A Phase IIa, Double-Blind, Randomised, Intracohort Placebo-Controlled, Multicentre Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of EHP-101 in Patients With Diffuse Cutaneous Systemic Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 暂停
- 发起方
- 入组人数
- 36
- 试验地点
- 23
- 主要终点
- Incidence and severity of Treatment Emergent Adverse Events
研究概览
简要总结
The purpose of this trial is to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of EHP-101 in adult subjects with diffuse cutaneous Systemic Sclerosis (dcSSc).
详细描述
An interventional, double-blind, randomized, intracohort placebo-controlled design will be used to test safety, tolerability, pharmacokinetics, and preliminary efficacy of EHP-101 in 36 patients ≥ 18 and ≤ 74 years of age with documented dcSSc. There is a screening period of 28 days, 84 days treatment period, and 28 days 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
- Double (Participant, Investigator)
盲法说明
Double (intracohort)
入排标准
- 年龄范围
- 18 Years 至 74 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients male and female ≥18 years and ≤74 years at the time of consent;
- •American College of Rheumatology/ European League Against Rheumatism 2013 Criteria for SSc; dcSSc (skin thickening on upper arms, upper legs, or trunk);
- •Documented SSc for up to 6 years from the first non-Raynaud's phenomenon with a total mRSS of ≥15;
- •No new or increased doses of immunosuppressants medications within 3 months prior to Screening;
- •Effective method of contraception for participants and their partners.
排除标准
- •Severe or unstable Systemic Sclerosis (SSc) or SSc with end-stage organ failure;
- •Patient with FVC <60%;
- •History of clinically significant medical condition or concurrent medical therapies that would exclude the patient, preclude participation in the clinical trial, influence response to study product, or interfere with study assessments;
- •History of gastrointestinal dysmotility requiring total parenteral nutrition or hospitalization within 6 months before Visit 1;
- •Any one of the following values for laboratory tests at screening:
- •Haemoglobin <9 g/dL;
- •Neutrophils <1.0 x 10^9/L;
- •Platelets <75 x 10^9/L;
- •Estimated creatinine clearance <50 mL/min according to the Cockcroft-Gault equation;
- •Serum transaminases >2.0 x upper normal limit;
- •Total bilirubin ≥1.5 x upper limit of normal.
研究组 & 干预措施
EHP-101 low dose once a day
干预措施: Patients will be randomized to receive EHP-101 or Placebo (Drug)
EHP-101 low dose twice a day
干预措施: Patients will be randomized to receive EHP-101 or Placebo (Drug)
EHP-101 high dose once a day
干预措施: Patients will be randomized to receive EHP-101 or Placebo (Drug)
EHP-101 high dose twice a day
干预措施: Patients will be randomized to receive EHP-101 or Placebo (Drug)
结局指标
主要结局
Incidence and severity of Treatment Emergent Adverse Events
时间窗: Day 113
This safety outcome combines the measure of the number of subjects experiencing adverse events (AEs), the nature and severity of those AEs and their relationship to the study treatments.
次要结局
- Treatment effect of EHP-101 compared to placebo as measured by the American College of Rheumatology composite response index in diffuse cutaneous Systemic Sclerosis(Day 85 and Day 113)
- Treatment effect of EHP-101 compared to placebo in modified Rodnan Skin Score(Day 85 and Day 113)
- Treatment effect of EHP-101 compared to placebo in Scleroderma Health Assessment Questionnaire - Disability Index(Day 85 and Day 113)
- Treatment effect of EHP-101 compared to placebo in forced vital capacity percent predicted(Day 85 and Day 113)
- Treatment effect of EHP-101 compared to placebo in physician global assessment score(Day 85 and Day 113)
- Treatment effect of EHP-101 compared to placebo in patient global assessments score(Day 85 and Day 113)