A Phase 2, Open-label, Single-arm, Intra-subject Dose-escalation Study to Investigate the Biological Activity, Safety, Tolerability, and Pharmacokinetics of Ponesimod in Subjects With Symptomatic Moderate or Severe Chronic GVHD Inadequately Responding to First or Second Line Therapy
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 1
- 主要终点
- Change in Peripheral Absolute Lymphocyte Count From Baseline to Week 4, 8 and 12
研究概览
简要总结
Chronic graft versus host diseasre (GVHD) is a serious reaction that might occur in a person (the host) who has received cells or organs (graft) from another person because the graft attacks the host's cells. Currently there are no approved therapies for chronic GVHD in the USA, and patients with chroninc GVHD are treated with immunosuppressant drugs. T-lymphocytes (a type of white blood cells) are likely to play a role in the development of chronic GVHD. Due to the capacity of ponesimod to block the traffic of T-lymphocytes, ponesimod may be a new therapeutic approach to treat chroninc GVHD.
The main objective of this study is to assess the effectiveness and safety of several doses of ponesimod in subjects with chronic GVHD who did not respond to standard available treatments.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Signed informed consent
- •Symptomatic moderate or severe chronic GVHD patients in need of a change of systemic immunosuppressant (IS) therapy
- •Women of child bearing potential must have a negative pregnancy test and use reliable methods of contraception
排除标准
- •Clinically significant medical conditions including active or uncontrolled infections, new or recurrent malignancy, serious cardiac, pulmonary, or renal disease, and uncontrolled diabetes.
- •Karnofsky Performance Score <
- •Immunosuppressant therapies other than allowed background therapy
- •Anti-arrhythmic and heart rate lowering drugs.
- •Any other circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol
研究组 & 干预措施
Ponesimod
Study treatment consists of 3 consecutive periods: 5 mg ponesimod treatment period (including up-titration), 10 mg treatment period (including up-titration) and a 20 mg treatment period.
干预措施: Ponesimod (Drug)
结局指标
主要结局
Change in Peripheral Absolute Lymphocyte Count From Baseline to Week 4, 8 and 12
时间窗: From baseline to Week 12
The primary pharmacodynamic endpoint assesses intra-subject dose response during the first 12 weeks of treatment.
次要结局
- Incident Rate of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)(From the first study drug intake up to 30 days after last study drug intake (Week 24))