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. 

Eligibility Criteria*

• 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 Design*

• 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 

Status

Phase 2, Open, Enrolling

Therapy Approach

Kinase Inhibitor

Study Sponsor

Ipsen

*For the complete list of eligibility criteria and details on this study, visit ClinicalTrials.gov and enter Identifier NCT05039515.

Participating Clinical Sites (as of March 18, 2024)

Aichi Children's Health and Medical Center (New)
Japan

Nagoya University Hospital 
Japan

The University of Tokyo Hospital (New)
Japan

Instituto Nacional De Rehabilitacion 
Mexico

VU University Medical Center (VUMC) (New)
Netherlands

Seoul National University Hospital
South Korea

Hospital Universitario y Politecnico La Femerge 
Spain

Ramon y Cajal University Hospital
Spain

Norrlands Universitetssjukhus
Sweden

Royal National Orthopaedic Hospital (Stanmore)
United Kingdom

Children's Hospital of Philadelphia
United States

Mayo Clinic 
United States

The Perelman School of Medicine - University of Pennsylvania
United States

University of California, San Francisco
United States

FALKON Trial Website

More information on the FALKON Trial 
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The IFOPA does not endorse nor recommend specific clinical trials. Please speak with your doctor if you are interested in participating in a clinical trial.