To Assess The Efficacy, Safety, And Tolerability Of INCB000928 In Participants With Fibrodysplasia Ossificans Progressiva

Overview

About this study

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).

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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.

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 07/14/2026. Questions regarding updates should be directed to the study team contact.

 

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Robert Pignolo, M.D., Ph.D.

Open for enrollment

More information

Publications

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Additional contact information

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