A Phase 2, Randomized Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
- Conditions
- Fibrodysplasia ossificans progressivaMedDRA version: 20.0Level: PTClassification code 10068715Term: Fibrodysplasia ossificans progressivaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2021-002286-17-FR
- Lead Sponsor
- Incyte Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 60
1. Informed consent/assent:
a. For adult participants (= 18 years of age), ability to comprehend and willingness to sign an ICF.
b. For adolescent participants (= 12 to < 18 years of age), written informed consent of the parent(s) or legal guardian and written assent from the adolescent participant.
2. Female and male adults and adolescents = 12 years of age.
3. Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft-tissue swelling, and/or progressive HO).
4. Participant-reported FOP disease activity within 1 year of the screening visit. This is defined as pain, swelling, and other signs and symptoms associated with FOP flare-ups or worsening of joint function or radiographic progression of HO (increase in site or number of HO lesions) with or without an association with flare-up episodes.
5. Ability to swallow and retain orally administered tablets, either whole or crushed and dispersed in foods or liquids
6. Willingness to avoid pregnancy or fathering children based on the criteria below.
a. Male participants with reproductive potential must agree to take appropriate precautions to avoid fathering children from screening through 90 days after the last dose of study drug and must refrain from donating sperm during this period.
b. Female participants of childbearing potential must have a negative pregnancy test at screening (serum) and before the first dose on Day 1 (urine). Female participants of childbearing potential must agree to take appropriate precautions to avoid pregnancy from screening through 190 days after the last dose of study drug
c. Women without childbearing potential are eligible.
7. Willing and able to undergo low-dose WBCT (excluding the head) imaging without requiring intubation.
8. Willing and able to comply with study procedures and requirements and attend all study visits as defined in this Protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 38
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2
1. Pregnant or breast-feeding.
2. CAJIS score = 24.
3. FOP disease severity that in the investigator's opinion precludes participation
4. History of uncontrolled or unstable cardiovascular, respiratory, renal, gastrointestinal, endocrine, hematopoietic, psychiatric, and/or neurological disease within 6 months of screening.
5. 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.
6. Presence of a clinically significant finding on echocardiogram
7. 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, QRS interval > 120 milliseconds, PR interval > 220 milliseconds, ECG evidence of Brugada syndrome, atrial fibrillation or atrial flutter, or Mobitz II or higher grade atrioventricular block.
8. 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
9. Use of the following medications:
a. Imatinib 30 days prior to baseline
b. Any medication that might interfere with HO formation in the 90 days before baseline
c. Bisphosphonates within 1 year of screening.
10. 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
11. Planning to receive a live vaccine during the course of the study or within 6 weeks after the last dose of study drug.
12. Known or suspected allergy to INCB000928 or any component of the study drug.
13. Known history of clinically significant drug or alcohol abuse as defined by the investigator in the l year before baseline
14. Chronic or current active infectious disease requiring systemic antibiotic, antifungal, or antiviral treatment.
15. HIV, HBV, or HCV infection.
16. Participants with laboratory values at screening defined in protocol
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To determine the efficacy of INCB000928 for the<br>prevention of new HO in participants with FOP.;Secondary Objective: To further evaluate the efficacy of INCB000928 in the reduction of flares and improvement in flare-related symptoms.<br>To evaluate the safety and tolerability of INCB000928 in participants with FOP.<br>To confirm the efficacy of INCB000928 at Week 48 in participants randomized to placebo during the double-blind period.<br>To characterize the PK of INCB000928 in participants with FOP.<br>To further evaluate the efficacy of INCB000928 in participants with FOP.;Primary end point(s): • Total volume of new HO as assessed by low-dose WBCT (excluding the head) at Week 24.;Timepoint(s) of evaluation of this end point: Week 24 and Safety assessments will be performed throughout the<br>study
- Secondary Outcome Measures
Name Time Method