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

Phase 2RecruitingNCT05090891

Incyte Corporation98 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Fibrodysplasia Ossificans Progressiva (FOP)

Interventions

INCB000928DRUG

INCBG000928 will be administered QD orally.

PlaceboDRUG

Placebo will be administered QD orally.

Eligibility

Sex: ALLMin age: 2 YearsMax age: 99 Years

Medical Language ↔ Plain English

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.

Locations (27)

Outcomes

Primary Outcomes

Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline

HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period.

Time frame: Week 24

Secondary Outcomes

Double Blind Period: Number of new HO lesions from baseline

HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period.

Time frame: Week 24

Double Blind Period: Total volume of new HO lesions from baseline

HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period.

Time frame: Week 24

Double Blind Period: Change in the total volume of all HO lesions from baseline

HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period.

Time frame: Week 24

Double Blind Period: Number of new flares from baseline

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

Time frame: 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.

Time frame: Up to 316 weeks

Open-Label Extension: Occurrence of new HO lesions from Week 24

Central Contacts

Incyte Corporation Call Center (US)

CONTACT

1.855.463.3463 medinfo@incyte.com

Incyte Corporation Call Center (ex-US)

CONTACT

+800 00027423 eumedinfo@incyte.com

Data from ClinicalTrials.gov

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University of California San Francisco Medical Center

San Francisco, California, United States

WITHDRAWN

Mayo Clinic Rochester

Rochester, Minnesota, United States

RECRUITING

Children'S Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

RECRUITING

Penn Medicine - Perelman Center For Advanced Medicine

Philadelphia, Pennsylvania, United States

ACTIVE_NOT_RECRUITING

Hospital Italiano de Buenos Aires

Ciudad Autonoma Buenos Aires, Argentina

NOT_YET_RECRUITING

Royal North Shore Hospital

St Leonards, New South Wales, Australia

COMPLETED

Murdoch Children'S Research Institute

Parkville, Victoria, Australia

RECRUITING

Albert Einstein Israelite Hospital

São Paulo, Brazil

RECRUITING

University Health Network Toronto General Hospital

Toronto, Ontario, Canada

ACTIVE_NOT_RECRUITING

Centro de Estudios Reumatologicos

Santiago, Chile

RECRUITING

...and 17 more locations

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.

Open-Label Extension: Number of new HO lesions from Week 24

Time frame: Week 48

Open-Label Extension: Total volume of new HO lesions from Week 24

Time frame: Week 48

Open-Label Extension: Change in the total volume of all HO lesions from Week 24

Time frame: Week 48

Open-Label Extension: Number of new flares from Week 24

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.

Time frame: Week 48

Pharmacokinetics Parameter: Cmax of INCB000928

Maximum observed concentration.

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

Pharmacokinetics Parameter: Tmax of INCB000928

Time to maximum concentration.

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

Pharmacokinetics Parameter: Cmin of INCB000928

Minimum observed concentration.

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

Pharmacokinetics Parameter: AUCt of INCB000928

Area under the plasma concentration-time curve from time 0 to the last quantifiable measurable plasma concentration

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