HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Active, not recruiting
Alnylam Pharmaceuticals
Phase 3
2019-02-14
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02)
in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). Participants will
receive vutrisiran or the reference comparator patisiran during the Treatment Period. The
Treatment Period is followed by a Treatment Extension Period during which all participants in
the patisiran group will switch to vutrisiran. This study will use the placebo arm of the
APOLLO study (NCT01960348) as an external comparator for the primary and most other efficacy
endpoints.
HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Active, not recruiting
Alnylam Pharmaceuticals
Phase 3
2019-11-26
This study will evaluate the efficacy and safety of vutrisiran 25 mg administered
subcutaneously (SC) once every 3 months (q3M) compared to placebo in patients with ATTR
amyloidosis with cardiomyopathy.
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