FOP patients harbor mutations in the ALK2 protein (also known as ACVR1) that drives excessive bone morphogenetic protein (BMP) signaling, which regulates cartilage and bone development.
Zilurgisertib (INCB000928) is an oral investigational drug designed to target and block this disease-causing mutant FOP protein hyperactivity. In preclinical studies, zilurgisertib demonstrated potency for the target kinase, selectivity, safety and strong suppression of heterotopic ossification (HO) in animal models.
About the Clinical Trial
The PROGRESS Phase 2 trial is a global, multi-center, placebo-controlled trial. Approximately 60 patients 12 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 efficacy, safety and tolerability of zilurgisertib compared with placebo. Total volume of new HO will be evaluated along with the number of new flares, flare-related symptoms and treatment emergent adverse events.
Eligibility Criteria*
- AGE: ≥ 12
- DISEASE ACTIVITY: Signs and symptoms associated with FOP flare-ups, worsening of joint function, or new heterotopic ossification (HO)
- MUTATIONS: R206H and other mutations of the ACVR1 gene resulting in HO
Study Design*
- STUDY TYPE: Interventional
- RANDOMIZED STUDY: Yes
- PLACEBO CONTROLLED: Yes, participants will be randomized to zilurgisertib or placebo
- LENGTH OF PARTICIPATION: 24 week (double-blind portion); 52 week (open-label portion)
- NUMBER OF STUDY VISITS: 9 onsite and 15 remote
Status
Phase 2, Active, Enrolling
Therapy Approach
Kinase Inhibitor
Study Sponsor
Incyte Corporation
*For the complete list of eligibility criteria and details on this study, visit ClinicalTrials.gov and enter Identifier NCT05090891.
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.
