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
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Contact Alnylam directly with any questions
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