A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial to Evaluate Efficacy and Safety of Lenabasum in Diffuse Cutaneous Systemic Sclerosis
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 365
- 试验地点
- 74
- 主要终点
- Efficacy of lenabasum compared to placebo for the American College of Rheumatology Combined Response Index in diffuse cutaneous Systemic Sclerosis score.
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 diffuse cutaneous systemic sclerosis (SSc). Approximately 354 subjects will be enrolled in this study at about 60 sites in North America, Europe, Australia, and Asia. The planned duration of treatment with study drug is 52 weeks.
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
- •≥ 18 years of age at the time Informed Consent is signed.
- •Diffuse cutaneous SSc (skin thickening on upper arms, upper legs, or trunk).
- •Disease duration ≤ 6 years from the first non-Raynaud's symptom.
- •No new or increased doses of immunosuppressive medications within 8 weeks prior to Screening.
排除标准
- •Unstable SSc or SSc with end-stage organ involvement at Screening or Visit
- •Any of the following values for laboratory tests at Screening:
- •A positive pregnancy test in women of childbearing potential;
- •Hemoglobin < 9 g/dL for males and < 8 g/dL for females;
- •Neutrophils < 1.0 ×10^9/L;
- •Platelets < 75 ×10^9/L;
- •Creatinine clearance < 50 mL/min according to the Modification of Diet in Renal Disease (MDRD) Study equation;
- •Aspartate aminotransferase or alanine aminotransferase > 2.0 × upper limit of normal.
- •Any medical condition or concurrent medical therapies at Screening or Visit 1, including a history of non-compliance with medical treatments, that may put the subject at greater safety risk, influence response to study product, or interfere with study assessments.
研究组 & 干预措施
Cohort 2
Lenabasum 20 mg BID
干预措施: Lenabasum 20 mg (Drug)
Cohort 1
Lenabasum 5 mg BID
干预措施: Lenabasum 5 mg (Drug)
Cohort 3
Placebo BID
干预措施: Placebo oral capsule (Other)
结局指标
主要结局
Efficacy of lenabasum compared to placebo for the American College of Rheumatology Combined Response Index in diffuse cutaneous Systemic Sclerosis score.
时间窗: American College of Rheumatology Combined Response Index score through study completion, up to 1 year.
The ACR CRISS exponential algorithm determines the predicted probability of improvement from baseline, incorporating change in mRSS, FVC % predicted, physician and patient global assessments, and HAQ-DI. The outcome is a continuous variable between 0.0 and 1.0 (0 - 100%). A higher score indicates greater improvement.
次要结局
- Efficacy of lenabasum compared to placebo for the change from baseline in modified Rodnan skin score.(Change from baseline through study completion, up to 1 year.)
- Efficacy of lenabasum compared to placebo for the change from baseline in Health Assessment Questionnaire - Disability Index.(Change from baseline through study completion, up to 1 year.)
- Efficacy of lenabasum compared to placebo for the change from baseline in forced vital capacity.(Change from baseline through study completion, up to 1 year.)