New drug aims to stop extra bone formation in rare disease
NCT ID NCT05090891
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 2 trial tests an experimental drug called INCB000928 (zilurgisertib) in people with fibrodysplasia ossificans progressiva (FOP), a rare genetic condition where soft tissues turn into bone. About 98 participants aged 2 and older will receive either the drug or a placebo daily for several months. The main goal is to see if the drug reduces new abnormal bone growth, which could help control the disease and maintain mobility.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- INCB000928 (zilurgisertib)
- What this could lead to
- If successful, this drug could slow or prevent the formation of extra bone in people with FOP, potentially reducing disability and improving quality of life.
- What could go wrong
- This is an early Phase 2 trial with a small number of participants. The drug may not work better than placebo, and side effects are still being studied.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 98 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2022
- Expected to finish
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Jan 2033
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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2 to 99 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
11 sites in 6 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
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Study contacts
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Contact
Email: •••••@•••••
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Contact
Email: •••••@•••••
Locations
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Albert Einstein Israelite Hospital
ACTIVE_NOT_RECRUITINGSão Paulo, 05652-900, Brazil
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Amsterdam Umc - Vu Medisch Centrum (Vumc)
RECRUITINGAmsterdam, 1081 HV, Netherlands
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Ap-Hp Hopital Lariboisiere
RECRUITINGParis, 75010, France
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Beijing Childrens Hospital Capital Medical University
RECRUITINGBeijing, 100045, China
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Centro de Estudios Reumatologicos
ACTIVE_NOT_RECRUITINGSantiago, 7501126, Chile
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Children'S Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Childrens Hospital of Fudan University
RECRUITINGShanghai, 201102, China
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Groote Schuur Hospital Radiation Oncology
ACTIVE_NOT_RECRUITINGCape Town, 07925, South Africa
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Hopital Necker-Enfants Malades
RECRUITINGParis, 75015, France
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Hospital Italiano de Buenos Aires
ACTIVE_NOT_RECRUITINGBuenos Aires, CO 1181, Argentina
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Hospital Universitario Ramon Y Cajal
RECRUITINGMadrid, 28034, Spain
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Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra
ACTIVE_NOT_RECRUITINGTlalpan, 14389, Mexico
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Mayo Clinic Rochester
RECRUITINGRochester, Minnesota, 55905, United States
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Murdoch Children'S Research Institute
ACTIVE_NOT_RECRUITINGParkville, Victoria, 03052, Australia
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Penn Medicine - Perelman Center For Advanced Medicine
ACTIVE_NOT_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore
ACTIVE_NOT_RECRUITINGRome, 00168, Italy
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Royal National Orthopaedic Hospital
ACTIVE_NOT_RECRUITINGStanmore, HA7 4LP, United Kingdom
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Royal North Shore Hospital
COMPLETEDSt Leonards, New South Wales, 02065, Australia
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Seoul National University Hospital
ACTIVE_NOT_RECRUITINGSeoul, 03080, South Korea
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Shanghai Childrens Medical Center
RECRUITINGShanghai, 200127, China
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Starship Childrens Hospital
ACTIVE_NOT_RECRUITINGAuckland, 01023, New Zealand
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Tongji Hospital of Tongji University
RECRUITINGShanghai, 200065, China
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Uniklinik Koln
RECRUITINGCologne, 50931, Germany
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University Health Network Toronto General Hospital
ACTIVE_NOT_RECRUITINGToronto, Ontario, M5G 2N2, Canada
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