Evaluating INCB000928 for treating Fibrodysplasia Ossificans Progressiva

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva

This study is testing a new drug called INCB000928 to see if it can help people with Fibrodysplasia Ossificans Progressiva manage their symptoms better.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 98 (estimated)
• Ages: 2 Years to 99 Years
• Sex: All
• Sponsor: Incyte Corporation (industry)
• Locations: 27 sites (San Francisco, California and 26 other locations)
• Trial ID: NCT05090891 on ClinicalTrials.gov

What this trial studies

This Phase 2 interventional study aims to assess the efficacy, safety, and tolerability of INCB000928 in participants diagnosed with Fibrodysplasia Ossificans Progressiva (FOP). The study is randomized, double-blind, and placebo-controlled, meaning that participants will be assigned to receive either the treatment or a placebo without knowing which they are receiving. The study will include participants from different age cohorts, ranging from 2 to 12 years and older, and will involve imaging assessments to monitor the effects of the treatment. The goal is to gather data on how well INCB000928 works in managing this rare and debilitating condition.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals aged 2 to 12 years with a clinical diagnosis of Fibrodysplasia Ossificans Progressiva.

Not a fit: Patients with severe FOP disease or other significant medical conditions that could interfere with participation may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of Fibrodysplasia Ossificans Progressiva, potentially reducing the severity of symptoms and enhancing quality of life for patients.

How similar studies have performed: While this approach is novel for FOP, similar studies targeting heterotopic ossification have shown promise in other contexts.

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 \< 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.

Where this trial is running

San Francisco, California and 26 other locations

• University of California San Francisco Medical Center — San Francisco, California, United States (WITHDRAWN)
• Mayo Clinic Rochester — Rochester, Minnesota, United States (RECRUITING)
• Children'S Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (RECRUITING)
• Penn Medicine - Perelman Center For Advanced Medicine — Philadelphia, Pennsylvania, United States (ACTIVE_NOT_RECRUITING)
• Hospital Italiano de Buenos Aires — Ciudad Autonoma Buenos Aires, Argentina (NOT_YET_RECRUITING)
• Royal North Shore Hospital — St Leonards, New South Wales, Australia (COMPLETED)
• Murdoch Children'S Research Institute — Parkville, Victoria, Australia (RECRUITING)
• Albert Einstein Israelite Hospital — São Paulo, Brazil (RECRUITING)
• University Health Network Toronto General Hospital — Toronto, Ontario, Canada (ACTIVE_NOT_RECRUITING)
• Centro de Estudios Reumatologicos — Santiago, Chile (RECRUITING)
• Beijing Childrens Hospital Capital Medical University — Beijing, China (RECRUITING)
• Tongji Hospital of Tongji University — Shanghai, China (RECRUITING)
• Shanghai Childrens Medical Center — Shanghai, China (RECRUITING)
• Childrens Hospital of Fudan University — Shanghai, China (RECRUITING)
• Ap-Hp Hopital Lariboisiere — Paris, France (RECRUITING)
• Hopital Necker-Enfants Malades — Paris, France (RECRUITING)
• Uniklinik Koln — Cologne, Germany (RECRUITING)
• Ospedale Pediatrico G. Gaslini — Genova, Italy (WITHDRAWN)
• Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore — Rome, Italy (RECRUITING)
• Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra — Tlalpan, Mexico (RECRUITING)
• Amsterdam Umc - Vu Medisch Centrum (Vumc) — Amsterdam, Netherlands (RECRUITING)
• Starship Childrens Hospital — Auckland, New Zealand (RECRUITING)
• Groote Schuur Hospital Radiation Oncology — Cape Town, South Africa (RECRUITING)
• Seoul National University Hospital — Seoul, South Korea (ACTIVE_NOT_RECRUITING)
• Hospital Universitario Ramon Y Cajal — Madrid, Spain (RECRUITING)
• Royal Manchester Childrens Hospital - Department of Paediatric Endocrinology — Manchester, United Kingdom (WITHDRAWN)
• Royal National Orthopaedic Hospital — Stanmore, United Kingdom (RECRUITING)

Study contacts

• Study coordinator: Incyte Corporation Call Center (US)

Conditions: Fibrodysplasia Ossificans Progressiva, fibrodysplasia ossificans progressiva, heterotopic ossification