Transfusion of Autologous Umbilical Cord Blood Plus Vitamin D and Omega 3 Fatty Acids to Preserve Beta Cell Function in Children With Recent Onset Type 1 Diabetes - A Pilot Study
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- C-Peptide following the 1 year mixed meal tolerance test
研究概览
简要总结
In this pilot study the investigators are trying to see if a single intravenous infusion of autologous (self) cord blood cells followed by 1 year of daily vitamin D and omega 3 fatty acid supplementation can preserve beta cell function (prolong "honeymoon") in children with type 1 diabetes. All subjects will continue to use insulin therapy as needed to maintain the best possible glucose control.
15 Subjects will be randomized such that 2 of every 3 (10 total) will receive cord blood plus vitamin D and Omega 3 while 1 of 3 (5 total) will serve as controls and will not receive cord blood, vitamin D, or Omega 3 supplementation.
The study will involve 5 visits over 1 year to the University of Florida
This study is a follow-up to our initial study of cord blood infusion alone in which 23 children received autologous cord blood. The initial study was 100% safe but additional studies like the one described above are needed to determine how to improve cord blood based therapy.
详细描述
Hypothesis: We hypothesize that the combination of intensive insulin therapy, autologous umbilical cord blood (UCB), Vitamin D, and Omega 3 fatty acids administered to children with T1D will preserve residual c-peptide when compared to children receiving intensive insulin therapy alone
Specific Aims:
- Randomize 15 children with recent onset T1D and available autologous cord blood such that 10 receive a combination of intensive insulin therapy, autologous UCB infusion, and daily Vitamin D while 5 receive intensive insulin therapy alone
- Document safety of combination therapy
- Study potential changes in glucose metabolism
- Study potential changes in immune function
Preliminary Studies: Our group has already performed autologous cord blood infusion in 23 children with T1D and has documented the safety of this approach. While conclusive data regarding the efficacy of the infusion in preserving beta cell function are lacking, our pilot study has demonstrated a potential for cord blood to low the rate of c-peptide decline (a measure of beta cell function/mass) in these young children with T1D. As documented above, considerable work both here and other institutions suggests the potential for a combination of Vitamin D and DHA to further augment the autoimmune response. Given the safety and potential efficacy of such an approach, we feel that a pilot study of the combination of UCB infusion, vitamin D supplementation, and DHA supplementation is warranted.
Screening: Only subjects who are still making at least a small amount of detectable insulin will be eligible for this study. To determine if a child is still making insulin, they will undergo a mixed meal tolerance test. This involves placing an IV in the morning prior to eating. The subject then drinks a "mixed meal" (the nutritional supplement Boost is used)and blood is taken via the IV at timepoints over the next 2 hours to determine if insulin is still being produced.
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
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •TID diagnosis confirmed by presence of at least 1 diabetes autoantibody Children ≥ 1 years
- •Stored autologous umbilical cord blood (15 sought) in an AABB and/or FACT accredited cord bank.
- •Stimulated C-peptide > 0.2pmol/L on MMTT
- •Cord blood meets all selection and testing criteria (see below).
- •Normal screening values for CBC, Renal function and electrolytes (BMP with Ca, Mg, and Phos).
- •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 mother's 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).
- •Allergy to DHA (Omega 3) or Vitamin D
- •Hypercalcemia
研究组 & 干预措施
Autologous UCB Plus Vit D Omega 3 FA
A single autologous (self) intravenous umbilical cord blood infusion followed by 1 year of daily Vitamin D and Omega 3 Fatty Acid supplementation give as liquid drops and gel capsules that can be swallowed or added to food
干预措施: Autologous UCB (Biological)
Autologous UCB Plus Vit D Omega 3 FA
A single autologous (self) intravenous umbilical cord blood infusion followed by 1 year of daily Vitamin D and Omega 3 Fatty Acid supplementation give as liquid drops and gel capsules that can be swallowed or added to food
干预措施: Omega 3 FA (Dietary Supplement)
Control
Subjects randomized to be controls will continue to use intensive insulin therapy in order to compare c-peptide production at 1 year in those receiving combination therapy vs those who do not
Autologous UCB Plus Vit D Omega 3 FA
A single autologous (self) intravenous umbilical cord blood infusion followed by 1 year of daily Vitamin D and Omega 3 Fatty Acid supplementation give as liquid drops and gel capsules that can be swallowed or added to food
干预措施: Vitamin D (Dietary Supplement)
结局指标
主要结局
C-Peptide following the 1 year mixed meal tolerance test
时间窗: 1 year post cord blood infusion
次要结局
- DHA Level(1 year post randomization)
- Vitamin D Level(1 year post randomization)
- HbA1c and Insulin Dose(1 year post randomization)
- Peripheral Blood T-cell assays(1 year post randomization)