Clinical trial NCT05090891: To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Fibrodysplasia Ossificans Progressiva (FOP)
Keywords: fibrodysplasia ossificans progressiva (FOP)
heterotopic ossification
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2021-10-25
Enrollment: 98
Sex: ALL
Minimum age (years): 2
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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 criteria: Inclusion Criteria:

* 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.
* 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.

Primary outcomes:
measure: Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline
timeFrame: Week 24
Secondary outcomes:
measure: Double Blind Period: Number of new HO lesions from baseline
timeFrame: Week 24
measure: Double Blind Period: Total volume of new HO lesions from baseline
timeFrame: Week 24
measure: Double Blind Period: Change in the total volume of all HO lesions from baseline
timeFrame: Week 24
measure: Double Blind Period: Number of new flares from baseline
timeFrame: Week 24
measure: Number of Participants with Treatment Emergent Adverse Events (TEAE)
timeFrame: Up to 316 weeks
measure: Open-Label Extension: Occurrence of new HO lesions from Week 24
timeFrame: Week 48
measure: Open-Label Extension: Number of new HO lesions from Week 24
timeFrame: Week 48
measure: Open-Label Extension: Total volume of new HO lesions from Week 24
timeFrame: Week 48
measure: Open-Label Extension: Change in the total volume of all HO lesions from Week 24
timeFrame: Week 48
measure: Open-Label Extension: Number of new flares from Week 24
timeFrame: Week 48
measure: Pharmacokinetics Parameter: Cmax of INCB000928
timeFrame: Baseline, Weeks 12, 24, 48 and 76
measure: Pharmacokinetics Parameter: Tmax of INCB000928
timeFrame: Baseline, Weeks 12, 24, 48 and 76
measure: Pharmacokinetics Parameter: Cmin of INCB000928
timeFrame: Baseline, Weeks 12, 24, 48 and 76
measure: Pharmacokinetics Parameter: AUCt of INCB000928
timeFrame: Baseline, Weeks 12, 24, 48 and 76
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California San Francisco Medical Center
state: California
city: Rochester
country: United States
facility: Mayo Clinic Rochester
state: Minnesota
city: Philadelphia
country: United States
facility: Children'S Hospital of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: Penn Medicine - Perelman Center For Advanced Medicine
state: Pennsylvania
city: Ciudad Autonoma Buenos Aires
country: Argentina
facility: Hospital Italiano de Buenos Aires
city: St Leonards
country: Australia
facility: Royal North Shore Hospital
state: New South Wales
city: Parkville
country: Australia
facility: Murdoch Children'S Research Institute
state: Victoria
city: San Paolo
country: Brazil
facility: Albert Einstein Israelite Hospital
city: Toronto
country: Canada
facility: University Health Network Toronto General Hospital
state: Ontario
city: Santiago
country: Chile
facility: Centro de Estudios Reumatologicos
city: Beijing
country: China
facility: Beijing Childrens Hospital Capital Medical University
city: Shanghai
country: China
facility: Tongji Hospital of Tongji University
city: Shanghai
country: China
facility: Shanghai Childrens Medical Center
city: Shanghai
country: China
facility: Childrens Hospital of Fudan University
city: Paris
country: France
facility: Ap-Hp Hopital Lariboisiere
city: Paris
country: France
facility: Hopital Necker-Enfants Malades
city: Koln
country: Germany
facility: Uniklinik Koln
city: Genova
country: Italy
facility: Ospedale Pediatrico G. Gaslini
city: Rome
country: Italy
facility: Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Tlalpan
country: Mexico
facility: Instituto Nacional de Rehabilitacion Luis Guillermo Ibarra
city: Amsterdam
country: Netherlands
facility: Amsterdam Umc - Vu Medisch Centrum (Vumc)
city: Auckland
country: New Zealand
facility: Starship Childrens Hospital
city: Lisbon
country: Portugal
facility: Centro Hospitalar Universitario de Lisboa Norte, Hospital Santa Maria
city: Cape Town
country: South Africa
facility: Groote Schuur Hospital Radiation Oncology
city: Madrid
country: Spain
facility: Hospital Universitario Ramon Y Cajal
city: Izmir
country: Turkey
facility: Ege University Faculty of Medicine
city: Manchester
country: United Kingdom
facility: Royal Manchester Childrens Hospital - Department of Paediatric Endocrinology
city: Stanmore
country: United Kingdom
facility: Royal National Orthopaedic Hospital
Study officials:
affiliation: Incyte Corporation
name: Amanda McBride, MD
role: STUDY_DIRECTOR
References: