RecruitingPhase 2NCT05090891

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

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva


Sponsor

Incyte Corporation

Enrollment

98 participants

Start Date

May 5, 2022

Study Type

INTERVENTIONAL

Conditions

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).


Eligibility

Min Age: 2 YearsMax Age: 99 Years

Inclusion Criteria7

  • Female and male participants:
  • Cohort 1: ≥ 12 years of age.
  • Cohort 2: 6 to < 12 years of age.
  • Cohort 3: 2 to < 6 years of age (after eDMC review of interim 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.

Exclusion Criteria6

  • 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:

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Interventions

DRUGINCB000928

INCBG000928 will be administered QD orally.

DRUGPlacebo

Placebo will be administered QD orally.


Locations(27)

University of California San Francisco Medical Center

San Francisco, California, United States

Mayo Clinic Rochester

Rochester, Minnesota, United States

Children'S Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Penn Medicine - Perelman Center For Advanced Medicine

Philadelphia, Pennsylvania, United States

Hospital Italiano de Buenos Aires

Ciudad Autonoma Buenos Aires, Argentina

Royal North Shore Hospital

St Leonards, New South Wales, Australia

Murdoch Children'S Research Institute

Parkville, Victoria, Australia

Albert Einstein Israelite Hospital

São Paulo, Brazil

University Health Network Toronto General Hospital

Toronto, Ontario, Canada

Centro de Estudios Reumatologicos

Santiago, Chile

Beijing Childrens Hospital Capital Medical University

Beijing, China

Tongji Hospital of Tongji University

Shanghai, China

Shanghai Childrens Medical Center

Shanghai, China

Childrens Hospital of Fudan University

Shanghai, China

Ap-Hp Hopital Lariboisiere

Paris, France

Hopital Necker-Enfants Malades

Paris, France

Uniklinik Koln

Cologne, Germany

Ospedale Pediatrico G. Gaslini

Genova, Italy

Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore

Rome, Italy

Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra

Tlalpan, Mexico

Amsterdam Umc - Vu Medisch Centrum (Vumc)

Amsterdam, Netherlands

Starship Childrens Hospital

Auckland, New Zealand

Groote Schuur Hospital Radiation Oncology

Cape Town, South Africa

Seoul National University Hospital

Seoul, South Korea

Hospital Universitario Ramon Y Cajal

Madrid, Spain

Royal Manchester Childrens Hospital - Department of Paediatric Endocrinology

Manchester, United Kingdom

Royal National Orthopaedic Hospital

Stanmore, United Kingdom

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NCT05090891


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