HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
| Status: | Recruiting |
|---|---|
| Conditions: | Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | 18 - 85 |
| Updated: | 3/14/2019 |
| Start Date: | January 18, 2019 |
| End Date: | May 2024 |
| Contact: | Alnylam Clinical Trial Information Line |
| Email: | clinicaltrials@alnylam.com |
| Phone: | 1-877-ALNYLAM |
HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02)
in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will
receive vutrisiran or the reference comparator patisiran during the Treatment Period. The
Treatment Period is followed by a Treatment Extension Period during which all participants in
the patisiran group will switch to vutrisiran. This study will use the placebo arm of the
APOLLO study (NCT01960348) as an external comparator for the co-primary and most other
efficacy endpoints.
in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will
receive vutrisiran or the reference comparator patisiran during the Treatment Period. The
Treatment Period is followed by a Treatment Extension Period during which all participants in
the patisiran group will switch to vutrisiran. This study will use the placebo arm of the
APOLLO study (NCT01960348) as an external comparator for the co-primary and most other
efficacy endpoints.
Inclusion Criteria:
- 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 Criteria:
- 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.
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