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

Fibrodysplasia Ossificans Progressiva (FOP) Clinical Trial

— Progress  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05090891
• Other study ID #: INCB 00928-201
• Status: Recruiting
• Phase: Phase 2
• Start date: May 5, 2022
• Est. completion date: April 28, 2028

Study information

• Verified date: April 2024
• Source: Incyte Corporation
• Contact: Incyte Corporation Call Center (US)
• Phone: 1.855.463.3463
• Email: medinfo[@]incyte.com
• Is FDA regulated: No
• Study type: Interventional

Clinical 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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 60
• Est. completion date: April 28, 2028
• Est. primary completion date: April 18, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 99 Years

Eligibility

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.

Related Conditions & MeSH terms

• Fibrodysplasia Ossificans Progressiva (FOP)

Intervention

Drug:
• INCB000928
INCBG000928 will be administered QD orally.
• placebo
placebo will be administered QD orally

Locations

Country	Name	City	State
Argentina	Hospital Italiano de Buenos Aires	Ciudad Autonoma Buenos Aires
Australia	Murdoch Children'S Research Institute	Parkville	Victoria
Australia	Royal North Shore Hospital	St Leonards	New South Wales
Australia	Westmead Hospital	Westmead
Brazil	Albert Einstein Israelite Hospital	San Paolo
Canada	University Health Network Toronto General Hospital	Toronto	Ontario
Chile	Centro de Estudios Reumatologicos	Santiago
China	Beijing Childrens Hospital Capital Medical University	Beijing
China	Childrens Hospital of Fudan University	Shanghai
China	Shanghai Childrens Medical Center	Shanghai
China	Tongji Hospital of Tongji University	Shanghai
France	Ap-Hp Hopital Lariboisiere	Paris
France	Hopital Necker-Enfants Malades	Paris
Germany	Uniklinik Koln	Koln
Italy	Ospedale Pediatrico G. Gaslini	Genova
Italy	Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore	Rome
Korea, Republic of	Seoul National University Hospital	Seoul
Mexico	Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra	Tlalpan
Netherlands	Amsterdam Umc - Vu Medisch Centrum (Vumc)	Amsterdam
New Zealand	Starship Childrens Hospital	Auckland
Portugal	Centro Hospitalar Universitario de Lisboa Norte, Hospital Santa Maria	Lisbon
Russian Federation	Irkutsk State Medical University	Irkutsk
Russian Federation	Nasonova Research Institute of Rheumatology	Moscow
Russian Federation	Saint Petersburg State Pediatric Medical University	St. Petersburg
South Africa	Groote Schuur Hospital Radiation Oncology	Cape Town
Spain	Hospital Universitario Ramon Y Cajal	Madrid
Turkey	Ege University Faculty of Medicine	Izmir
United Kingdom	Royal Manchester Childrens Hospital - Department of Paediatric Endocrinology	Manchester
United Kingdom	Royal National Orthopaedic Hospital	Stanmore
United States	Children'S Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Penn Medicine - Perelman Center For Advanced Medicine	Philadelphia	Pennsylvania
United States	Mayo Clinic Rochester	Rochester	Minnesota
United States	University of California San Francisco Medical Center	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Incyte Corporation

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Brazil,  Canada,  Chile,  China,  France,  Germany,  Italy,  Korea, Republic of,  Mexico,  Netherlands,  New Zealand,  Portugal,  Russian Federation,  South Africa,  Spain,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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