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NCT03759379 · ClinicalTrials.gov registry record · Phase 3
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
A Phase 3 study, sponsored by Alnylam Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 164
- Enrollment target
NCT03759379: Completed Phase 3 study, sponsored by Alnylam Pharmaceuticals.
NCT03759379 is a Phase 3 study that has completed, run by Alnylam Pharmaceuticals. The registered enrollment target is 164 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (79% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03759379, a Phase 3 study, has completed, sponsored by Alnylam Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 164 participants
- Enrollment target
Study 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 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.
Primary Outcome
The mNIS+7 is a composite score that measures neurologic impairment which includes the following components: physical exam of lower limbs, upper limbs and cranial nerves to assess motor strength/weakness, electrophysiologic measurement of small and large nerve fiber function, sensory testing and postural blood pressure. The mNIS+7 is scored from 0 (no impairment) to 304 points (maximum impairment). A higher score indicates a worse outcome.
Interventions
- DRUG Vutrisiran
- DRUG Patisiran
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 164 participants |
| Start Date | 2019-02-14 |
| Est. Completion | 2025-11-05 |
| Phase | Phase 3 |
What the finished NCT03759379 record still lists
NCT03759379 is an interventional study that assigns participants to a tested intervention. The registered 164 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (79% lower).
The record links to 0 conditions, and to 2 interventions - of which Vutrisiran is the first listed.
NCT03759379 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03759379 about?
NCT03759379 is a clinical study titled "HELIOS-A: A Study of Vutrisiran (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 participants with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will receive vutrisiran subcutaneous (SC) injection once every 3 months (q3M) or the reference comparator patisiran...
What is the current status of trial NCT03759379?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 164 participants. The study started on 2019-02-14. Estimated completion is 2025-11-05.
What interventions are being tested in trial NCT03759379?
The interventions under investigation include: Vutrisiran (DRUG), Patisiran (DRUG).
Who is sponsoring clinical trial NCT03759379?
This trial is sponsored by Alnylam Pharmaceuticals, which has 49 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03759379's enrollment target sits among peer trials
164 1470th of 2000 higher than 530 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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