A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial to Evaluate Efficacy and Safety of Lenabasum in Dermatomyositis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 176
- 试验地点
- 52
- 主要终点
- Efficacy of lenabasum 20 mg BID compared to placebo BID as measured by Total Improvement Score (TIS)
研究概览
简要总结
This is a Phase 3 multicenter, double-blind, randomized, placebo-controlled study assessing the efficacy and safety of lenabasum for the treatment of dermatomyositis. Approximately 150 subjects will be enrolled in this study at about 60 sites in North America, Europe, and Asia. The planned duration of double-blind treatment with study drug is up to 52 weeks.
详细描述
Subjects will be randomized to receive lenabasum 20 mg twice per day, lenabasum 5 mg twice per day, or placebo twice per day in a 2:1:2 ratio. The primary efficacy outcome at Week 28 will compare lenabasum 20 mg BID to placebo the Total Improvement Score (TIS), which is a weighted composite measure of improvement from baseline in six endpoints: Physician Global Assessment of Disease Activity, Physician Assessment of Extramuscular Disease Activity, Patient Global Assessment of Disease Activity, Health Assessment Questionnaire (patient-reported disability), Manual Muscle Testing (MMT), and muscle enzymes.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Fulfill at least one of the following criteria for dermatomyositis:
- •Bohan and Peter criteria (Bohan and Peter, 1975a; Bohan and Peter 1975b)
- •ACR/EULAR criteria (Lundberg et al, 2017)
- •Disease activity/severity fulfills at least one of the following three criteria:
- •MDGA ≥ 3 cm (0 - 10 cm Visual Analog Scale [VAS]) and MMT-8 score ≤ 142 (out of 150 total possible)
- •Sum of MDGA, PtGA and EMGA VAS scores is ≥ 10 cm (0-10 cm VAS for each)
- •MDGA ≥ 3 cm (0-10 cm VAS) and CDASI activity score of > 14
- •Stable doses of immunosuppressive medications for DM as defined by:
- •Unchanged dose of oral corticosteroids ≤ 20 mg per day prednisone or equivalent for ≥ 4 weeks before Visit 1
- •Unchanged dose of immunosuppressive medications other than oral corticosteroids for ≥ 8 weeks before Screening
排除标准
- •Unstable DM or DM with end-stage organ involvement at Screening or Visit 1
- •Significant diseases or conditions other than DM that may influence response to the study drug or safety
- •Any of the following values for laboratory tests at Screening:
- •A positive pregnancy test (or at Visit 1)
- •Hemoglobin < 9 g/dL in males and < 8 g/dL in females
- •Neutrophils < 1.0 × 10^9/L
- •Platelets < 75 × 10^9/L
- •Creatinine clearance < 50 mL/min on screening blood test, per the Modification of Diet in Renal Disease Study or in 24 hour urine creatine clearance measurement
研究组 & 干预措施
Lenabasum 20 mg
Subjects will receive lenabasum 20 mg twice daily
干预措施: Lenabasum 20 mg (Drug)
Lenabasum 5 mg
Subjects will receive lenabasum 5 mg twice daily
干预措施: Lenabasum 5 mg (Drug)
Placebo
Subjects will receive placebo twice daily
干预措施: Placebo (Drug)
结局指标
主要结局
Efficacy of lenabasum 20 mg BID compared to placebo BID as measured by Total Improvement Score (TIS)
时间窗: Week 28
TIS from IMAC Core Set Measures (CSM) will be calculated following Aggarwal et al (2017) recommendations. Scores are based on a 0 - 100 scale; higher scores indicate better improvement in myositis.
次要结局
- Subjects who achieve Definition of Improvement (DOI)(Week 28)
- TIS in subjects receiving immunosuppressive therapies (including corticosteroids) for > 1 year at Baseline(Week 52)
- Change in Forced vital capacity (FVC) absolute, in all subjects and those with interstitial lung disease (ILD) at Baseline.(Week 28)
- Subjects who improve by at least one category on the Investigator Global Assessment (IGA) scale of skin activity(Week 28)
- Subjects who achieve TIS >= 40 (at least moderate improvement)(Week 28)
- Change in Forced vital capacity (FVC) percent predicted, in all subjects and those with interstitial lung disease (ILD) at Baseline.(Week 28)
- TIS at Visit 10(Week 52)
- Change in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) activity score(Week 28)
- TIS, lenabasum 5 mg BID versus placebo(Week 28)