An Open-Label, First-in-Human Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With Type 1 Diabetes Mellitus (T1D)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 4
- 主要终点
- Incidence of adverse events with causality related to VCTX211 units, the surgical procedures and/or medical interventions required to implant and explant the VCTX211 units.
研究概览
简要总结
This is an open-label, multicenter, Phase 1/2 study evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects with T1D
详细描述
VCTX211 combination product (unit) compromises 2 components: (1) allogeneic pancreatic endoderm cells (PEC211) genetically modified using Cluster 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 PEC211 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 diabetes 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
结局指标
主要结局
Incidence of adverse events with causality related to VCTX211 units, the surgical procedures and/or medical interventions required to implant and explant the VCTX211 units.
时间窗: From implantation up to 12 months post implantation
Assess the clinical efficacy of VCTX211 units via evaluation of C-peptide increase from the baseline.
时间窗: From implantation up to 12 months post implantation
次要结局
- Assess the clinical efficacy of VCTX211 units via evaluation of changes in number of hypoglycemic evens from baseline.(From implantation up to 12 months post implantation)
- The percentage of viable graft cells per unit using immunohistochemical staining.(From implantation up to 12 months post implantation)
- Incidence of adverse events reported in patients implanted with VCTX211 units.(From implantation up to 12 months post implantation)
- Assess the clinical efficacy of VCTX211 units via evaluation of changes in exogenous insulin use from baseline.(From implantation up to 12 months post implantation)
- Assess the clinical efficacy of VCTX211 units via evaluation of changes in hemoglobin A1C levels from baseline.(From implantation up to 12 months post implantation)
- Assess the clinical efficacy of VCTX211 units via evaluation of percentage of time in pre-defined glycemic ranges, as measured by a continuous glucose monitor, from baseline.(From implantation up to 12 months post implantation)
- Qualitative evaluation of immune response to VCTX211 units assessed by histological staining for markers of host adaptive immune cells within the graft.(From implantation up to 12 months post implantation)
- Incidence of new alloreactive antibodies found in the blood of patients post implantation.(From implantation up to 12 months post implantation)
- Incidence of new autoreactive antibodies found in the blood of patients post implantation.(From implantation up to 12 months post implantation)
- The percentage of graft cells per unit that have differentiated into endocrine/beta cells as determined by immunohistochemical staining.(From implantation up to 12 months post implantation)