HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
ClinicalTrials.gov ID: NCT03759379
Sponsor: Alnylam Pharmaceuticals
Information provided by: Alnylam Pharmaceuticals (Responsible Party)
Last Update Posted: 2023-09-22
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 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 SC injection once every 6 months (q6M) or q3M in the Randomized Treatment Extension (RTE) Period.
Official Title:
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)
Intervention / Treatment:
- Drug: Patisiran
- Drug: Vutrisiran
| Category | Value |
|---|---|
| Study Start (Actual) | 2019-02-14 |
| Primary Completion (Actual) |
2020-11-10
|
| Study Completion (Estimated) | 2026-10 |
| Enrollment (Actual) | 164 |
| Study Type | Interventional |
| Phase | Phase 3 |
| Other Study ID Numbers |
ALN-TTRSC02-002 2018-002098-23 (EudraCT Number) |