HELIOS-A: ALN-TTRSC02 in Patients with hATTR Amyloidosis
Research type
Research Study
Full title
HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
IRAS ID
262126
Contact name
Julian Gillmore
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc.
Eudract number
2018-002098-23
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
Hereditary transthyretin amyloidosis (hATTR) is a rare genetic disease that tends to run in some families. hATTR amyloidosis is caused by certain variations in the gene for a protein called transthyretin, or TTR. The liver is the main organ that produces TTR protein, and TTR then circulates in the blood stream. Abnormal TTR protein can gradually deposit in many tissues and organs of the body, in collections of proteins called amyloid fibrils. These amyloid fibril collections can often affect the function of important organs such as the nerves, the heart, and the gut. This leads to the symptoms of amyloidosis, including weakness, numbness, shortness of breath, lightheadedness, and diarrhoea.
ALN-TTRSC02 is the investigational drug. ALN-TTRSC02 consists of a small interfering ribonucleic acid (siRNA) molecule attached to a sugar molecule, which helps deliver the siRNA to the liver, where the TTR protein is made. The siRNA in ALN-TTRSC02 works by interfering with the ability of RNA (ribonucleic acid), a molecule that the cells use to produce proteins, to make TTR proteins in the liver. ALN-TTRSC02 therefore reduces the body’s TTR protein levels, which may in turn reduce the number of amyloid fibrils in the organs of patients with hATTR amyloidosis.
The main goals of this study are to:
- determine if the study drug, ALN-TTRSC02, is able to improve neurological symptoms, quality of life and physical function in hATTR amyloidosis patients.
- determine if ALN-TTRSC02 is as effective as patisiran-LNP at lowering TTR protein in the blood.
- evaluate the safety of ALN-TTRSC02 and how patients with hATTR amyloidosis are affected by it.It is estimated that about 160 adult patients diagnosed with hATTR amyloidosis will take part in this study. There will be about 80 study centers participating worldwide.
REC name
London - City & East Research Ethics Committee
REC reference
19/LO/0674
Date of REC Opinion
8 Jul 2019
REC opinion
Further Information Favourable Opinion