A Phase 2, Double-blind, Randomized, Placebo-controlled Study to Investigate the Safety, Tolerability, and Efficacy of JBT-101 in Subjects With Dermatomyositis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 22
- 试验地点
- 1
- 主要终点
- Change in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) From Baseline in Part A.
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability and efficacy of JBT-101 in adult subjects with skin-predominant, dermatomyositis (DM) that is refractory to at least 3 months treatment with hydroxychloroquine.
详细描述
Part A: An interventional, double-blind, randomized, placebo-control design will be used to test JBT-101 in about 22 eligible male or female subjects ≥ 18 and ≤ 70 years of age with moderate-to-severe active skin-predominant dermatomyositis.
Part B: A one-year open-label design to test JBT-101 in subjects who completed Part A without permanent discontinuation of study product because of safety or tolerability reasons.
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
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
JBT-101
Part A: JBT-101 20 mg capsule once a day on Days 1-28, then 20 mg capsule twice a day on Days 29-84.
Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE.
干预措施: JBT-101 (Drug)
Placebo
Part A: Placebo capsule once a day on Days 1-28, then placebo capsule twice a day on Days 29-84.
Part B: Placebo twice daily on Days 1 - 365 of the OLE.
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) From Baseline in Part A.
时间窗: Part A: 84-day treatment period (Change from the Baseline CDSAI score at Day 84)
The CDASI is a validated outcome measure that systematically quantifies cutaneous DM disease activity, In the CDASI, DM skin disease activity is scored from 0 to 100 based on the physician's evaluation of erythema, scale, and erosion or ulceration at 15 anatomic locations as well as alopecia, Gottron's sign or papules on the hands, and periungual changes. A 5-point or greater decrease in the CDASI activity score indicates clinically relevant improvement based on statistical analysis using a receiver operating characteristic curve to maximize sensitivity and specificity
Number of Participants With Treatment Emergent Adverse Events as a Measure of Safety and Tolerability
时间窗: Part A: to Day 84
Number of participants with treatment emergent adverse events were assessed as a measure of safety and tolerability
次要结局
- Change in Patient-reported Outcomes From Baseline at 84 Days for Part A(Part A: 84-day treatment period)