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

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).

Study Overview

• Status: Recruiting
• Conditions: Fibrodysplasia Ossificans Progressiva (FOP)
• Intervention / Treatment: Drug: INCB000928; Drug: placebo

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Incyte Corporation Call Center (US); Phone Number: 1.855.463.3463; Email: medinfo@incyte.com
• Study Contact Backup: Name: Incyte Corporation Call Center (ex-US); Phone Number: +800 00027423; Email: eumedinfo@incyte.com

Study Locations

• Argentina
  • Ciudad Autonoma Buenos Aires, Argentina, C1199ABB
    • Not yet recruiting
    • Hospital Italiano de Buenos Aires
• Australia
  • Westmead, Australia, 02145
    • Withdrawn
    • Westmead Hospital
  • New South Wales
    • St Leonards, New South Wales, Australia, 02065
      • Recruiting
      • Royal North Shore Hospital
  • Victoria
    • Parkville, Victoria, Australia, 03052
      • Recruiting
      • Murdoch Children's Research Institute
• Brazil
  • San Paolo, Brazil, 05652-900
    • Recruiting
    • Albert Einstein Israelite Hospital
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 2C4
      • Recruiting
      • University Health Network Toronto General Hospital
• Chile
  • Santiago, Chile, 7501126
    • Recruiting
    • Centro de Estudios Reumatológicos
• China
  • Beijing, China, 100045
    • Recruiting
    • Beijing Childrens Hospital Capital Medical University
  • Shanghai, China, 200065
    • Recruiting
    • Tongji Hospital of Tongji University
  • Shanghai, China, 200127
    • Recruiting
    • Shanghai Childrens Medical Center
  • Shanghai, China, 201102
    • Recruiting
    • Childrens Hospital of Fudan University
• France
  • Paris, France, 75015
    • Recruiting
    • Hôpital Necker-Enfants Malades
  • Paris, France, 75010
    • Recruiting
    • Ap-Hp Hopital Lariboisiere
• Germany
  • Koln, Germany, 50931
    • Recruiting
    • Uniklinik Koln
• Italy
  • Genova, Italy, 16147
    • Withdrawn
    • Ospedale Pediatrico G. Gaslini
  • Rome, Italy, 00168
    • Recruiting
    • Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore
• Korea, Republic of
  • Seoul, Korea, Republic of, 03080
    • Recruiting
    • Seoul National University Hospital
• Mexico
  • Tlalpan, Mexico, 14389
    • Recruiting
    • Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra
• Netherlands
  • Amsterdam, Netherlands, 1081 HV
    • Not yet recruiting
    • Amsterdam UMC - VU Medisch Centrum (VUmc)
• New Zealand
  • Auckland, New Zealand, 01023
    • Not yet recruiting
    • Starship Childrens Hospital
• Portugal
  • Lisbon, Portugal, 1649-035
    • Recruiting
    • Centro Hospitalar Universitario de Lisboa Norte, Hospital Santa Maria
• Russian Federation
  • Irkutsk, Russian Federation, 664003
    • Withdrawn
    • Irkutsk State Medical University
  • Moscow, Russian Federation, 115478
    • Withdrawn
    • Nasonova Research Institute of Rheumatology
  • St. Petersburg, Russian Federation, 194100
    • Withdrawn
    • Saint Petersburg State Pediatric Medical University
• South Africa
  • Cape Town, South Africa, 07925
    • Recruiting
    • Groote Schuur Hospital Radiation Oncology
• Spain
  • Madrid, Spain, 28034
    • Recruiting
    • Hospital Universitario Ramón y Cajal
• Turkey
  • Izmir, Turkey, 35100
    • Not yet recruiting
    • Ege University Faculty of Medicine
• United Kingdom
  • Manchester, United Kingdom, M13 9PL
    • Withdrawn
    • Royal Manchester Childrens Hospital - Department of Paediatric Endocrinology
  • Stanmore, United Kingdom, HA7 4LP
    • Recruiting
    • Royal National Orthopaedic Hospital
• United States
  • California
    • San Francisco, California, United States, 94143
      • Withdrawn
      • University of California San Francisco Medical Center
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic Rochester
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Children's Hospital of Philadelphia
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Penn Medicine - Perelman Center for Advanced Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 99 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Female and male adults and adolescents ≥ 12 years of age with a 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Group A: INCB000928; Participants will receive INCB000928 for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 52 weeks.	Drug: INCB000928; INCBG000928 will be administered QD orally.; Other Names: INB000928 will be administered QD orally; Drug: placebo; placebo will be administered QD orally
Placebo Comparator: Group B: Placebo followed by INCB000928; Participants will receive placebo for 24 weeks (double-blind period). Participants who completed the double-blind period will receive INCB000928 in the 52 week open-label extension period.	Drug: placebo; placebo will be administered QD orally

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Double Blind Period: Total volume of new heterotopic ossification (HO)	HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period.	Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Double-blind Period: Total number of new flares	Defined as number of new flares compared to baseline during the double-blind period.	Baseline, Week 12 and Week 24
Double-blind Period: Proportion of participants with a clinically meaningful improvement in the flare-related symptoms	Flare related symptoms will be assessed by via an electronic PRO during the double-blind period.	Baseline, Week 12 and 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.	Up to 80 weeks

Collaborators and Investigators

• Sponsor: Incyte Corporation
• Investigators: Study Director: Kurt Brown, MD, Incyte Corporation

Study record dates

Study Major Dates

• Study Start (Actual): May 5, 2022
• Primary Completion (Estimated): April 18, 2025
• Study Completion (Estimated): April 28, 2028

Study Registration Dates

• First Submitted: October 8, 2021
• First Submitted That Met QC Criteria: October 14, 2021
• First Posted (Actual): October 25, 2021

Study Record Updates

• Last Update Posted (Actual): April 19, 2024
• Last Update Submitted That Met QC Criteria: April 18, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: fibrodysplasia ossificans progressiva (FOP); heterotopic ossification
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Muscular Diseases; Myositis; Myositis Ossificans
• Other Study ID Numbers: INCB 00928-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.
• IPD Sharing Time Frame: Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
• IPD Sharing Access Criteria: Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No