Phase II DB PC Study for Efficacy Safety with INCB000928 Patients FOP
Research type
Research Study
Full title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants with Fibrodysplasia Ossificans Progressiva
IRAS ID
301533
Contact name
Richard Keen
Contact email
Sponsor organisation
Incyte Corporation
Eudract number
2021-002286-17
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
144,841, IND Number
Duration of Study in the UK
2 years, 10 months, 11 days
Research summary
Fibrodysplasia ossificans progressive (FOP) is an ultra-rare genetic disease of heterotopic ossification (HO). The prevalence of FOP is approximately 1.36 per million people.
During the first decade of life, episodic flare-ups (painful soft tissue swellings) occur with most resulting in HO.
There are no approved disease modifying treatments for FOP.
The study will enroll approximately 60 adult and adolescent participants (≥ 12 years of age).
The study consists of three parts
Participants will be screened for up to 32 days to check if they are eligible for the study.
Approximately 60 participants will take the oral study drug daily for 24 weeks. (Blinded)
A Data Management Committee will check the Safety Results.
A further Open Label Extension period for participants to take the oral study drug daily for up to 52 weeks.
A Post Treatment Safety Follow-up for participants will be for 4 Weeks after the study drug has been stopped.REC name
London - Brent Research Ethics Committee
REC reference
22/LO/0037
Date of REC Opinion
13 Apr 2022
REC opinion
Further Information Favourable Opinion