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

May 19, 2026 updated by: Incyte Corporation

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 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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
98

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Incyte Corporation Call Center (US)
  • Phone Number: 1.855.463.3463
  • Email: medinfo@incyte.com

Study Contact Backup

  • Name: Incyte Corporation Call Center (ex-US)
  • Phone Number: +800 00027423
  • Email: eumedinfo@incyte.com

Study Locations

  • Argentina
      • Buenos Aires, Argentina, CO 1181
        • Active, not recruiting
        • Hospital Italiano de Buenos Aires
  • Australia
    • New South Wales
      • St Leonards, New South Wales, Australia, 02065
        • Completed
        • Royal North Shore Hospital
    • Victoria
      • Parkville, Victoria, Australia, 03052
        • Active, not recruiting
        • Murdoch Children's Research Institute
  • Brazil
      • São Paulo, Brazil, 05652-900
        • Active, not recruiting
        • Albert Einstein Israelite Hospital
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 2N2
        • Active, not recruiting
        • University Health Network Toronto General Hospital
  • Chile
      • Santiago, Chile, 7501126
        • Active, not recruiting
        • Centro de Estudios Reumatologicos
  • China
      • Beijing, China, 100045
        • Recruiting
        • Beijing Childrens Hospital Capital Medical University
      • Shanghai, China, 200065
        • Recruiting
        • Tongji Hospital of Tongji University
      • Shanghai, China, 200127
        • Recruiting
        • Shanghai Childrens Medical Center
      • Shanghai, China, 201102
        • Recruiting
        • Childrens Hospital of Fudan University
  • France
      • Paris, France, 75015
        • Recruiting
        • Hôpital Necker-Enfants Malades
      • Paris, France, 75010
        • Recruiting
        • Ap-Hp Hopital Lariboisiere
  • Germany
      • Cologne, Germany, 50931
        • Recruiting
        • Uniklinik Köln
  • Italy
      • Rome, Italy, 00168
        • Active, not recruiting
        • Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore
  • Mexico
      • Tlalpan, Mexico, 14389
        • Active, not recruiting
        • Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra
  • Netherlands
      • Amsterdam, Netherlands, 1081 HV
        • Recruiting
        • Amsterdam Umc - Vu Medisch Centrum (Vumc)
  • New Zealand
      • Auckland, New Zealand, 01023
        • Active, not recruiting
        • Starship Childrens Hospital
  • South Africa
      • Cape Town, South Africa, 07925
        • Active, not recruiting
        • Groote Schuur Hospital Radiation Oncology
  • South Korea
      • Seoul, South Korea, 03080
        • Active, not recruiting
        • Seoul National University Hospital
  • Spain
      • Madrid, Spain, 28034
        • Recruiting
        • Hospital Universitario Ramón y Cajal
  • United Kingdom
      • Stanmore, United Kingdom, HA7 4LP
        • Active, not recruiting
        • Royal National Orthopaedic Hospital
  • United States
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic Rochester
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
      • Philadelphia, Pennsylvania, United States, 19104
        • Active, not recruiting
        • Penn Medicine - Perelman Center for Advanced Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

12 years to 99 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: 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.
Drug: INCB000928
INCBG000928 will be administered QD orally.
Other Names:
  • zilurgisertib
Drug: Placebo
Placebo will be administered QD orally.
Experimental: 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.
Drug: INCB000928
INCBG000928 will be administered QD orally.
Other Names:
  • zilurgisertib
Drug: Placebo
Placebo will be administered QD orally.
Experimental: Cohort 3
Participants (2 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.
Drug: INCB000928
INCBG000928 will be administered QD orally.
Other Names:
  • zilurgisertib
Drug: Placebo
Placebo will be administered QD orally.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline
Time Frame: 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.
Week 24

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Double Blind Period: Number of new HO lesions from baseline
Time Frame: 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.
Week 24
Double Blind Period: Total volume of new HO lesions from baseline
Time Frame: 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.
Week 24
Double Blind Period: Change in the total volume of all HO lesions from baseline
Time Frame: 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.
Week 24
Double Blind Period: Number of new flares from baseline
Time Frame: Week 24
Based on Fibrodysplasia Ossificans Progressiva - Patient RepOrted syMPtoms Tool (FOP-PROMPT).
Week 24
Number of Participants with Treatment Emergent Adverse Events (TEAE)
Time Frame: 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.
Up to 316 weeks
Open-Label Extension: Occurrence of new HO lesions from Week 24
Time Frame: 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.
Week 48
Open-Label Extension: Number of new HO lesions from Week 24
Time Frame: 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.
Week 48
Open-Label Extension: Total volume of new HO lesions from Week 24
Time Frame: 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.
Week 48
Open-Label Extension: Change in the total volume of all HO lesions from Week 24
Time Frame: 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.
Week 48
Open-Label Extension: Number of new flares from Week 24
Time Frame: 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.
Week 48
Pharmacokinetics Parameter: Cmax of INCB000928
Time Frame: Baseline, Weeks 12, 24, 48 and 76
Maximum observed concentration.
Baseline, Weeks 12, 24, 48 and 76
Pharmacokinetics Parameter: Tmax of INCB000928
Time Frame: Baseline, Weeks 12, 24, 48 and 76
Time to maximum concentration.
Baseline, Weeks 12, 24, 48 and 76
Pharmacokinetics Parameter: Cmin of INCB000928
Time Frame: Baseline, Weeks 12, 24, 48 and 76
Minimum observed concentration.
Baseline, Weeks 12, 24, 48 and 76
Pharmacokinetics Parameter: AUCt of INCB000928
Time Frame: Baseline, Weeks 12, 24, 48 and 76
Area under the plasma concentration-time curve from time 0 to the last quantifiable measurable plasma concentration
Baseline, Weeks 12, 24, 48 and 76

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Incyte Corporation

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Amanda McBride, MD, Incyte Corporation

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 5, 2022

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 30, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 20, 2033

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 8, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 14, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 25, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 22, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 19, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • INCB 00928-201
  • 2021-002286-17 (EudraCT Number)
  • 2023-504129-38-00 (Registry Identifier: EU CT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

IPD Sharing Time Frame

Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.

IPD Sharing 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.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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