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NCT Number: NCT05090891

To Assess 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).

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Key information

Conditions

Fibrodysplasia Ossificans Progressiva (FOP) Muscular Diseases Musculoskeletal Diseases Myositis Myositis Ossificans Ossification, Heterotopic Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

2 year–99 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hospital Italiano de Buenos Aires, Buenos Aires, Argentina

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for 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.

Treatment and study plan

INCB000928

Drug

INCBG000928 will be administered QD orally.

Other names: zilurgisertib

Placebo

Drug

Placebo will be administered QD orally.

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. Double Blind Period: Number of new flares from baseline

    Time frame: Week 24

    Based on Fibrodysplasia Ossificans Progressiva - Patient RepOrted syMPtoms Tool (FOP-PROMPT).

  5. 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.

  6. 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.

  7. 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.

  8. 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.

  9. 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.

  10. 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.

  11. Pharmacokinetics Parameter: Cmax of INCB000928

    Time frame: Baseline, Weeks 12, 24, 48 and 76

    Maximum observed concentration.

  12. Pharmacokinetics Parameter: Tmax of INCB000928

    Time frame: Baseline, Weeks 12, 24, 48 and 76

    Time to maximum concentration.

  13. Pharmacokinetics Parameter: Cmin of INCB000928

    Time frame: Baseline, Weeks 12, 24, 48 and 76

    Minimum observed concentration.

  14. 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

Study contacts

Contact information is provided by the study sponsor or research team.

Incyte Corporation Call Center (US)

CONTACT

[email protected]

1.855.463.3463

Incyte Corporation Call Center (ex-US)

CONTACT

[email protected]

+800 00027423

Sponsors and collaborators

Lead sponsor

Incyte Corporation

Industry

Registry information

Official study 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

Acronym: Progress

Important dates

Study start
2022
Primary completion
2027
Study completion
2033
First posted
Oct 25, 2021
Registry last updated
May 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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