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

Phase 2

Recruiting

• Conditions: Fibrodysplasia Ossificans Progressiva (FOP)
• Interventions: Drug: INCB000928; Drug: Placebo

• Registration Number: NCT05090891
• Lead Sponsor: Incyte Corporation

Brief Summary

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-10-08
• Study First Submitted QC: 2021-10-14
• Study First Posted: 2021-10-25
• Study Start: 2022-05-05
• Status Verified: 2025-06
• Last Update Submitted: 2025-06-30
• Last Update Posted: 2025-07-01
• Primary Completion: 2025-07-04
• Study Completion: 2032-12-23

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 98

Inclusion Criteria

• Female and male participants:

  • Cohort 1: ≥ 12 years of age.
  • Cohort 2: 6 to < 12 years of age.
  • Cohort 3: 2 to < 6 years of age (after eDMC review of interim data from Cohort 2).

• Clinical 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 1	INCB000928	Participants (≥ 12 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.
Cohort 1	Placebo	Participants (≥ 12 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.
Cohort 2	INCB000928	Participants (6 to \< 12 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.
Cohort 2	Placebo	Participants (6 to \< 12 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.
Cohort 3	INCB000928	Participants (2 to \< 6 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.
Cohort 3	Placebo	Participants (2 to \< 6 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.

Primary Outcome Measures

Name	Time	Method
Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline	Week 24	HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period.

Secondary Outcome Measures

Name	Time	Method
Open-Label Extension: Number of new flares from Week 24	Week 48	Based on Fibrodysplasia Ossificans Progressiva - Patient RepOrted syMPtoms Tool (FOP-PROMPT) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period.
Pharmacokinetics Parameter: Cmax of INCB000928	Baseline, Weeks 12, 24, 48 and 76	Maximum observed concentration.
Pharmacokinetics Parameter: Tmax of INCB000928	Baseline, Weeks 12, 24, 48 and 76	Time to maximum concentration.
Pharmacokinetics Parameter: Cmin of INCB000928	Baseline, Weeks 12, 24, 48 and 76	Minimum observed concentration.
Double Blind Period: Change in the total volume of all HO lesions from baseline	Week 24	HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period.
Open-Label Extension: Number of new HO lesions from Week 24	Week 48	HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period.
Open-Label Extension: Change in the total volume of all HO lesions from Week 24	Week 48	HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period.
Pharmacokinetics Parameter: AUCt of INCB000928	Baseline, Weeks 12, 24, 48 and 76	Area under the plasma concentration-time curve from time 0 to the last quantifiable measurable plasma concentration
Open-Label Extension: Occurrence of new HO lesions from Week 24	Week 48	HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period.
Double Blind Period: Number of new HO lesions from baseline	Week 24	HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period.
Double Blind Period: Total volume of new HO lesions from baseline	Week 24	HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period.
Double Blind Period: Number of new flares from baseline	Week 24	Based on Fibrodysplasia Ossificans Progressiva - Patient RepOrted syMPtoms Tool (FOP-PROMPT).
Number of Participants with Treatment Emergent Adverse Events (TEAE)	Up to 316 weeks	Defined as any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug.
Open-Label Extension: Total volume of new HO lesions from Week 24	Week 48	HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period.

Trial Locations

Locations (29)

University of California San Francisco Medical Center; 🇺🇸 San Francisco, California, United States

Mayo Clinic Rochester; 🇺🇸 Rochester, Minnesota, United States

Children'S Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Penn Medicine - Perelman Center For Advanced Medicine; 🇺🇸 Philadelphia, Pennsylvania, United States

Hospital Italiano de Buenos Aires; 🇦🇷 Ciudad Autonoma Buenos Aires, Argentina

Royal North Shore Hospital; 🇦🇺 St Leonards, New South Wales, Australia

Murdoch Children'S Research Institute; 🇦🇺 Parkville, Victoria, Australia

Albert Einstein Israelite Hospital; 🇧🇷 San Paolo, Brazil

University Health Network Toronto General Hospital; 🇨🇦 Toronto, Ontario, Canada

Centro de Estudios Reumatologicos; 🇨🇱 Santiago, Chile

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