A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP).
Stopped early · Phase 2 · Has a placebo group
Conditions studied: Fibrodysplasia Ossificans Progressiva
In brief
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by the presence of bone in soft tissue where bone normally does not exist, known as Heterotopic Ossification (HO). It is often associated with painful, recurrent episodes of soft tissue swelling (flare-ups) that lead to abnormal stiffening and immobility (ankyloses) of major joints with cumulative and irreversible loss of movement and disability. This study will evaluate the efficacy of 2 dosing regimens of IPN60130 in inhibiting new HO volume compared with placebo (a dummy treatment) in adult and paediatric participants with FOP. It will be assessed by a scan (provides internal images of the body) called low dose Whole Body Computed Tomography (WBCT), excluding head. Adults and participants 5 years of age or older are also eligible for a sub study to evaluate HO lesions assessed by another type of scan, Fluorine-18-labelled natrium fluoride Positron Emission Tomography-Computed Tomography (\[18F\]NaF PET-CT ).
Key facts
- Study ID
- NCT05039515
- Run by
- Clementia Pharmaceuticals Inc.
- People needed
- 113
- Starts
- 2021-12-01
- Expected to finish
- 2026-03-27
- Last updated by the study team
- 2026-04-24
Who can join
Age: 5 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of California San Francisco (UCSF) — San Francisco, California, United States
- Mayo Clinic — Rochester, Minnesota, United States
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- The Perelman School of Medicine - The University of Pennsylvania — Philadelphia, Pennsylvania, United States
- Hospital Italiano de Buenos Aires — Buenos Aires, Argentina
- Royal North Shore Hospital - New South Wales — Sydney, Australia
- University Hospitals Leuven — Leuven, Belgium
- University of Alberta, Alberta Health Services (AHS) — Edmonton, Canada
- University Health Network (UHN), Toronto General Hospital (TGH) — Toronto, Canada
- Children's Hospital Capital Institute of Pediatrics (CIP) — Beijing, China
- Peking Union Medical College Hospital — Beijing, China
- Shangai Children Medical Center — Shanghai, China
- Tongi University - Tongi Hospital — Shanghai, China
- Groupe Hospitalier Necker Enfants Malades — Paris, France
- Hopital Lariboisiere — Paris, France
- Irccs Gaslini Institute — Genoa, Italy
- Nagoya University Hospital — Nagoya, Japan
- Aichi Children's Health and Medical Center — Ōbu, Japan
- The University of Tokyo Hospital — Tokyo, Japan
- Instituto Nacional De Rehabilitacion — Mexico City, Mexico
- Hospital Center Lisbon North, E.P.E- Hospital Santa Maria — Lisbon, Portugal
- Seoul National University Hospital — Seoul, South Korea
- Hospital Universitario Ramon y Cajal — Pozuelo de Alarcón, Spain
- Hospital Universitario Y Politecnico La Femerge — Valencia, Spain
- Norrlands Universitetssjukhus — Umeå, Sweden
Full record on ClinicalTrials.gov
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