Revusiran in patients with TTR-mediated FAP
Research type
Research Study
Full title
An Open-Label Study To Evaluate The Efficacy And Safety Of Revusiran In Patients With Transthyretin-Mediated Familial Amyloidotic Polyneuropathy With Disease Progression Post Orthotopic Liver Transplant
IRAS ID
187864
Contact name
Philip Hawkins
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc
Eudract number
2015-002603-29
Duration of Study in the UK
2 years, 0 months, 1 days
Research summary
Transthyretin-mediated amyloidosis is a progressively disabling disease caused by a mutation in the TTR gene. TTR is a protein produced mainly in the liver and is normally a carrier for substances such as vitamin A in the blood. TTR amyloid deposits build up in the heart causing familial cardiac amyloidosis (FAC) or in the nerves causing familial amyloidotic polyneuropathy (FAP). FAP mainly affects the nervous system and is characterised by symptoms such as limb weakness, loss of sensation, bladder and bowel disturbances, and eventual inability to walk. It is estimated that FAP affects approximately 5,000-10,000 people worldwide, with a life expectancy of 10 years from onset of symptoms.
Liver transplant is currently used as a treatment option for FAP, however transplantations have limitations including limited organ availability and life-threatening complications due to rejection or infection. Despite undergoing liver transplantation as a potential cure for FAP, many patients experience progression of their underlying disease after transplant, most commonly presenting as worsening of nerve damage and/or heart disease. There is a significant need for novel therapies that have the ability to halt disease progression.
Alnylam Pharmaceuticals, Inc. have developed a new drug, revusiran, that has been designed to help deliver small interfering ribonucleic acids (siRNA) to the liver. The siRNA blocks production of the TTR protein in the liver leading to lower amounts of TTR in the blood and potentially less TTR amyloid deposits.
The study is being done to evaluate the change in TTR levels in the blood as well as evaluate patient responses to revusiran when the drug is taken once a day for 5 days, once on Day 7, and then once a week for 18 months.
This is an open-label multicentre study which will take place across Europe. It is anticipated that approximately 12 patients aged ≥ 18 years will be enrolled worldwide.REC name
East of England - Cambridge South Research Ethics Committee
REC reference
15/EE/0355
Date of REC Opinion
19 Oct 2015
REC opinion
Further Information Favourable Opinion