Alnylam
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Enrollment Complete
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.
Trial at a Glance
Trial ID
ALN-TTRSC02-002
Condition
Transthyretin Amyloidosis (ATTR)
Drug/Treatment
Patisiran, Vutrisiran
Does this trial use a placebo?
No
Trial Type
Interventional
Number of Participants
164 participants
Trial dates
February 14, 2019 - October 1, 2026
For more information:NCT03759379
Who can participate?
AGE
18 to 85 Years
SEX
All
ACCEPTS HEALTHY VOLUNTEERS?
No
Study Summaries
View and download easy-to-read summaries of this study.
Protocol Summary:
Results Summary:
Inquire about a clinical trial
Contact Alnylam directly with any questions
about our clinical trials.
Copyright © 2025 Alnylam Pharmaceuticals, Inc. — All Rights Reserved
Follow Us: