To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
Progress
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
3 other identifiers
interventional
98
17 countries
27
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started May 2022
Longer than P75 for phase_2
27 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 8, 2021
CompletedFirst Posted
Study publicly available on registry
October 25, 2021
CompletedStudy Start
First participant enrolled
May 5, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 30, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
January 20, 2033
December 5, 2025
December 1, 2025
5.2 years
October 8, 2021
December 4, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline
HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period.
Week 24
Secondary Outcomes (14)
Double Blind Period: Number of new HO lesions from baseline
Week 24
Double Blind Period: Total volume of new HO lesions from baseline
Week 24
Double Blind Period: Change in the total volume of all HO lesions from baseline
Week 24
Double Blind Period: Number of new flares from baseline
Week 24
Number of Participants with Treatment Emergent Adverse Events (TEAE)
Up to 316 weeks
- +9 more secondary outcomes
Study Arms (3)
Cohort 1
EXPERIMENTALParticipants (≥ 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
EXPERIMENTALParticipants (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
EXPERIMENTALParticipants (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.
Interventions
INCBG000928 will be administered QD orally.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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:
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (27)
University of California San Francisco Medical Center
San Francisco, California, 94143, United States
Mayo Clinic Rochester
Rochester, Minnesota, 55905, United States
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Penn Medicine - Perelman Center For Advanced Medicine
Philadelphia, Pennsylvania, 19104, United States
Hospital Italiano de Buenos Aires
Ciudad Autonoma Buenos Aires, C1199ABB, Argentina
Royal North Shore Hospital
St Leonards, New South Wales, 02065, Australia
Murdoch Children'S Research Institute
Parkville, Victoria, 03052, Australia
Albert Einstein Israelite Hospital
São Paulo, 05652-900, Brazil
University Health Network Toronto General Hospital
Toronto, Ontario, M5G 2C4, Canada
Centro de Estudios Reumatologicos
Santiago, 7501126, Chile
Beijing Childrens Hospital Capital Medical University
Beijing, 100045, China
Tongji Hospital of Tongji University
Shanghai, 200065, China
Shanghai Childrens Medical Center
Shanghai, 200127, China
Childrens Hospital of Fudan University
Shanghai, 201102, China
Ap-Hp Hopital Lariboisiere
Paris, 75010, France
Hopital Necker-Enfants Malades
Paris, 75015, France
Uniklinik Koln
Cologne, 50931, Germany
Ospedale Pediatrico G. Gaslini
Genova, 16147, Italy
Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore
Rome, 00168, Italy
Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra
Tlalpan, 14389, Mexico
Amsterdam Umc - Vu Medisch Centrum (Vumc)
Amsterdam, 1081 HV, Netherlands
Starship Childrens Hospital
Auckland, 01023, New Zealand
Groote Schuur Hospital Radiation Oncology
Cape Town, 07925, South Africa
Seoul National University Hospital
Seoul, 03080, South Korea
Hospital Universitario Ramon Y Cajal
Madrid, 28034, Spain
Royal Manchester Childrens Hospital - Department of Paediatric Endocrinology
Manchester, M13 9PL, United Kingdom
Royal National Orthopaedic Hospital
Stanmore, HA7 4LP, United Kingdom
Related Publications (1)
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. 2025 Dec 8. doi: 10.1002/bcp.70372. Online ahead of print.
PMID: 41360500DERIVED
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Amanda McBride, MD
Incyte Corporation
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 8, 2021
First Posted
October 25, 2021
Study Start
May 5, 2022
Primary Completion (Estimated)
July 30, 2027
Study Completion (Estimated)
January 20, 2033
Last Updated
December 5, 2025
Record last verified: 2025-12
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP
- Time Frame
- Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
- Access Criteria
- Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.
Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.