INCB000928 for Fibrodysplasia Ossificans Progressiva

Inclusion criteria

• {"criterion_text":"-Informed consent/assent: a. For adult participants (≥ 18 years of age), ability to comprehend and willingness to sign an ICF. b. For children (2 to < 12 years of age) and adolescent participants (12 to < 18 years of age), written informed consent of the parent(s) or legal guardian and written assent from the underage participant.\n- Female and male participants: a.\tCohort 1: ≥ 12 years of age. b.\tCohort 2: 6 to < 12 years of age. Cohort 3: 2 to < 6 years of age\n- Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft-tissue swelling, and/or progressive HO).\n- Participant-reported FOP disease activity within 1 year of the screening visit.\n- Ability to swallow and retain orally administered tablets, either whole or crushed and dispersed in foods or liquids, or ability to receive and retain crushed tablets via a feeding tube\n- Willingness to avoid pregnancy or fathering children\n- Willing and able to undergo low-dose WBCT (excluding the head) imaging without requiring intubation.\n- Willing and able to comply with study procedures and requirements and attend all study visits as defined in this Protocol."}

Exclusion criteria

• {"criterion_text":"-Pregnant or breast-feeding\n-Participation in an investigational drug study for the treatment of FOP or any other indication within 30 days or 5 half-lives (whichever is longer) before baseline (Day 1 visit).\n-Planning to receive a live vaccine during the course of the study or within 6 weeks after the last dose of study drug.\n-Known or suspected allergy to INCB000928 or any component of the study drug.\n-Known history of clinically significant drug or alcohol abuse as defined by the investigator in the l year before baseline (Day 1 visit).\n-Chronic or current active infectious disease requiring systemic antibiotic, antifungal, or antiviral treatment.\n-HIV, HBV, or HCV infection\n-Participants with laboratory values at screening defined\n-Weight < 30 kg at screening (Cohort 1 only).\n-The following participants are excluded in France: a. Vulnerable populations according to article L.1121-6 of the French Public Health Code. b. Adults under legal protection or who are unable to express their consent per article L.1121-8 of the French Public Health Code. c. Individuals not affiliated with the social security system.\n-CAJIS score ≥ 24.\n-FOP disease severity that in the investigator's opinion precludes participation\n-History of uncontrolled or unstable cardiovascular, respiratory, renal, gastrointestinal, endocrine, hematopoietic, psychiatric, and/or neurological disease within 6 months of screening.\n-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.\n-Presence of a clinically significant finding on echocardiogram\n-Presence of an abnormal finding on ECG at screening that in the investigator's opinion is clinically significant and/or the following ECG parameters: QTcF interval > 450 milliseconds, ECG evidence of Brugada syndrome, atrial fibrillation or atrial flutter, or Mobitz II or higher grade atrioventricular block.\n-Current treatment with a potent/strong inhibitor or inducer of CYP3A4 within 5 half-lives before the first dose of study treatment or expected to receive such treatment during the study\n-Use of the following medications: a. Imatinib 30 days prior to baseline (Day 1 visit). b. Any medication that might interfere with HO formation in the 30 days or 5 half-lives, whichever is shorter before baseline"}

Primary endpoints

• {"endpoint_text":"-Occurrence of new HO lesions as assessed by low-dose WBCT (excluding the head) from baseline to Week 24.","definition_or_measurement_approach":"Assessed by low-dose whole-body CT (WBCT) excluding the head; occurrence of new heterotopic ossification (HO) lesions measured from baseline to Week 24."}

Secondary endpoints

• {"endpoint_text":"-•\tNumber of new HO lesions as assessed by low-dose WBCT (excluding the head) from baseline to Week 24.","definition_or_measurement_approach":"Count of new HO lesions measured by low-dose WBCT (excluding head) from baseline to Week 24."}
• {"endpoint_text":"-•\tTotal volume of new HO lesions as assessed by low-dose WBCT (excluding the head) from baseline to Week 24.","definition_or_measurement_approach":"Total volume of new HO lesions measured by low-dose WBCT (excluding head) from baseline to Week 24."}
• {"endpoint_text":"-•\tChange in the total volume of all HO lesions as assessed by low dose WBCT (excluding the head) from baseline to Week 24.","definition_or_measurement_approach":"Change in total volume of all HO lesions measured by low-dose WBCT (excluding head) from baseline to Week 24."}
• {"endpoint_text":"-•\tNumber of new flares based on FOP PROMPT from baseline to Week 24.","definition_or_measurement_approach":"Number of new disease flares as reported by the FOP-PROMPT instrument from baseline to Week 24."}
• {"endpoint_text":"-•\tAEs and SAEs, assessed by changes in vital signs, ECGs, echocardiograms, physical examinations, PFTs, BMD, laboratory data, and knee epiphyseal closure (2 to < 21 years of age).","definition_or_measurement_approach":"Safety assessed by incidence and severity of adverse events (AEs) and serious AEs (SAEs) and by changes in vital signs, ECGs, echocardiograms, physical exams, pulmonary function tests (PFTs), bone mineral density (BMD), laboratory data, and knee epiphyseal closure for ages 2 to <21 years."}
• {"endpoint_text":"-•\tOccurrence of new HO lesions as assessed by low dose 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.","definition_or_measurement_approach":"Occurrence of new HO lesions measured by low-dose WBCT (excluding head) from Week 24 to Week 48 compared with baseline to Week 24 in participants who crossed over from placebo to active at Week 24."}
• {"endpoint_text":"-•\tNumber of new HO lesions as assessed by low-dose 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.","definition_or_measurement_approach":"Number of new HO lesions by low-dose WBCT (excluding head) from Week 24 to Week 48 compared with baseline to Week 24 for placebo-randomized participants."}
• {"endpoint_text":"-•\tTotal volume of new HO lesions as assessed by low-dose 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.","definition_or_measurement_approach":"Total volume of new HO lesions measured by low-dose WBCT (excluding head) from Week 24 to Week 48 compared to baseline to Week 24 for placebo-randomized participants."}
• {"endpoint_text":"-•\tChange in the total volume of all HO lesions as assessed by low dose 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.","definition_or_measurement_approach":"Change in total volume of all HO lesions by low-dose WBCT (excluding head) from Week 24 to Week 48 vs baseline to Week 24 in placebo-randomized participants."}
• {"endpoint_text":"-INCB000928 PK parameters in plasma and/or saliva: Cmax, tmax, Cmin, and AUCt.","definition_or_measurement_approach":"Pharmacokinetic parameters of INCB000928 measured in plasma and/or saliva: Cmax, tmax, Cmin and AUCt."}
• {"endpoint_text":"-•\tNumber of new flares based on FOP PROMPT from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period","definition_or_measurement_approach":"Number of new FOP-PROMPT–reported flares from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the double-blind period."}

France

Latest Decision Or Authorization Date

30-06-2025

Number Of Sites

2

Number Of Participants

3

Germany

Latest Decision Or Authorization Date

02-07-2025

Number Of Sites

1

Number Of Participants

5

Italy

Latest Decision Or Authorization Date

30-06-2025

Number Of Sites

1

Number Of Participants

3

Portugal

Latest Decision Or Authorization Date

30-06-2025

Number Of Sites

1

Number Of Participants

1

Spain

Latest Decision Or Authorization Date

30-06-2025

Number Of Sites

1

Number Of Participants

5

Netherlands

Latest Decision Or Authorization Date

11-07-2025

Number Of Sites

1

Number Of Participants

1

Primary sponsor

Full Name

Incyte Corp.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States