Clinical Trial: ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Phase 3 Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ALN TTRSC in Patients With Transthyretin (TTR) Mediated Familial Amyloid
Brief Summary: The purpose of this study was to evaluate the safety and efficacy of revusiran (ALN-TTRSC) in patients with transthyretin (TTR) mediated Familial Amyloidotic Cardiomyopathy. Dosing has been discontinued; patients are being followed-up for safety.
Detailed Summary:
Sponsor: Alnylam Pharmaceuticals
Current Primary Outcome:
- 6 Minute Walk Distance (6-MWD) [ Time Frame: 18 months ]The difference between Revusiran (ALN-TTRSC) and placebo group in change from baseline to 18 months in the total distance walked in 6 minutes
- Serum TTR levels [ Time Frame: 18 months ]The difference between Revusiran (ALN-TTRSC) and placebo group in the percent reduction in serum TTR levels over 18 months
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Composite cardiovascular (CV) mortality and cardiovascular (CV) hospitalization [ Time Frame: 18 months ]Number of cardiovascular-related deaths and cardiovascular-related hospitalizations in the placebo group compared to the Revusiran (ALN-TTRSC) treatment group
- New York Heart Association (NYHA) Class [ Time Frame: 18 months ]The difference between Revusiran (ALN-TTRSC) and placebo group in the change from baseline to 18 months in the NYHA class
- Kansas City Cardiomyopathy Questionnaire (KCCQ) [ Time Frame: 18 months ]The difference between Revusiran (ALN-TTRSC) and placebo group in the change from Baseline to 18 months in the Kansas City Cardiomyopathy Questionnaire
- Cardiovascular (CV) Mortality [ Time Frame: 18 months ]Number of cardiovascular-related deaths in the placebo group compared to the Revusiran (ALN-TTRSC) treatment group
- Cardiovascular (CV) hospitalization [ Time Frame: 18 months ]Number of cardiovascular-related hospitalizations in the placebo group compared to the Revusiran (ALN-TTRSC) treatment group
- All-cause mortality [ Time Frame: 18 months ]Total number of deaths in the placebo group compared to the Revusiran (ALN-TTRSC) treatment group
Original Secondary Outcome: Same as current
Information By: Alnylam Pharmaceuticals
Dates:
Date Received: December 12, 2014
Date Started: December 2014
Date Completion:
Last Updated: May 23, 2017
Last Verified: May 2017