A Phase 1 Open-Label Safety, Tolerability, and Early Efficacy Study of a Renal Autologous Cell Therapy (REACT) in Patients With Chronic Kidney Disease From Congenital Anomalies of the Kidney and Urinary Tract (CAKUT) (REGEN-004)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Prokidney
- 入组人数
- 6
- 试验地点
- 9
- 主要终点
- Assess change in eGFR and observe incidence of renal-specific procedure and/or product related adverse events (AEs) through 18 months following two Renal Autologous Cell Therapy (REACT) injections [Safety].
研究概览
简要总结
A phase 1, open-label safety, tolerability and early efficacy study of a Renal Autologous Cell Therapy (REACT) in patients with Chronic Kidney Disease from Congenital Anomalies of the Kidney and Urinary Tract (CAKUT) (REGEN-004)
详细描述
It is anticipated that all subjects will receive two planned Renal Autologous Cell Therapy (REACT) injections to allow dose-finding and evaluate the duration of effects. The scientific rationale, based on non-clinical studies, is that the biologically active component of REACT (homologous, autologous, SRC) delays progression of experimental models of Chronic Kidney Disease (CKD) by augmenting renal structure and function. As a result, the more cells that can be infused, the greater the potential improvement in renal function.
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
- •The patient is male or female, 18 to 65 years of age on the date of informed consent.
- •The patient has a documented history of a corrected abnormality of the kidney and/or urinary tract (CAKUT) leading to renal dysfunction.
- •The patient has an established diagnosis of Stage III/IV CKD not requiring renal dialysis.
- •The subject has blood pressure less than 150/90 at the Screening Visit, prior to renal biopsy, and prior to REACT injection(s).
排除标准
- •The patient has a history of renal transplantation.
- •The patient has a diagnosis of hydronephrosis, SFU Grade 4 or
- •The patient has an uncorrected VUR Grade
- •The patient has a diffuse cortical thickness throughout the kidney measuring < 5 mm on MRI that prevents safe cortical biopsy.
结局指标
主要结局
Assess change in eGFR and observe incidence of renal-specific procedure and/or product related adverse events (AEs) through 18 months following two Renal Autologous Cell Therapy (REACT) injections [Safety].
时间窗: 18 months following last REACT injection
The primary objective is to assess the safety and optimal delivery of Renal Autologous Cell Therapy (REACT) injected at one site in a recipient kidney as measured by procedure- and/or product related adverse events (AEs) through 18 months post-treatment.
次要结局
- Number of subjects with renal-specific adverse events over a 18-month period following injection of Renal Autologous Cell Therapy (REACT).(18 months following last REACT injection)