HELIOS-B
Research type
Research Study
Full title
HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)
IRAS ID
1003107
Contact name
Marianna Fontana
Contact email
Eudract number
2019-003153-28
Research summary
Transthyretin amyloidosis (ATTR) is a rare, serious disease affecting multiple body
systems including the heart. It is caused by certain differences in protein called
transthyretin (TTR). These differences can be caused by genetic mutation (hereditary) or
aging (wild-type). The liver is the main producer of TTR, and TTR then circulates in the
blood stream. As it circulates, abnormal TTR can gradually enter many tissues and
organs. This results in deposits of proteins called amyloid. These deposits can often affect
organs, such as heart, nerves, and gut function. Most common effects of ATTR
amyloidosis are heart failure (cardiomyopathy) and nerve damage (polyneuropathy).
Treatment options for ATTR with cardiomyopathy patients are limited. Therefore, a more
direct therapy may be beneficial. The drug being tested is called vutrisiran. Vutrisiran is
a ribonucleic acid (RNA) interference molecule (RNAi), made up of small pieces of
genetic material, which selectively prevent TTR production in the liver and reduce
amount spreading and depositing as amyloid in organs. Vutrisiran has been well
tolerated in previous and ongoing studies.
This study is sponsored by Alnylam Pharmaceuticals, Inc. Study purpose is to evaluate
effectiveness and safety of vutrisiran in approximately 600 ATTR amyloidosis patients
with cardiomyopathy. As a double-blind study, neither study doctor nor participants
know which treatment they receive. Participants will have a 1 in 2 chance of receiving
25mg of vutrisiran or placebo (inactive substance, like saline) via under-skin injection
every 3 months.
The study consists of 45-day screening, up to 36-month treatment and up to 18-month
follow up periods. Examples of procedures participants will undergo include: walking
tests, health and symptoms questionnaires, echocardiogram, electrocardiogram, physical
exams and biological samples. In addition to trial participation consent, participants will
be asked to sign a medical records release form allowing the study doctor access to
medical records during the study.REC name
London - Surrey Borders Research Ethics Committee
REC reference
20/LO/0629
Date of REC Opinion
21 May 2020
REC opinion
Further Information Favourable Opinion