Fibrodysplasia Ossificans Progressiva (FOP)Phase 2ProgressNCT05090891
To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
A Phase 2 study testing INCB000928 in people with Fibrodysplasia Ossificans Progressiva (FOP), run by Incyte Corporation at 24 sites in 17 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 2 to 99 years
- Conditions the study is for
- Fibrodysplasia Ossificans Progressiva (FOP)
- What is being tested?
- INCB000928
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 98 participants
- Where
- 24 sitesin 17 countries
- Study dates
- 5 May 2022to 20 Jan 2033
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
24 sites in 17 countries. Contact the study team or a site near you to ask about taking part.
24 sites in 17 countries
China4 sites, 4 recruiting
- Recruiting
Beijing Childrens Hospital Capital Medical University
Beijing, 100045
- Recruiting
Tongji Hospital of Tongji University
Shanghai, 200065
- Recruiting
Shanghai Childrens Medical Center
Shanghai, 200127
- Recruiting
Childrens Hospital of Fudan University
Shanghai, 201102
United States3 sites, 2 recruiting
- Recruiting
Children'S Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Active, not recruiting
Penn Medicine - Perelman Center For Advanced Medicine
Philadelphia, Pennsylvania, 19104
- Recruiting
Mayo Clinic Rochester
Rochester, Minnesota, 55905
Australia2 sites
- Active, not recruiting
Murdoch Children'S Research Institute
Parkville, Victoria, 03052
- Completed
Royal North Shore Hospital
St Leonards, New South Wales, 02065
France2 sites, 2 recruiting
- Recruiting
Ap-Hp Hopital Lariboisiere
Paris, 75010
- Recruiting
Hopital Necker-Enfants Malades
Paris, 75015
Argentina1 site
- Active, not recruiting
Hospital Italiano de Buenos Aires
Buenos Aires, CO 1181
Brazil1 site
- Active, not recruiting
Albert Einstein Israelite Hospital
São Paulo, 05652-900
Canada1 site
- Active, not recruiting
University Health Network Toronto General Hospital
Toronto, Ontario, M5G 2N2
Chile1 site
- Active, not recruiting
Centro de Estudios Reumatologicos
Santiago, 7501126
Italy1 site
- Active, not recruiting
Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore
Rome, 00168
Mexico1 site
- Active, not recruiting
Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra
Tlalpan, 14389
Netherlands1 site, 1 recruiting
- Recruiting
Amsterdam Umc - Vu Medisch Centrum (Vumc)
Amsterdam, 1081 HV
New Zealand1 site
- Active, not recruiting
Starship Childrens Hospital
Auckland, 01023
South Africa1 site
- Active, not recruiting
Groote Schuur Hospital Radiation Oncology
Cape Town, 07925
South Korea1 site
- Active, not recruiting
Seoul National University Hospital
Seoul, 03080
Spain1 site, 1 recruiting
- Recruiting
Hospital Universitario Ramon Y Cajal
Madrid, 28034
United Kingdom1 site
- Active, not recruiting
Royal National Orthopaedic Hospital
Stanmore, HA7 4LP
About this study
Official title: 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).
- Being tested
- INCB000928, Placebo
- Keywords
- fibrodysplasia ossificans progressiva (FOP), heterotopic ossification
- Medical subject headings
- Myositis Ossificans, Ossification, Heterotopic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
INCB000928Drug
Also called zilurgisertib
INCBG000928 will be administered QD orally.
PlaceboDrug
Placebo will be administered QD orally.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 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. Receives: Drug: INCB000928; Drug: Placebo |
| Cohort 2 | Experimental | 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. Receives: Drug: INCB000928; Drug: Placebo |
| Cohort 3 | Experimental | 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. Receives: Drug: INCB000928; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Week 24
Secondary outcomes (14)
Double Blind Period: Number of new 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.
Measured: Week 24
Double Blind Period: Total volume of new 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.
Measured: Week 24
Double Blind Period: Change in the total volume of all 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.
Measured: Week 24
Double Blind Period: Number of new flares from baseline
Based on Fibrodysplasia Ossificans Progressiva - Patient RepOrted syMPtoms Tool (FOP-PROMPT).
Measured: Week 24
Number of Participants with Treatment Emergent Adverse Events (TEAE)
Defined as any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug.
Measured: Up to 316 weeks
Open-Label Extension: Occurrence of new HO lesions from Week 24
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.
Measured: Week 48
Open-Label Extension: Number of new HO lesions from Week 24
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.
Measured: Week 48
Open-Label Extension: Total volume of new HO lesions from Week 24
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.
Measured: Week 48
Open-Label Extension: Change in the total volume of all HO lesions from Week 24
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.
Measured: Week 48
Open-Label Extension: Number of new flares from Week 24
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.
Measured: Week 48
Pharmacokinetics Parameter: Cmax of INCB000928
Maximum observed concentration.
Measured: Baseline, Weeks 12, 24, 48 and 76
Pharmacokinetics Parameter: Tmax of INCB000928
Time to maximum concentration.
Measured: Baseline, Weeks 12, 24, 48 and 76
Pharmacokinetics Parameter: Cmin of INCB000928
Minimum observed concentration.
Measured: Baseline, Weeks 12, 24, 48 and 76
Pharmacokinetics Parameter: AUCt of INCB000928
Area under the plasma concentration-time curve from time 0 to the last quantifiable measurable plasma concentration
Measured: Baseline, Weeks 12, 24, 48 and 76
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2021
- Study start5 May 2022
- Primary completion30 Jul 2027 (estimated)
- Study completion20 Jan 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fibrodysplasia Ossificans Progressiva (FOP)Studies of the same condition, open and under way.
- More trials run by Incyte CorporationThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 98 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Incyte Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Amanda McBride, MD, Study director, Incyte Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05090891
- Sponsor's protocol ID
- INCB 00928-201
- Other IDs
- 2023-504129-38-00, 2021-002286-17, 2023
- Acronym
- Progress
- Status last verified
- Oct 2026
- First submitted
- 8 Oct 2021
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Publications
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. 2026 May;92(5):1339-1351. doi: 10.1002/bcp.70372. Epub 2025 Dec 8.
PubMed 41360500