APOLLO-B
Research type
Research Study
Full title
APOLLO-B: A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Patisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)
IRAS ID
268594
Contact name
Julian Gillmore
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc.
Eudract number
2019-001458-24
Duration of Study in the UK
2 years, 7 months, 18 days
Research summary
Transthyretin amyloidosis (ATTR) is a rare and serious disease caused by problems with a protein in the body called ‘transthyretin’ (TTR) which is deposited as “amyloid” in tissues. The amyloid impairs the normal function of organs such as the heart, gut and nerves. Currently there are limited treatments for patients whose hearts are affected.\n\nIn this study, the medicine Onpattro® (patisiran) will be tested as a treatment for ATTR in the heart. Onpattro is a ‘small interfering RNA’ (siRNA), a very short piece of synthetic genetic material that has been designed to attach to and block the genetic material in the cell that makes transthyretin. Inhibiting transthyretin production reduces the amyloid in the tissues.\n\nOnpattro is approved in the European Union and other countries for the treatment of a type of this disease which runs in families and is called hereditary ATTR (hATTR) amyloidosis. It is approved for patients with hATTR amyloidosis who have nerve damage as a result of amyloid.\n\nThis study will investigate the effectiveness and safety of patisiran in approximately 300 patients with ATTR amyloidosis whose hearts are affected as a result of amyloid. One study group will receive patisiran. A second study group will receive placebo (a dummy treatment). Neither the investigators nor patients will know the study group of a patient.\n\nThe study starts with a screening period of up to 45 days followed by a 12-month treatment period. A patient will receive intravenous infusions of either patisiran or placebo every 3 weeks. After the first 12-month period, all patients in the study will receive patisiran for an additional 12-months after which there is a 28-day follow-up period. Several tests will be performed such as the 6-minute walk test, physical examinations, blood tests, patient questionnaires, electrocardiogram and echocardiogram.
REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
19/EE/0270
Date of REC Opinion
29 Oct 2019
REC opinion
Further Information Favourable Opinion