HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Completed · Phase 3
Conditions studied: Amyloidosis, Hereditary, Transthyretin Amyloidosis
In brief
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 vutrisiran subcutaneous (SC) injection once every 3 months (q3M) or the reference comparator patisiran intravenous (IV) injection once every 3 weeks (q3w) during the 18 month Treatment Period. This study will use the placebo arm of the APOLLO study (NCT01960348) as an external comparator for the primary and most other efficacy endpoints during the 18 Month Treatment Period. Following the 18 Month Treatment Period, all participants will be randomized to receive vutrisiran 50 mg SC injection once every 6 months (q6M) or vutrisiran 25 mg q3M in the Randomized Treatment Extension (RTE) Period. Upon implementation of Amendment 6, participants receiving vutrisiran SC 50 mg q6M will transition to vutrisiran SC 25 mg q3M at their next scheduled dosing.
Key facts
- Study ID
- NCT03759379
- Run by
- Alnylam Pharmaceuticals
- People needed
- 164
- Starts
- 2019-02-14
- Expected to finish
- 2025-11-05
- Last updated by the study team
- 2026-01-12
Who can join
Age: 18 and older, up to 85. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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).
You may not qualify if…
- 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.
Where it is running
- Clinical Trial Site — Aurora, Colorado, United States
- Clinical Trial Site — Jacksonville, Florida, United States
- Clinical Trial Site — Chicago, Illinois, United States
- Clinical Trial Site — Fairway, Kansas, United States
- Clinical Trial Site — Baltimore, Maryland, United States
- Clinical Trial Site — Boston, Massachusetts, United States
- Clinical Trial Site — Rochester, Minnesota, United States
- Clinical Trial Site — St Louis, Missouri, United States
- Clinical Trial Site — New York, New York, United States
- Clinical Trial Site — Chapel Hill, North Carolina, United States
- Clinical Trial Site — Columbus, Ohio, United States
- Clinical Trial Site — Portland, Oregon, United States
- Clinical Trial Site — Philadelphia, Pennsylvania, United States
- Clinical Trial Site — Dallas, Texas, United States
- Clinical Trial Site — Buenos Aires, Argentina
- Clinical Trial Site — Northmead, New South Wales, Australia
- Clinical Trial Site — Brisbane, Queensland, Australia
- Clinical Trial Site — Melbourne, Victoria, Australia
- Clinical Trial Site — Brussels, Belgium
- Clinical Trial Site — Leuven, Belgium
- Clinical Trial Site — Rio de Janeiro, Brazil
- Clinical Trial Site — Sofia, Bulgaria
- Clinical Trial Site — Montreal, Canada
- Clinical Trial Site — Vancouver, Canada
- Clinical Trial Site — La Mesa, California, United States
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.