Phase 1/ Phase 2 Study of the Therapeutic Effect of Ex-vivo Expanded Umbilical Cord Blood Regulatory T Cells on Autoimmune Diabetes
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Number of Participants with Adverse events as a Measure of Safety and Tolerability
研究概览
简要总结
The purpose of this study is to investigate the safety and therapeutic effect of ex-vivo expanded umbilical cord blood regulatory T cells on autoimmune diabetes.
详细描述
The regulatory T cells (Treg) used in this study will be produced from human umbilical cord blood. The umbilical cord blood will be freshly collected and immediately separated to buffy coat in a GMP laboratory, thus to be further sorted into Treg and other subsets. Then the Treg will be expanded ex-vivo for approximately 14 days until tested for its surface markers and suppressive ability. During the time of ex-vivo expansion, eligible autoimmune diabetes patients will be recruited and admitted to our hospital in order to go through required examinations. Once the Treg product has reached the safety and efficacy standards, the patient will receive the Treg product infusion under close surveillance of doctors and then go through regular follow-ups to investigate the safety and therapeutic effect of the Treg product on autoimmune diabetes patients.
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
- 6 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Autoimmune diabetes patients are screened for enrollment in the study if both clinical signs and laboratory tests meet the diagnosis standards of American Diabetes Association
- •Diagnosis of Autoimmune Diabetes within 3 years of screening
- •Between 6 to 60 years of age
- •Positive for at least one of the anti-islet autoantibodies: GADA, IA2A, ZnT8A
- •Fasting or postprandial plasma C-peptide more than 200 pmol/L
- •Written informed consent from the patient or the patient's parents for patients under the age of 18 years
排除标准
- •Any clinically significant diseases in liver (ALT and AST over 2 times upper normal limit), kidney (Scr over 133umol/L), and heart
- •Presence of anemia (Hb ≤100g/L), leukopenia (<3.5×10^9/L)
- •Presence of disorder in coagulation or anticoagulation, or thrombocytopenia (platelets <100×10^9/L)
- •Presence of acute metabolic disorders; In the case of acute ketone acidosis, with blood ketone over 0.3mmol/L and pH lower than 7.30
- •Presence of any kind of chronic infection or immune deficiency, including hepatitis B, hepatitis C, HIV, syphilis or tuberculosis
- •Chronic use of systemic glucocorticoids or other immunosuppressive agents for over 3 months
- •Any history of malignancy
- •Female patients who are pregnant or breastfeeding; any female who is unwilling to use a reliable and effective form of contraception for 2 years afer recruitment
- •Presence of any infectious diseases, including active skin infections, flu, fever, upper or lower respiratory track infections; those who wish to participate in the study should keep the infection under control for at least 1 week before receiving Treg product infusion
- •Presence of diabetic microvascular or macrovascular diseases
- •Presence of hypertension
- •Any medical condition that, in the opinion of the investigator, will interfere with safe participation in the trial
研究组 & 干预措施
Treg Treatment + Insulin
subjects will be treated with Umbilical Cord Blood Regulatory T cells Therapy and insulin according to routine clinical practice at the discretion of the treating physician
干预措施: Umbilical Cord Blood Regulatory T cells Therapy (Biological)
Treg Treatment + Insulin
subjects will be treated with Umbilical Cord Blood Regulatory T cells Therapy and insulin according to routine clinical practice at the discretion of the treating physician
干预措施: Insulin (Drug)
Insulin
subjects will be treated with insulin according to routine clinical practice at the discretion of the treating physician
干预措施: Insulin (Drug)
结局指标
主要结局
Number of Participants with Adverse events as a Measure of Safety and Tolerability
时间窗: 2 years
The number of participants with adverse events, laboratory abnormalities and other signs of toxicity. Particular focus will be on the number and severity of infusion reactions, complications related to infection, and any potential negative impact on the course of diabetes.
次要结局
- C-peptide(2 years)
- Autoimmune Status(2 years)
- Insulin requirement(2 years)
- HbA1c(2 years)
- Blood glucose(2 years)
研究者
Zhiguang Zhou
MD/PhD
Second Xiangya Hospital of Central South University