NCT05039515

A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP).

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.

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 113 (actual)
• Sponsor: Clementia Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

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 ).

Detailed description

Note on Primary Completion Date and Results Disclosure: The Primary Completion Date (PCD) reflects the date when data collection is completed for all primary outcomes, as defined in the Final Rule (42 CFR Part 11). In this study, safety outcomes are designated as primary and collected through the end of study. Therefore the PCD has been updated to align with this definition. The interim analyses will proceed per the protocol and SAP. Results will be disclosed in line with applicable regulatory timelines.

Conditions

• Fibrodysplasia Ossificans Progressiva

Interventions

Type	Name	Description
DRUG	IPN60130	Immediate-release capsule containing high dose of the drug substance.
DRUG	IPN60130	Immediate-release capsule containing low dose of the drug substance.
DRUG	Placebo	Placebo will be supplied as powder filled hard capsules

Timeline

• Start date: 2021-12-01
• Primary completion: 2029-03-30
• Completion: 2029-03-30
• First posted: 2021-09-09
• Last updated: 2026-04-01

Locations

25 sites across 14 countries: United States, Argentina, Australia, Belgium, Canada, China, France, Italy, Japan, Mexico, Portugal, South Korea, Spain, Sweden

Regulatory

• FDA-regulated drug study