MOVE Study
Research type
Research Study
Full title
A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)
IRAS ID
232097
Contact name
Richard Keen
Contact email
Sponsor organisation
Clementia Pharmaceuticals
Eudract number
2017-002541-29
Duration of Study in the UK
2 years, 7 months, 3 days
Research summary
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterised by the growth of bone (heterotopic ossification, HO) within muscles, tendons, and ligaments often associated with painful, recurrent episodes of soft tissue swelling (flare ups). The formation of HO begins in early childhood and result in segments, sheets and ribbons of extra bone developing through the body and across joints progressively restricting movement. Rapidly growing bony spurs have been known to protrude through the skin causing pain and risk of infection. Periods of flare-up are interspersed with variable-length intervals of apparent remission. The majority of patients with FOP are confined to a wheelchair by the third decade of life, and require caregiver assistance to perform daily activities. Thoracic insufficiency syndrome leads to a markedly shortened survival.
Currently there are no effective medical treatment options to prevent the formation of HO in FOP patients, and until the Clementia program, there have been no well-controlled trials of therapeutics in FOP. Removal of bone is avoided, because surgical trauma to tissues is likely to induce additional bone formation.
There are about 800 cases of FOP globally and a recurrent point mutation is identified in most affected individuals. This provides a specific target for drug development. Retinoid signalling has been observed to inhibit formation of cartilage, which could result in the interruption of bone formation. Palovarotene is an orally bioavailable retinoic acid, previously tested in humans for treatment of COPD.
This study, sponsored by Clementia Pharmaceuticals Inc., aims to investigate the safety and efficacy of daily oral palovarotene with increased dosing during flare-ups in the treatment of FOP. A total of up to 80 subjects worldwide will be treated for 24 months; assessments will include imaging by whole body computed tomography, patient-reported outcome measures, electrocardiograms, and blood tests for safety and pharmacokinetics.REC name
East of England - Essex Research Ethics Committee
REC reference
17/EE/0449
Date of REC Opinion
14 Dec 2017
REC opinion
Further Information Favourable Opinion