Fibrodysplasia Ossificans Progressiva (FOP)
Conditions
Keywords
fibrodysplasia ossificans progressiva (FOP), heterotopic ossification
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).
Interventions
DRUGINCB000928
INCBG000928 will be administered QD orally.
DRUGPlacebo
Placebo will be administered QD orally.
Sponsors
Incyte Corporation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
2 Years to 99 Years
Healthy volunteers
No
Inclusion criteria
* Female and male participants: * Cohort 1: ≥ 12 years of age. * Cohort 2: 6 to \< 12 years of age. * Cohort 3: 2 to \< 12 years of age (after eDMC review of safety 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline | 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
| Measure | Time frame | Description |
|---|---|---|
| Double Blind Period: Number of new HO lesions from baseline | 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. |
| Double Blind Period: Total volume of new HO lesions from baseline | 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. |
| Double Blind Period: Change in the total volume of all HO lesions from baseline | 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. |
| Double Blind Period: Number of new flares from baseline | Week 24 | Based on Fibrodysplasia Ossificans Progressiva - Patient RepOrted syMPtoms Tool (FOP-PROMPT). |
| Number of Participants with Treatment Emergent Adverse Events (TEAE) | Up to 316 weeks | Defined as any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug. |
| Open-Label Extension: Occurrence of new HO lesions from Week 24 | Week 48 | 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 | Week 48 | 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: Total volume of new HO lesions from Week 24 | Week 48 | 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: Change in the total volume of all HO lesions from Week 24 | Week 48 | 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 flares from Week 24 | Week 48 | 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. |
| Pharmacokinetics Parameter: Cmax of INCB000928 | Baseline, Weeks 12, 24, 48 and 76 | Maximum observed concentration. |
| Pharmacokinetics Parameter: Tmax of INCB000928 | Baseline, Weeks 12, 24, 48 and 76 | Time to maximum concentration. |
| Pharmacokinetics Parameter: Cmin of INCB000928 | Baseline, Weeks 12, 24, 48 and 76 | Minimum observed concentration. |
| Pharmacokinetics Parameter: AUCt of INCB000928 | Baseline, Weeks 12, 24, 48 and 76 | Area under the plasma concentration-time curve from time 0 to the last quantifiable measurable plasma concentration |
Countries
Argentina, Australia, Brazil, Canada, Chile, China, France, Germany, Italy, Mexico, Netherlands, New Zealand, South Africa, South Korea, Spain, United Kingdom, United States
Contacts
CONTACTIncyte Corporation Call Center (US)
CONTACTIncyte Corporation Call Center (ex-US)
STUDY_DIRECTORAmanda McBride, MD
Incyte Corporation
Outcome results
None listed