A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
Overview
- Phase
- Phase 2
- Intervention
- INCB000928
- Conditions
- Fibrodysplasia Ossificans Progressiva (FOP)
- Sponsor
- Incyte Corporation
- Enrollment
- 98
- Locations
- 27
- Primary Endpoint
- Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline
- Status
- Recruiting
- Last Updated
- 4 months ago
Overview
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).
Investigators
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 ≥
- •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.
Arms & Interventions
Cohort 1
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.
Intervention: INCB000928
Cohort 1
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.
Intervention: Placebo
Cohort 2
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.
Intervention: INCB000928
Cohort 2
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.
Intervention: Placebo
Cohort 3
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.
Intervention: INCB000928
Cohort 3
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.
Intervention: Placebo
Outcomes
Primary Outcomes
Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline
Time Frame: Week 24
HO will be assessed by low dose whole-body computed tomography (WBCT) (excluding the head) compared to baseline during the double-blind period.
Secondary Outcomes
- Open-Label Extension: Occurrence of new HO lesions from Week 24(Week 48)
- Double Blind Period: Number of new HO lesions from baseline(Week 24)
- Double Blind Period: Total volume of new HO lesions from baseline(Week 24)
- Double Blind Period: Change in the total volume of all HO lesions from baseline(Week 24)
- Double Blind Period: Number of new flares from baseline(Week 24)
- Number of Participants with Treatment Emergent Adverse Events (TEAE)(Up to 316 weeks)
- Open-Label Extension: Number of new HO lesions from Week 24(Week 48)
- Open-Label Extension: Total volume of new HO lesions from Week 24(Week 48)
- Open-Label Extension: Change in the total volume of all HO lesions from Week 24(Week 48)
- Open-Label Extension: Number of new flares from Week 24(Week 48)
- Pharmacokinetics Parameter: Cmax of INCB000928(Baseline, Weeks 12, 24, 48 and 76)
- Pharmacokinetics Parameter: Tmax of INCB000928(Baseline, Weeks 12, 24, 48 and 76)
- Pharmacokinetics Parameter: Cmin of INCB000928(Baseline, Weeks 12, 24, 48 and 76)
- Pharmacokinetics Parameter: AUCt of INCB000928(Baseline, Weeks 12, 24, 48 and 76)