FOP is caused by a mutation in the ACVR1 gene that results in excessive bone morphogenetic protein (BMP) signaling, which regulates cartilage and bone development. The FOP mutation increases BMP signaling, resulting in the formation of heterotopic bone.
IPN60130 (also known as fidrisertib) is an oral investigational drug designed to selectively target the mutant FOP receptor (ACVR1/ALK2), the underlying cause of FOP. FDA has granted Fast Track Designation to fidrisertib for the treatment of FOP.
About the Clinical Trial
The FALKON Phase 2 trial is a global, multi-center, placebo-controlled trial. Approximately 98 patients 5 years of age or older with the R206H ACVR1 mutation or other FOP variants associated with progressive HO will be enrolled. The trial is designed to evaluate the safety of 2 dosing regimens of oral fidrisertib in inhibiting new HO volume compared with placebo (a dummy treatment) in adult and pediatric participants with FOP.
- AGE: ≥ 15 years of age (The FALKON trial will be expanded to patients ≥ 5 years of age at a later date after safety data has been established)
- DISEASE ACTIVITY: Participants must have disease progression in the preceding year of the screening visit
- MUTATIONS: R206H ACVR1 mutation or other FOP variants
- STUDY TYPE: Interventional
- RANDOMIZED STUDY: Yes
- PLACEBO CONTROLLED: Yes, participants will be randomized to placebo or high or low dose fidrisertib only for the first 12 months (part A); then all participants will receive active treatment in the following 48 months (12 months in part B and 36 months in part C long-term extension)
- LENGTH OF PARTICIPATION: 63 months
- NUMBER OF STUDY VISITS: 17 visits at the clinical site
Phase 2, Open, Enrolling
*For the complete list of eligibility criteria and details on this study, visit ClinicalTrials.gov and enter Identifier NCT05039515.
Participating Clinical Sites (as of September 18, 2023)
Nagoya University Hospital
Aichi Children's Health and Medical Center
VU University Medical Center
Seoul National University Hospital
Hospital Universitario y Politecnico La Femerge
Ramon y Cajal University Hospital
Royal National Orthopaedic Hospital (Stanmore)
Children's Hospital of Philadelphia
University of California, San Francisco
The Perelman School of Medicine - University of Pennsylvania
FALKON Trial Website
More information on the FALKON Trial
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