CK0802 for Treatment of Steroid Refractory Graft vs. Host Disease (GVHD)
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 12
- 主要终点
- Phase 1b
研究概览
简要总结
The purpose of this study is to determine the efficacy and safety of CK0802 Tregs for treatment of refractory GVHD (graft-versus-host disease)
详细描述
This clinical trial will be conducted in two parts where the first part of Phase 1b will determine the safety and tolerability of CK0802 in patients with steroid refractory GVHD and the second part of Phase 2a will examine the preliminary efficacy of CK0802 in patients with steroid refractory GVHD
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1. Age ≥12 years old (first 3 subjects will be ≥ 18 years)
- •2. Grade 2-4 acute GVHD (aGVHD) at baseline
- •3. No other prior aGVHD treatment
- •4. Steroid Refractory
- •Progressed after 3 days of treatment with 2 mg/kg methylprednisolone (MPE)
- •No improvement after 7 days of treatment with 2 mg/kg MPE
- •Progressed to a new organ after treatment with 1 mg/kg MPE for skin or upper gastrointestinal (UGI) aGVHD
- •Progressed from nadir during or after a steroid taper
- •5. Female subjects of childbearing potential (FPCP) must have a negative urine or serum pregnancy test.
- •6. Agreed to abide by all protocol required procedures including study-related assessments, visits and long term follow up.
- •7. Willing and able to provide written informed consent.
- •8. Signed informed consent
排除标准
- •1. 30 days prior treatment with any other investigational agent after allogeneic HSCT.
- •2. Known HIV seropositivity.
- •3. Uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy. The Protocol PI is the final arbiter of eligibility.
- •4. Subjects with uncontrolled inter-current illness that in the opinion of the investigator would place the subject at greater risk of severe toxicity and/or impair the activity of CK
- •5. Subjects who are pregnant or breastfeeding.
研究组 & 干预措施
Treatment Arm
CK0802 Tregs will be administered intravenously at a fixed dose of 100 million Treg cells.
干预措施: CK0802 (Allogeneic, Cryopreserved, Cord blood-derived T-regulatory cells, cells that are not HLA matched) (Biological)
结局指标
主要结局
Phase 1b
时间窗: Day +29
Dose limiting toxicities of CK0802 in SR GVHD as assessed by the incidence and severity of AE and SAEs determined by the NCI-CTCAE Version 6.0. Non-Hematologic: Any ≥ Grade 3 non-hematologic AE as per CTCAE v6.0 Hematologic: Any new or worsened Grade 4 neutropenia, thrombocytopenia, or anemia compared with baseline that persists for \>7 days despite supportive care and is not clearly and incontrovertibly attributable to the underlying disease or another extraneous cause. Death: Death within 28 days that is not clearly and incontrovertibly attributable to the underlying disease or another extraneous cause
Phase 2a
时间窗: D +29
Overall Response (OR) at day +29. Overall response (OR) will include complete response (CR) and partial response (PR). Response to be determined from the maximum acute GVHD stage and grade in each organ. Complete Response (CR): Stage 0 in all organs (skin, liver, and GI tract) and no intervening additional therapy Partial Response (PR): Improvement of at least 1 stage in 1 or more organs without progression in other organs, and no intervening additional therapy Overall Response (OR): Includes both CR and PR Very Good PR (VGPR): Improvement by at least one stage in one or more organs and * Skin: No rash, or bullae, and residual erythematous rash involving \<25% of the body surface * Liver: Total serum bilirubin concentration of \<2 mg/dL or \<25% of baseline at enrollment * Gut: Tolerating food or enteral feeding, predominantly formed stools, no overt GI bleeding or abdominal cramping, no more than occasional nausea or vomiting * No intervening additional therapy
次要结局
未报告次要终点