An Open-Label, First-In-Human Study Evaluating the Safety and Tolerability of VCTX210A Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 7
- 试验地点
- 3
- 主要终点
- Incidence of adverse events with causality related to VCTX210A units and/or the surgical procedures required to implant and explant the VCTX210A units.
研究概览
简要总结
This is an open-label, multicenter, Phase 1 study evaluating the safety and tolerability of VCTX210A combination product in patients with T1D
详细描述
VCTX210A combination product (unit) comprises 2 components: (1) allogeneic pancreatic endoderm cells (PEC210A) genetically modified using Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated protein 9 (CRISPR/Cas9) to promote immune evasiveness and survival, and (2) a durable, removable, perforated device designed to deliver and retain the PEC210A cells.
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 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of T1D for a minimum of 5 years
- •Stable, optimized diabetic regimen for at least 3 months prior to enrollment
排除标准
- •Medical history of islet cell, kidney, and/or pancreas transplant
- •Occurrence of 2 or more severe, unexplained hypoglycemic events within 6 months prior to enrollment
- •Known causes of diabetes other than T1D
- •Immunosuppressant therapy in the previous 30 days and/or requirements for chronic immunosuppressive therapy during the study
- •Prior treatment with gene therapy or edited product
研究组 & 干预措施
VCTX210A combination product
Up to seven (7) units will be implanted
干预措施: VCTX210A unit (Combination Product)
结局指标
主要结局
Incidence of adverse events with causality related to VCTX210A units and/or the surgical procedures required to implant and explant the VCTX210A units.
时间窗: From implantation up to 6 months post implantation
次要结局
- The percentage of graft cells per unit that have differentiated into endocrine/beta cells as determined by immunohistochemical staining.(From implantation up to 6 months post implantation)
- Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host innate immune cells within the graft.(From implantation up to 6 months post implantation)
- Qualitative evaluation of immune response to VCTX210A units assessed by histological staining for markers of host adaptive immune cells within the graft.(From implantation up to 6 months post implantation)
- Incidence of new alloreactive antibodies found in the blood of patients post implantation.(From implantation up to 6 months post implantation)
- Incidence of new autoreactive antibodies found in the blood of patients post implantation.(From implantation up to 6 months post implantation)
- The percentage of viable graft cells per unit using immunohistochemical staining.(From implantation up to 6 months post implantation)