An Open-Label Phase 1 Study to Investigate the Safety and Efficacy of CYP-001 for the Treatment of Adults With Steroid-Resistant Acute Graft Versus Host Disease
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 7
- 主要终点
- Incidence and severity of treatment emergent adverse events [safety and tolerability]
研究概览
简要总结
The purpose of this study is to assess the safety, tolerability and efficacy of two infusions of CYP-001 in adults with steroid-resistant GvHD.
详细描述
This is a multi-centre, open label, dose escalation study to assess the safety, tolerability and efficacy of two infusions of CYP-001, in adults who have steroid-resistant GvHD.
Participants will receive standard of care treatment throughout the study, according to local procedures. The first eight participants will be enrolled in Cohort A and receive a CYP-001 dose of 1 million cells per kg, up to a maximum dose of 100 million cells, on Day 0 and Day 7. Subject to a safety review of data from Cohort A, an additional eight participants will be enrolled into Cohort B and receive a CYP-001 dose of 2 million cells/kg, up to a maximum dose of 200 million cells, on Day 0 and Day 7. The primary evaluation period concludes for each participant 100 days after the first dose of CYP-001. Participants will have study visits on Days 0, 3, 7, 14, 21, 28, 60 and 100. Subsequently, participants will enter a long term follow-up period, which concludes 2 years after the first dose of CYP-001.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 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
- •Diagnosis using consensus grading with steroid-resistant Grade II-IV acute GvHD, after a haematopoietic stem cell transplant for a haematological disorder.
- •Life expectancy of at least one month.
- •Agree to have follow-up data collected for two years after their initial dose of CYP-001 (under a separate protocol).
排除标准
- •Pregnant or breastfeeding or plan to become pregnant within three months of receiving their last dose of CYP-
- •Have received any investigational research agent within 30 days or five half-lives (whichever is longer) prior to the first dose of IMP.
- •Known or suspected current alcohol or substance abuse problem.
- •Progressive or relapsing haematological malignancy, a current solid tumour, or previous malignant solid tumour that is likely to recur during the period of the study (with the exception of a past history of basal or squamous cell carcinomas).
- •Heart failure (NYHA Functional Class II-IV) and/or pulmonary failure.
- •Haemodynamically unstable and/or at high risk of cardiovascular events.
- •Terminal organ failure.
- •Meningitis, pneumonia with hypoxemia, HIV or another severe or uncontrolled systemic infection, which in the opinion of the investigator is likely to impact on the ability of the patient to participate in the trial.
结局指标
主要结局
Incidence and severity of treatment emergent adverse events [safety and tolerability]
时间窗: 28 days
Safety
Incidence and severity of serious adverse events deemed possibly related to CYP-001 [safety and tolerability]
时间窗: 100 days
Safety
次要结局
- Overall Survival at Day 28(28 days)
- Partial Response by Day 100(100 days)
- Complete Response by Day 28(28 days)
- Partial Response by Day 28(28 days)
- Complete Response by Day 100(100 days)
- Overall Survival at Day 100(100 days)