Transfusion of Autologous Umbilical Cord Blood to Reverse Hyperglycemia in Children With Type 1 Diabetes - A Pilot Study
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 23
- 试验地点
- 2
- 主要终点
- Children With T1D Underwent a Single Autologous UCB Transfusion
研究概览
简要总结
While this study is now completely enrolled, we do hope to develop a "next generation" cord blood based study sometime in early 2009. Please continue to contact us if you have a child with newly diagnosed Type 1 Diabetes (T1D) who alo has their OWN cord blood in storage.
详细描述
Objective:
Our goal is to transfuse autologous umbilical cord blood into 23 children with T1D in an attempt to re-establish immune tolerance and perhaps regenerate pancreatic islet insulin-producing beta cells and improve blood glucose control. As secondary goals, we aim to study the potential changes in metabolism/immune function leading to islet regeneration.
Background/Rationale:
Stem cells provide an exciting approach towards curing T1D. Autologous bone marrow transplants have been used successfully for patients undergoing high dose chemotherapy, and for a variety of cancers and autoimmune disorders such as multiple sclerosis, lupus, and rheumatic disorders. Recent studies in immunodeficient mice with chemically-induced pancreatic damage have shown that bone marrow-derived stem cells preferentially home to the pancreas and may have the capacity to initiate pancreatic regeneration, thereby restoring the endothelial interactions in the pancreas and correcting the associated elevated blood sugar levels Human umbilical cord blood cells transfused into a model of amyotrophic lateral sclerosis (ALS) resulted in delayed disease progression of two to three weeks and increased lifespan. Umbilical cord blood has shown promise as an excellent source for deriving stem cell populations, and has been used successfully in transplantation for a variety of diseases, including acute lymphocytic and myeloid leukemia, lymphoma, Fanconi anemia, and sickle cell disease. Furthermore, umbilical cord blood-derived stem cells have the capacity to differentiate into a variety of non-blood cell types, including hepatocytes, neural cells, and endothelial cells. In addition, umbilical cord blood contains a greater proportion of hematopoietic stem cells than bone marrow.
In addition, cord blood contains a large number of immune cells called regulatory T cells, These regulatory T cells may be helpful in diminishing autoimmunity. The need to re-establish tolerance in patients with established autoimmunity provides another potential mechanism for cord blood as a therapy for type 1 diabetes.
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
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must have a diagnosis of T1D and have stored umbilical cord blood in an AABB and/or FACT accredited cord bank.
- •TID diagnosis will be defined as having a clear history of polydipsia, polyphagia, polyuria, and weight loss consistent with a clinical diagnosis, diagnosis will mot be based solely upon the presence of autoantibodies.
- •Cord blood meets all selection and testing criteria (see below).
- •Able to complete mixed meal tolerance / glucagon stimulation test.
- •Normal screening values for CBC, Renal function and electrolytes (BMP).
- •Willing to comply with intensive diabetes management
排除标准
- •Complicating medical issues that would interfere with blood drawing or monitoring.
- •Chronic use of steroids or other immunosuppressive agents for other conditions.
- •Positive infectious disease markers from mothers' blood or cord at time of collection (See below for details).
- •Any evidence of illness on planned infusion date (i.e. fever >38.5 C, vomiting, diarrhea, wheezing, or crackles).
结局指标
主要结局
Children With T1D Underwent a Single Autologous UCB Transfusion
时间窗: Baseline to Year 2
All participants were monitored for 2 years. Baseline and post-infusion mixed meal tolerance tests were performed to determine whether autologous cord blood infusion preserved endogenous insulin production. The change in median area under the curve for C-peptide (measure of insulin production) from baseline to to 2 years during a 2 hour mixed meal tolerance test was used as the primary outcome measure and was reported in ng/ml/120 minutes
次要结局
未报告次要终点