Recruiting NCT05090891
To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: INCB000928, Placebo.
- Who it may be relevant to
- Registry conditions: Fibrodysplasia Ossificans Progressiva (FOP). Basic parameters: 2 years — 99 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Argentina, Australia, Brazil, Canada +12
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
Overview
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).
Interventions
- Drug INCB000928
INCBG000928 will be administered QD orally. - Drug Placebo
Placebo will be administered QD orally.
Primary outcome measures
- Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline [Time frame: Week 24]
Secondary outcome measures (12)
- Double Blind Period: Number of new HO lesions from baseline [Time frame: Week 24]
- Double Blind Period: Total volume of new HO lesions from baseline [Time frame: Week 24]
- Double Blind Period: Change in the total volume of all HO lesions from baseline [Time frame: Week 24]
- Double Blind Period: Number of new flares from baseline [Time frame: Week 24]
- Number of Participants with Treatment Emergent Adverse Events (TEAE) [Time frame: Up to 316 weeks]
- Open-Label Extension: Occurrence of new HO lesions from Week 24 [Time frame: Week 48]
- Open-Label Extension: Number of new HO lesions from Week 24 [Time frame: Week 48]
- Open-Label Extension: Total volume of new HO lesions from Week 24 [Time frame: Week 48]
- Open-Label Extension: Change in the total volume of all HO lesions from Week 24 [Time frame: Week 48]
- Open-Label Extension: Number of new flares from Week 24 [Time frame: Week 48]
- Pharmacokinetics Parameter: Cmax of INCB000928 [Time frame: Baseline, Weeks 12, 24, 48 and 76]
- Pharmacokinetics Parameter: Tmax of INCB000928 [Time frame: Baseline, Weeks 12, 24, 48 and 76]
Eligibility criteria
Inclusion criteria
- Female and male participants:
- Cohort 1: ≥ 12 years of age.
- Cohort 2: 6 to < 12 years of age.
- Cohort 3: 2 to < 12 years of age (after eDMC review of safety 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Double blind
- Primary purpose
- Treatment
Study locations
China · 4 centers
- Beijing Childrens Hospital Capital Medical University — Beijing
- Tongji Hospital of Tongji University — Shanghai
- Shanghai Childrens Medical Center — Shanghai
- Childrens Hospital of Fudan University — Shanghai
United States · 3 centers
- Mayo Clinic Rochester — Rochester
- Children'S Hospital of Philadelphia — Philadelphia
- Penn Medicine - Perelman Center For Advanced Medicine — Philadelphia
Australia · 2 centers
- Royal North Shore Hospital — St Leonards
- Murdoch Children'S Research Institute — Parkville
France · 2 centers
- Ap-Hp Hopital Lariboisiere — Paris
- Hopital Necker-Enfants Malades — Paris
Argentina · 1 center
- Hospital Italiano de Buenos Aires — Buenos Aires
Brazil · 1 center
- Albert Einstein Israelite Hospital — São Paulo
Canada · 1 center
- University Health Network Toronto General Hospital — Toronto
Chile · 1 center
- Centro de Estudios Reumatologicos — Santiago
Germany · 1 center
- Uniklinik Koln — Cologne
Italy · 1 center
- Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore — Rome
Mexico · 1 center
- Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra — Tlalpan
Netherlands · 1 center
- Amsterdam Umc - Vu Medisch Centrum (Vumc) — Amsterdam
New Zealand · 1 center
- Starship Childrens Hospital — Auckland
South Africa · 1 center
- Groote Schuur Hospital Radiation Oncology — Cape Town
South Korea · 1 center
- Seoul National University Hospital — Seoul
Spain · 1 center
- Hospital Universitario Ramon Y Cajal — Madrid
United Kingdom · 1 center
- Royal National Orthopaedic Hospital — Stanmore
Publications
- Yang YO, Fang Y, Wang P, Liu X, Getsy J, Rockich K. Effects of Renal and Hepatic Impairment on the Pharmacokinetics of Zilurgisertib. Br J Clin Pharmacol. 2026 May;92(5):1339-1351. doi: 10.1002/bcp.70372. Epub 2025 Dec 8. PMID 41360500
Identifiers
NCT: NCT05090891 · INCB 00928-201 · 2023-504129-38-00 · 2021-002286-17