Double-blind, Randomized, 8-week Placebo-controlled, and 16-week Open-label Extension Study Investigating the Safety, Pharmacokinetics and Pharmacodynamics of SAR100842 Given Orally to Patients With Diffuse Cutaneous Systemic Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Sanofi
- 入组人数
- 32
- 试验地点
- 13
- 主要终点
- Safety and tolerability during the 8 week treatment period (core part): Number of patients reporting adverse events
研究概览
简要总结
Primary Objective:
- To evaluate safety and tolerability of 8-week oral administration of SAR100842 in patients with diffuse cutaneous systemic sclerosis.
Secondary Objectives:
- To evaluate the pharmacodynamic effect of SAR100842 in patients with systemic sclerosis as measured by disease related biomarkers and Lysophosphatidic acid (LPA) receptor signaling markers in blood and skin;
- To explore the effect of SAR100842 on skin thickness in patients with systemic sclerosis as measured by the modified Rodnan Skin Score (mRSS);
- To explore the effect of SAR100842 on quality of life as measured by the Scleroderma Modified Health Assessment Questionnaire (SHAQ);
- To document long term safety of SAR100842 during the extension part.
详细描述
Core part: randomized, double-blind, placebo-controlled study - 8-week treatment Extension part for participants completing the core part: Open label non-controlled study - 16-week treatment
Each patient's participation in the study will be approximately 13 or 33 weeks depending on their participation in the extension part: up to 2 weeks of screening, 8 weeks of treatment in the core part, 1 to 30 days wash-out between core part and extension part , 16 weeks of treatment in the extension part and 3 weeks of follow up.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
SAR100842
Core part: SAR100842 300 mg, oral administration twice daily, for 8 weeks
Extension part: SAR100842 300 mg, oral administration twice daily, for 16 additional weeks
干预措施: SAR100842 (Drug)
Placebo
Core part: Placebo (for SAR100842), oral administration twice daily, for 8 weeks
Extension part: SAR100842 300 mg, oral administration twice daily, for 16 additional weeks
干预措施: SAR100842 (Drug)
Placebo
Core part: Placebo (for SAR100842), oral administration twice daily, for 8 weeks
Extension part: SAR100842 300 mg, oral administration twice daily, for 16 additional weeks
干预措施: Placebo (for SAR100842) (Drug)
结局指标
主要结局
Safety and tolerability during the 8 week treatment period (core part): Number of patients reporting adverse events
时间窗: Up to 8 weeks
次要结局
- Change from baseline to Week 8 in biomarkers obtained from blood and skin(Day 1 and Week 8 (core part))
- Change from baseline to Week 8 in Modified Rodnan Skin Score (mRSS)(Day 1 and Week 8 (core part))
- Change from baseline to Week 8 in Scleroderma Health Assessment Questionnaire (SHAQ) score(Day 1 and Week 8 (core part))