A Single-arm, Open-label, Multi-center, Phase I/II Study Evaluating the Safety and Clinical Activity of QEL-001, an Autologous CAR T Regulatory Cell Treatment Targeting HLA-A2, in HLA-A2/ A28neg Patients That Have Received an HLA-A2pos Liver Transplant.
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 33
- 试验地点
- 10
- 主要终点
- Safety and Tolerability
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability of QEL-001 in the prevention of liver transplant rejection following immunosuppression withdrawal. QEL-001 is a product made from a patients own cells, which are genetically modified and designed to help the transplant recipient's body accept their donated liver and prevent their immune system from rejecting it once immune suppression is withdrawn.
详细描述
This study is a multicenter, first-in-human, open-label, single-arm study of an autologous CAR T regulatory (CAR-Treg) in HLA-A2 mismatched liver transplant recipients. The aim is for the CAR-Tregs to be activated on recognition of HLA-A2 antigens present on the donated liver and subsequently induce and maintain immunological tolerance to the organ.
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 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent.
- •Subject who are HLA A2/A28 negative who have received HLA A2-mismatch liver transplant 12 months to 5 years prior to study entry.
- •Able and willing to use contraception.
- •Be on stable maintenance of immunosuppression for at least 12 weeks prior to study entry.
排除标准
- •Severe cardiac, respiratory disease or any other major organ dysfunction.
- •Subjects with prior non-liver solid organ or hematopoietic stem cell transplant.
- •Known hypersensitivity to study medication ingredients, protocol defined immunosuppressive medications, or a significant allergic reaction to any drug.
- •Positive serology for human immunodeficiency virus (HIV), active or latent tuberculosis (TB) or other clinically active local or systemic infection.
- •Use of investigational agents within 3 months of screening.
- •Subjects with history of autoimmune disease requiring use of immunosuppression or biologics within 24 months prior to study entry.
- •Subject with history of malignancy in the past 5 years.
- •Medical or social condition that is not compatible with adequate study follow-up and any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study.
- •Protocol defined laboratory value for the following parameters:
- •Alanine aminotransferase (ALT) and either alkaline phosphatase (ALP) or gamma-glutamyl transferase (GGT),
- •Kidney function e.g. eGFR,
- •White blood cells,
- •Hemoglobin,
- •Platelets.
研究组 & 干预措施
Treatment group
干预措施: QEL-001 (Drug)
结局指标
主要结局
Safety and Tolerability
时间窗: 28 Days post infusion
Incidence of protocol defined Dose Limiting Toxicities (DLTs).
Long-term safety
时间窗: Day of infusion through to Week 82 and up to 15 years post infusion
Incidence and grade of treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs) according to CTCAE V5.0.
次要结局
- Immunosuppression related outcome(2 months and 1 year post withdrawal of immune suppression)
- Tolerance related outcome(1 year following immune suppression withdrawal)
- Composite efficacy failure outcome(1 year following immune suppression withdrawal)
研究者
Head of Regulatory Affairs
Scientific
Quell Therapeutics Limited