To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
- Conditions
- Fibrodysplasia Ossificans Progressiva (FOP)
- Interventions
- 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
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 98
-
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.
- 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: 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: 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.
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: 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: 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.
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.
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.
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
Trial Locations
- Locations (29)
Beijing Childrens Hospital Capital Medical University
🇨🇳Beijing, China
Tongji Hospital of Tongji University
🇨🇳Shanghai, China
Shanghai Childrens Medical Center
🇨🇳Shanghai, China
Childrens Hospital of Fudan University
🇨🇳Shanghai, China
Ap-Hp Hopital Lariboisiere
🇫🇷Paris, France
Hopital Necker-Enfants Malades
🇫🇷Paris, France
Hospital Universitario Ramon Y Cajal
🇪🇸Madrid, Spain
Ege University Faculty of Medicine
🇹🇷Izmir, Turkey
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
Uniklinik Koln
🇩🇪Koln, Germany
Ospedale Pediatrico G. Gaslini
🇮🇹Genova, Italy
Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore
🇮🇹Rome, Italy
Seoul National University Hospital
🇰🇷Seoul, Korea, Republic of
Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra
🇲🇽Tlalpan, Mexico
Amsterdam Umc - Vu Medisch Centrum (Vumc)
🇳🇱Amsterdam, Netherlands
Starship Childrens Hospital
🇳🇿Auckland, New Zealand
Centro Hospitalar Universitario de Lisboa Norte, Hospital Santa Maria
🇵🇹Lisbon, Portugal
Groote Schuur Hospital Radiation Oncology
🇿🇦Cape Town, South Africa
Royal Manchester Childrens Hospital - Department of Paediatric Endocrinology
🇬🇧Manchester, United Kingdom
Royal National Orthopaedic Hospital
🇬🇧Stanmore, United Kingdom