HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Public ClinicalTrials.gov record NCT03759379. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT03759379
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 164 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years to 85 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 13, 2019
- Primary completion
- Nov 9, 2020
- Completion
- Nov 4, 2025
- Last update posted
- Jan 11, 2026
2019 – 2025
United States locations
- U.S. sites
- 15
- U.S. states
- 15
- U.S. cities
- 15
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Clinical Trial Site | La Mesa | California | 91942 | — |
| Clinical Trial Site | Aurora | Colorado | 80045 | — |
| Clinical Trial Site | Jacksonville | Florida | 32224 | — |
| Clinical Trial Site | Chicago | Illinois | 60611 | — |
| Clinical Trial Site | Fairway | Kansas | 66205 | — |
| Clinical Trial Site | Baltimore | Maryland | 21224 | — |
| Clinical Trial Site | Boston | Massachusetts | 02118 | — |
| Clinical Trial Site | Rochester | Minnesota | 55902 | — |
| Clinical Trial Site | St Louis | Missouri | 63130 | — |
| Clinical Trial Site | New York | New York | 10032 | — |
| Clinical Trial Site | Chapel Hill | North Carolina | 27599 | — |
| Clinical Trial Site | Columbus | Ohio | 43210 | — |
| Clinical Trial Site | Portland | Oregon | 97239 | — |
| Clinical Trial Site | Philadelphia | Pennsylvania | 19104 | — |
| Clinical Trial Site | Dallas | Texas | 75246 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 49 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03759379, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jan 11, 2026 · Synced Sep 3, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03759379 live on ClinicalTrials.gov.