Pamrevlumab or placebo in male patients with ambulatory DMD & treated with corticosteroids
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in ambulatory subjects with Duchenne Muscular Dystrophy (DMD)
IRAS ID
1003519
Contact name
Bassem Elmankabadi
Contact email
Sponsor organisation
FibroGen, Inc.
Eudract number
2020-000699-39
Research summary
Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterised by progressive muscle degeneration and weakness due to the alterations of dystrophin, a protein that stabilises muscle cells. Over time, the loss of dystrophin function leads to loss of muscle fibres and to their replacement by fibrosis and fat, making muscle tissues less able to complete everyday activity.
Symptoms are usually present before five years of age and wheelchair dependency occurs before the age of 13 years. In about one third of the DMD patients there are cognitive and/or behavioural abnormalities. After 18 years almost all patients are affected by cardiomyopathy. Only a few DMD patients survive beyond the third decade due to respiratory complications and heart failure due to cardiomyopathy.
There is no known cure for DMD. At present, treatment is almost exclusively symptomatic, including medical and physical therapies to improve cardiac and respiratory function as well as corticosteroids to improve skeletal muscle strength. However, corticosteroids are not formally approved for treatment of DMD in all countries and their use is often limited due to significant side effects. Development of new treatments is warranted to alleviate disease progression.
FibroGen, Inc. sponsor of the present study, is developing a medicinal product (pamrevlumab) for improvement of muscle performance. At approximately 55 centres globally, DMD patients who are able to walk will receive pamrevlumab or placebo in combination with systemic corticosteroid treatment for 52 weeks. The study will investigate the safety and efficacy of pamrevlumab.
This study includes an open label extension that will offer treatment with pamrevlumab and systemic corticosteroids for participants that complete 52 weeks of treatment and meet the eligibility requirements. The open label extension study will look at the longer term safety effects of pamrevlumab.
REC name
South West - Central Bristol Research Ethics Committee
REC reference
21/SW/0019
Date of REC Opinion
6 Apr 2021
REC opinion
Further Information Favourable Opinion