FALKON
Research type
Research Study
Full title
A Phase 2 study to assess the efficacy and safety of 2 dosage regimens of oral fidrisertib (IPN60130) for the treatment of fibrodysplasia ossificans progressiva in male and female paediatric and adult participants
IRAS ID
291202
Contact name
Richard Keen
Contact email
Sponsor organisation
Clementia Pharmaceuticals Inc, an Ipsen Company
Eudract number
2020-002858-24
Clinicaltrials.gov Identifier
140834, IND
Duration of Study in the UK
years, months, days
Research summary
This study is a Phase 2 double-blind study to assess the efficacy and safety of 2 doses of oral IPN60130 for the treatment of fibrodysplasia ossificans progressiva (FOP) in male and female participants 5 years of age and older. FOP is is an ultra-rare and severely disabling genetic disease characterized by bone formation in muscles, tendons, and ligaments. These bone formations accumulate, progressively restricting movement. The prognosis of patients with FOP is poor and life expectancy is low. Presently there are no approved medical treatment options to prevent the formation of bone in FOP.
There are two parts of this study, Part A and Part B. In Part A, participants will receive either a high or low dose of IPN60130, or placebo. After completing Part A, all participants will continue into Part B and receive the same dose as in Part A (if they had received IPN60130), or will receive one of the 2 doses (if they were on placebo). Overall participation in the study (both Part A and Part B) will last approximately 26 months.REC name
London - City & East Research Ethics Committee
REC reference
21/LO/0602
Date of REC Opinion
1 Nov 2021
REC opinion
Further Information Favourable Opinion