To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva

NCT ID: NCT05090891

Last Updated: 2025-12-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

98 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-05-05

Study Completion Date

2033-01-20

Brief Summary

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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

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Conditions

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Fibrodysplasia Ossificans Progressiva (FOP)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Cohort 1

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.

Group Type EXPERIMENTAL

INCB000928

Intervention Type DRUG

INCBG000928 will be administered QD orally.

Placebo

Intervention Type DRUG

Placebo will be administered QD orally.

Cohort 2

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.

Group Type EXPERIMENTAL

INCB000928

Intervention Type DRUG

INCBG000928 will be administered QD orally.

Placebo

Intervention Type DRUG

Placebo will be administered QD orally.

Cohort 3

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.

Group Type EXPERIMENTAL

INCB000928

Intervention Type DRUG

INCBG000928 will be administered QD orally.

Placebo

Intervention Type DRUG

Placebo will be administered QD orally.

Interventions

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INCB000928

INCBG000928 will be administered QD orally.

Intervention Type DRUG

Placebo

Placebo will be administered QD orally.

Intervention Type DRUG

Other Intervention Names

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zilurgisertib

Eligibility Criteria

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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.

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:
Minimum Eligible Age

2 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Incyte Corporation

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Amanda McBride, MD

Role: STUDY_DIRECTOR

Incyte Corporation

Locations

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University of California San Francisco Medical Center

San Francisco, California, United States

Site Status WITHDRAWN

Mayo Clinic Rochester

Rochester, Minnesota, United States

Site Status RECRUITING

Children'S Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Penn Medicine - Perelman Center For Advanced Medicine

Philadelphia, Pennsylvania, United States

Site Status ACTIVE_NOT_RECRUITING

Hospital Italiano de Buenos Aires

Ciudad Autonoma Buenos Aires, , Argentina

Site Status NOT_YET_RECRUITING

Royal North Shore Hospital

St Leonards, New South Wales, Australia

Site Status COMPLETED

Murdoch Children'S Research Institute

Parkville, Victoria, Australia

Site Status RECRUITING

Albert Einstein Israelite Hospital

São Paulo, , Brazil

Site Status RECRUITING

University Health Network Toronto General Hospital

Toronto, Ontario, Canada

Site Status ACTIVE_NOT_RECRUITING

Centro de Estudios Reumatologicos

Santiago, , Chile

Site Status RECRUITING

Beijing Childrens Hospital Capital Medical University

Beijing, , China

Site Status RECRUITING

Tongji Hospital of Tongji University

Shanghai, , China

Site Status RECRUITING

Shanghai Childrens Medical Center

Shanghai, , China

Site Status RECRUITING

Childrens Hospital of Fudan University

Shanghai, , China

Site Status RECRUITING

Ap-Hp Hopital Lariboisiere

Paris, , France

Site Status RECRUITING

Hopital Necker-Enfants Malades

Paris, , France

Site Status RECRUITING

Uniklinik Koln

Cologne, , Germany

Site Status RECRUITING

Ospedale Pediatrico G. Gaslini

Genova, , Italy

Site Status WITHDRAWN

Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore

Rome, , Italy

Site Status RECRUITING

Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra

Tlalpan, , Mexico

Site Status RECRUITING

Amsterdam Umc - Vu Medisch Centrum (Vumc)

Amsterdam, , Netherlands

Site Status RECRUITING

Starship Childrens Hospital

Auckland, , New Zealand

Site Status RECRUITING

Groote Schuur Hospital Radiation Oncology

Cape Town, , South Africa

Site Status RECRUITING

Seoul National University Hospital

Seoul, , South Korea

Site Status ACTIVE_NOT_RECRUITING

Hospital Universitario Ramon Y Cajal

Madrid, , Spain

Site Status RECRUITING

Royal Manchester Childrens Hospital - Department of Paediatric Endocrinology

Manchester, , United Kingdom

Site Status WITHDRAWN

Royal National Orthopaedic Hospital

Stanmore, , United Kingdom

Site Status RECRUITING

Countries

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United States Argentina Australia Brazil Canada Chile China France Germany Italy Mexico Netherlands New Zealand South Africa South Korea Spain United Kingdom

Central Contacts

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Incyte Corporation Call Center (US)

Role: CONTACT

1.855.463.3463

Incyte Corporation Call Center (ex-US)

Role: CONTACT

+800 00027423

Related Links

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Other Identifiers

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2023-504129-38-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

2021-002286-17

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

INCB 00928-201

Identifier Type: -

Identifier Source: org_study_id

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