To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva

Study Overview
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Brief Summary
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This Phase 2, Randomized, Double-Blind, Placebo-Controlled Study is intended to evaluate the Efficacy, Safety, and Tolerability and PK of INCB000928 administered to participants with a clinical diagnosis of fibrodysplasia ossificans progressiva (FOP).

Official Title
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A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva

Conditions
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Fibrodysplasia Ossificans Progressiva (FOP)

Intervention / Treatment
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* Drug: INCB000928
* Drug: placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Female and male adults and adolescents ≥ 12 years of age with a diagnosis of FOP. Willingness to avoid pregnancy or fathering children based on the criteria below. Willing and able to undergo low-dose WBCT (excluding the head) imaging without requiring intubation. Further inclusion criteria apply. Exclusion Criteria: Pregnant or breast-feeding. CAJIS score ≥ 24. FOP disease severity that in the investigator's opinion precludes participation. Any clinically significant medical condition other than FOP that would, in the investigator's judgment, interfere with full participation in the study, pose a significant risk to the participant, or interfere with interpretation of study data. Chronic or current active infectious disease requiring systemic antibiotic, antifungal, or antiviral treatment. HIV, HBV, or HCV infection. Note: Further exclusion criteria apply.

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 99 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Group A: INCB000928<br>Participants will receive INCB000928 for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 52 weeks. | Drug: INCB000928<br>* INCBG000928 will be administered QD orally.<br>* Other names: INB000928 will be administered QD orally;Drug: placebo<br>* placebo will be administered QD orally<br>|
| Placebo Comparator: Group B: Placebo followed by INCB000928<br>Participants will receive placebo for 24 weeks (double-blind period). Participants who completed the double-blind period will receive INCB000928 in the 52 week open-label extension period. | Drug: placebo<br>* placebo will be administered QD orally<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Double Blind Period: Total volume of new heterotopic ossification (HO) | HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period. | Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Double-blind Period: Total number of new flares | Defined as number of new flares compared to baseline during the double-blind period. | Baseline, Week 12 and Week 24 | 
| Double-blind Period: Proportion of participants with a clinically meaningful improvement in the flare-related symptoms | Flare related symptoms will be assessed by via an electronic PRO during the double-blind period. | Baseline, Week 12 and Week 24 | 
| Number of Participants with Treatment Emergent Adverse Events (TEAE) | Defined as any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug. | Up to 80 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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fibrodysplasia ossificans progressiva (FOP), heterotopic ossification