Clementia 202
Research type
Research Study
Full title
A Phase 2, Open-Label Extension, Efficacy and Safety Study of a RARγ-Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects with Fibrodysplasia Ossificans Progressiva (FOP)
IRAS ID
186927
Contact name
Richard Keen
Contact email
Sponsor organisation
Clementia Pharmaceuticals Inc.
Eudract number
2014-002496-28
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 2 months, 20 days
Research summary
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flareups) and abnormal bone formation (called heterotopic ossification (HO)) in soft tissue such as muscle, tendon, or ligament. This begins in early childhood and results in progressive loss of movement.
Approximately 1 in every 2 million people will have FOP. It is caused by a gene mutation. Currently there are no effective medical treatments to prevent the HO. Most treatments are aimed at managing the symptoms rather than the underlying cause. The identification of the specific mutation that causes FOP now allows for a specific target for drug development.Palovarotene is an orally bioavailable retinoic acid receptor gamma (RARγ) selective agonist and was previously under development by Roche as a potential therapeutic in patients with chronic obstructive pulmonary disease (COPD). It is now being developed by Clementia Pharmaceuticals Inc. as a repurposed drug for the treatment of FOP. RARγ agonists potently stop HO by redirecting the fate of mesenchymal stem cells (MSCs).
In this open label extension study, patients who completed study PVO-1A-201 will be able to continue to be followed up for up to 36 months and receive Palovarotene should the subject experience flare ups (up to 2 new flare ups). This study will continue to test if Palovarotene is safe and effective in the treatment of Fibrodysplasia Ossificans Progressiva(FOP), by preventing new bone formation following flareups.
Clementia Pharmaceuticals Inc., is Sponsoring this study, approximately 40 participants are expected to take part in this study at sites in the US,Europe and Argentina.
REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
15/LO/1669
Date of REC Opinion
13 Nov 2015
REC opinion
Further Information Favourable Opinion