Cellular Therapy for Type 1 Diabetes Using Mesenchymal Stem Cells
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 60
- 试验地点
- 2
- 主要终点
- 12 month Change in C-peptide area under the curve after a 2-hour MMTT
研究概览
简要总结
The goal of this study is to determine the safety and efficacy of fresh metabolically active allogeneic umbilical cord-derived mesenchymal stromal cells (UC-MSCs) for the treatment of new-onset type 1 diabetes (T1D) and to understand the mechanisms of protection. If proven effective, such a strategy can be used as a therapeutic option for T1D patients and potentially other autoimmune disorders.
详细描述
This study seeks to find and enroll participants between the ages of 18 to 40 with new onset Type 1 diabetes (T1D) within 6 months of the first dose of insulin. T1D is an autoimmune disease in which T cells attack and destroy insulin-secreting pancreatic β cells leading to insulin deficiency and hyperglycemia in patients. Life-long insulin therapy is the major treatment option. However, insulin therapy is not a cure and a safer and more effective therapy is needed.
Mesenchymal Stromal Cells (MSCs) have emerged as a novel biopharmaceutical approach for many disorders. MSCs are a cellular product that can be derived from a patient's own body (autologous) or from a donor (allogeneic). This study will obtain MSCs from umbilical cords at the time of delivery from normal women who have been extensively screened for infectious diseases. These cells produced at the MUSC Center for Cellular therapy will be used within 3 passages after collection.
Evidence from animal models and clinical trials suggests that MSC infusion suppresses autoimmune and inflammatory diseases such as T1D. One clear message from these trials is that MSCs are effective at suppressing autoimmunity and seem generally safe. This study will measure safety and efficacy of MSCs over the course of 1 year.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 40 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A new diagnosis of T1D based on the ADA criteria within 6 months of randomization.
- •Male and female between the ages of 18 and 40
- •Mentally stable and able to comply with the procedures of the study protocol
- •Positivity for at least one T1D-associated autoantibody, such as GAD, IA-2 or ZnT8 autoantibodies
- •At screening, patients must have residual β cell function with a stimulated peak C-peptide >0.2 nmol/l during a 2 hour MMTT
- •Must be willing to comply with "intensive diabetes management" (* See diabetes management at MUSC below) as directed by the participant's clinician with the goal of maintaining blood glucose as close to normal as possible
- •Subject must be willing to comply with the schedule of study visits and protocol requirements
- •Subject with normal laboratory values of: White blood cell counts: between 4,500 to 11,000 per microliter; Platelet counts: 140,000 to 450,000 platelets per microliter of blood; Serum creatinine range is 0.6-1.3 mg/dL, Hepatic function: ALT 5 to 55 units per liter (U/L), AST 5 to 48 U/L.
排除标准
- •Evidence of retinopathy at baseline based on ophthalmologic examination or medical record review.
- •Body Mass Index < 14 or >35
- •Presence of malignancy
- •Subject has abnormally high lipid levels that exceeds > 3 times the upper limit of normal for LDL cholesterol or triglycerides
- •Subject has blood pressure greater than 160 mmHg systolic or 100 mmHg diastolic at time of consent
- •Subject is being treated for severe active infection of any type
- •A female subject who is breast-feeding, pregnant, or intends to become pregnant during the study.
- •Subject with clinically relevant uncontrolled medical condition not associated with diabetes (e.g. severe psychiatric, hematologic, renal, hepatic, neurologic, cardiac, or respiratory disorder)
- •Subjects with HgbA1c >12%, and/or fasting blood glucose >270 mg/dL and/or frequent episodes of hypoglycemia (>2 episodes per week of blood glucose levels <60 mg/dL).
研究组 & 干预措施
Group A Treatment
2.5 x 10^6 MSC per kg will be infused intravenously on Day 1
干预措施: Mesenchymal Stem Cells (MSCs) (Biological)
Group B Placebo
Plasmalyte with 0.5% Human Serum Albumin will be infused intravenously on Day 1
干预措施: Placebo Infusion (Plasmalyte A with 0.5% human serum albumin) (Other)
结局指标
主要结局
12 month Change in C-peptide area under the curve after a 2-hour MMTT
时间窗: 1 year (plus or minus 30 days) after infusion
Change in beta cell function
次要结局
- 6 Month Change in C-Peptide area under the curve after a 2-hour MMTT(6 months (plus or minus 14 days) after infusion)
- 6 Month peak C-peptide after a 2-hour MMTT(6 months (plus or minus 14 days) after infusion)
- Change in 24-hour insulin dose per kilogram between baseline and 1 year measurements(1 year (plus or minus 30 days) after infusion)
- 1 year peak C-peptide after a 2-hour MMTT(1 year (plus or minus 30 days) after infusion)
研究者
Hongjun Wang
Professor
Medical University of South Carolina