Phase 3 ALN-TTRSC in Familial Amyloidotic Cardiomyopathy
Research type
Research Study
Full 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 Amyloidotic Cardiomyopathy (FAC)
IRAS ID
167022
Contact name
Phillip Hawkins
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc.
Eudract number
2014-003835-20
Duration of Study in the UK
3 years, 6 months, 1 days
Research summary
Familial amyloid cardiomyopathy (FAC), results from collection of mutant/wild-type transport protein, called Transthyretin, in the heart.
Eventually, progression of the disease in the heart muscle limits patient capabilities (restrictive cardiomyopathy) and heart failure is seen.
The purpose of this study is to collect information about how patients with FAC respond to the study drug over 18 months. This includes studying any potential improvements in their disease and any side effects that they may have from the study drug. In addition, the study will examine how the body handles (distributes, breaks down, and eliminates) the study drug by measuring the amount of drug that gets into the blood after dosing.
This is a multicentre, multinational, randomised, double-blind study comparing the efficacy and safety of ALN-TTRSC to placebo in patients with FAC caused by Transthyretin.
Participants will be randomised to receive either 500 mg ALN-TTRSC or placebo in a 2:1 ratio (ALN-TTRSC:placebo) in a blinded manner.
Treatment groups will be balanced on entry depending on severity of disease based on assessments of patients’ heart function, how far they can walk in 6minutes under controlled conditions and the type of TTR mutation.
Patients will receive ALN-TTRSC or placebo injections for 5 daily doses during the first week and then once every week for approximately 18 months. Patients will attend a follow-up visit approximately 4 weeks after their last dose.
Patients will undergo efficacy and safety assessments at Screening/Baseline,
monthly for 3 months and then at 3-6 month intervals. Additionally, pharmacokinetics (PK) of ALN-TTRSC and its effect on quality of life assessments will be performed at selected time points.Study Sites: This study is to be conducted at approximately 60 study sites worldwide.
REC name
London - Hampstead Research Ethics Committee
REC reference
14/LO/2014
Date of REC Opinion
26 Nov 2014
REC opinion
Further Information Favourable Opinion