusion
- Male or female of 18 to 85 years of age (inclusive);
- Has a diagnosis of hATTR amyloidosis with transthyretin (TTR) mutation;
- Has adequate neurologic impairment score (NIS);
- Has adequate polyneuropathy disability (PND) score;
- Has adequate Karnofsky Performance Status (KPS).
Exclusion
- Had a prior liver transplant or is likely to undergo liver transplantation during the study;
- Has known other (non-hATTR) forms of amyloidosis or leptomeningeal amyloidosis;
- Has New York Heart Association heart failure classification \>2;
- Clinically significant liver function test abnormalities;
- Has known human immunodeficiency virus (HIV), hepatitis C virus (HCV) or hepatitis B virus (HBV) infection;
- Received an experimental drug within 30 days of dosing;
- Received prior TTR-lowering treatment;
- Has other known causes of neuropathy.
Key Trial Info
Start Date :
February 14 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 5 2025
Estimated Enrollment :
164 Patients enrolled
Trial Details
Trial ID
NCT03759379
Start Date
February 14 2019
End Date
November 5 2025
Last Update
December 2 2025
Active Locations (53)
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1
Clinical Trial Site
San Diego, California, United States, 92120
2
Clinical Trial Site
Aurora, Colorado, United States, 80045
3
Clinical Trial Site
Chicago, Illinois, United States, 97239
4
Clinical Trial Site
Baltimore, Maryland, United States, 21205