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

Incyte

Status and phase: Enrolling; Phase 2

Conditions: Fibrodysplasia Ossificans Progressiva (FOP)

Treatments: Drug: Placebo; Drug: INCB000928

Study type: Interventional

Funder types: Industry

Identifiers

NCT05090891

2021-002286-17 (EudraCT Number)

INCB 00928-201

2023-504129-38-00 (Registry Identifier)

Details and patient eligibility

About

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).

Enrollment: 98 estimated patients

Sex: All

Ages: 2 to 99 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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98 participants in 3 patient groups

Cohort 1

Experimental group

Description:

Participants (≥ 12 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.

Treatment:

Drug: INCB000928

Drug: Placebo

Cohort 2

Experimental group

Description:

Participants (6 to \< 12 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.

Treatment:

Drug: INCB000928

Drug: Placebo

Cohort 3

Experimental group

Description:

Participants (2 to \< 6 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.

Treatment:

Drug: INCB000928

Drug: Placebo