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).
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Sponsor: Alnylam Pharmaceuticals
Enrolling: Male and Female Patients
IRB Number: AAAS4033
U.S. Govt. ID: NCT03759379
Contact: Raisy Fayerman, CRC: 212-305-6035 / rf2632@cumc.columbia.edu
Additional Study Information: HELIOS-A is a global Phase 3 randomized, open-label study designed to evaluate the safety and efficacy of ALN-TTRSC02 in adult patients with hATTR amyloidosis experiencing neurologic symptoms brought on by the disease. The study will also evaluate any changes in quality of life experienced by study participants. ALN-TTRSC02 utilizes the mechanism of RNA interference (RNAi) to selectively degrade TTR mRNA and thereby reduce the expression of its corresponding protein. The ability to selectively and potently degrade the mRNA offers a targeted approach for the treatment of hATTR amyloidosis. Patients will be randomized 3:1 to ALN-TTRSC02 or patisiran-LNP. Visits are approximately every 3 weeks for the first 18 months of the study. Travel and accommodation support may be available, including the option of study visits provided at home by a home nursing specialist.
This study is closed
Thomas Brannagan, MD
Do You Qualify?
Are you between the ages of 18 and 85? Yes No
Do you have a diagnosis of hATTR amyloidosis with documented TTR mutation? Yes No
Are you able to walk on your own or with 1-2 walking sticks? Yes No
Are you a liver transplant recipient or do you anticipate a liver transplant during the length of the trial? Yes No
Do you have a diagnosis of type I or type II diabetes (5 years)? Yes No
You may be eligible for this study

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For more information, please contact:
Raisy Fayerman, CRC