A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 98
- 试验地点
- 51
- 主要终点
- Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 2 Years 至 99 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
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.
干预措施: Placebo (Drug)
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.
干预措施: INCB000928 (Drug)
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.
干预措施: INCB000928 (Drug)
Cohort 3
Participants (2 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.
干预措施: INCB000928 (Drug)
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.
干预措施: Placebo (Drug)
Cohort 3
Participants (2 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.
干预措施: Placebo (Drug)
结局指标
主要结局
Double Blind Period: Occurrence of new heterotopic ossification (HO) lesions from baseline
时间窗: 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.
次要结局
- 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)