Bone Marrow vs Liver as Site for Islet Transplantation in Patients With Type 1 Diabetes: Pilot Study to Evaluate Efficacy
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 9
- 试验地点
- 2
- 主要终点
- Insulin secretion under stimulation
研究概览
简要总结
The goal of this study is to evaluate safety and efficacy of bone marrow (BM) as site for pancreatic islet transplantationin humans. Our hypothesis is that BM represents a better site than liver (currently the location of choice for this procedure) thanks to its potential capacity to favor islet engraftment. To address our hypothesis we propose herein a randomized phase II trial to compare BM and liver as sites for islet transplantation in T1D patients.
详细描述
The study is a phase II, single center, open label, pilot study. We will recruit 12 patients with T1D to be randomly (1:1) assigned to receive islet either into the liver through the portal venous circulation (standard procedure; arm A, n=6) or directly into the BM at the level of the iliac crest (arm B, n=6). Patients will be selected from those eligible for islet Tx based on local practice and guidelines.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diabetic patients eligible for pancreatic islet transplantation based on local accepted practice and guidelines. This includes at least: a)clinical history compatible with T1D with insulin-dependence for >5 years; b) undetectable stimulated (arginine or MMTT) C-peptide levels (<0.3 ng/mL) in the 12 months before transplant c)presence of severe hypoglycaemic events
排除标准
- •presence of hematologic disease
结局指标
主要结局
Insulin secretion under stimulation
时间窗: month 12 post-Tx
basal and -10 to 120 min time course of glucose, C-pep levels and insulin derived from the mixed meal tolerance test
次要结局
- Islet function(month 1, 3, 6, 9, 12 post-Tx)
- Incidence and severity of Adverse Events (AE) and Serious Adverse Events (SAE)(throughout the study up to 1 year after first transplant)
- Insulin requirement(month 1, 3, 6, 9, 12 post- transplant)
研究者
Piemonti Lorenzo
Head of Beta Cell Biology Unit, CO-Director Human Islet Transplantation Program (ITP)
Ospedale San Raffaele