Long-term safety and efficacy of revusiran in patients with TTR-FAC
Research type
Research Study
Full title
A Multicenter, Multinational, Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Revusiran in Patients with Transthyretin-mediated Familial Amyloidotic Cardiomyopathy
IRAS ID
205612
Contact name
Phillip Hawkins
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc.
Eudract number
2015-005333-49
Duration of Study in the UK
3 years, 6 months, 15 days
Research summary
Familial amyloidotic cardiomyopathy (FAC) is a fatal condition resulting from the progressive deposition of a transport protein, called Transthyretin, in the heart and sometimes the nerves. Eventually, progression of the disease in the heart muscle limits patient capabilities (restrictive cardiomyopathy) and eventually heart failure is seen. Additionally patients often experience abnormal heart rhythms.
The purpose of this study is to see if long-term treatment with revusiran is safe and can be tolerated in patients with FAC. This includes studying the side effects of revusiran, as well as the effect of revusiran on the heart (echocardiograpy) as well as physical function (6 minute walk test) and quality of life (questionnaires).
This is a multicentre, multinational, open-label extension study for participants who have previously completed the ALN-TTRSC-004 parent study.
Participants will receive revusiran once every week for the duration of the study (up to 24 months). Revusiran will be administered by injections under the skin at a dose or 500mg or 250mg (if the participant received the reduced dose as part of the ALN-TTRSC-004 parent study or if the participant experiences an adverse event which meets the dose reduction criteria). Site staff will phone participants approximately 4 weeks after their last dose of revusiran to assess any changes in adverse events or medication. Participants will undergo efficacy and/or safety assessments at Screening/Baseline, each month for first 3 months and then at 3- to 12-month intervals, as outlined in the Schedule of Assessments. This study is to be conducted at approximately 65 study sites worldwide.
REC name
London - Fulham Research Ethics Committee
REC reference
16/LO/0876
Date of REC Opinion
30 Jun 2016
REC opinion
Further Information Favourable Opinion