Investigation of Dose Escalation and Cohort Expansion Study on the Safety and Efficacy of Allogeneic Umbilical Cord Mesenchymal Stem Cells(UCMSCs) Combined With Standard Therapy for Newly Diagnosed Type 1 Diabetes
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Safety assessment
研究概览
简要总结
The aim is to investigate the safety and tolerability of intravenous infusion of allogeneic umbilical cord mesenchymal stem cells in pediatric patients diagnosed with newly onset type 1 diabetes
详细描述
This is an IIT study, where the first part is an open, dose escalating study consisting of 9 patients, 8-18 years of age. The second part is a randomized, double-blinded, placebo-controlled, IIT study in parallel design comparing allogeneic UCMSCs treatment to placebo in pediatric patients diagnosed with newly onset type 1 diabetes. Besides safety, preservation of endogenous insulin production (measured as C-peptide concentrations) together with metabolic control, diabetes treatment satisfaction and immunological profile will be assessed.
A total number of 24 patients will be enrolled in the study and followed for one year after UCMSCs/placebo treatment. Patients 8-18 years of age, both male and female, diagnosed for type 1 diabetes will be eligible. Providing informed consent and fulfillment of inclusion criteria and no exclusion criteria, they will within 6 months of diagnosis be randomized.
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
- 8 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Meets the diagnostic criteria for diabetes according to the World Health Organization (WHO) guidelines from
- •Presence of at least one pancreatic autoantibody, or idiopathic type 1 diabetes mellitus (T1DM) with negative autoantibody testing.
- •Ages 8 to 18 years, regardless of gender.
- •Newly diagnosed T1DM within the past 6 months.
- •Fasting C-peptide ≥0.1 nmol/L and postprandial 2-hour C-peptide >0.2 nmol/L.
- •Voluntary acceptance of stem cell transplantation therapy by the individual, their family members, or legal guardians, and signing of an informed consent form.
排除标准
- •Diabetic ketoacidosis is not under control.
- •Severe allergic constitution.
- •BMI <14 or >
- •History of other autoimmune diseases, hematologic disorders.
- •HIV positive, carrier of viral hepatitis, active phase of viral hepatitis, or other uncontrolled infectious diseases.
- •History of acute pancreatitis, pulmonary embolism, or other thrombotic diseases, as well as severe diseases of the heart, liver, kidneys, respiratory system, nervous system, etc.
- •Suffering from gestational diabetes, monogenic diabetes, diabetes caused by pancreatic injury, or other secondary diabetes (such as Cushing's syndrome, thyroid dysfunction, or acromegaly-induced diabetes);
- •Pregnancy or planning pregnancy within 3 months before or after treatment, as well as breastfeeding women.
- •Mental illness, alcohol or drug abuse, inability to comply with treatment.
- •Known or suspected tumors.
- •According to the investigator's judgment, there are other clinical conditions that may endanger the safety of the subjects.
研究组 & 干预措施
Experimental
干预措施: UCMSCs (Drug)
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Safety assessment
时间窗: Throughout the study until Week 52
Changes in safety parameters of patients during the study period since baseline. Measured through the registration of adverse events and other safety parameters.
Efficacy Assessment
时间窗: Week 52
Delta-change of C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at Week 52 following UCMSCs/Placebo infusion when compared to test performed before start of treatment.
次要结局
- Efficacy Assessment: HbA1c(Week 52)
- Efficacy Assessment: Dose of exogenous insulin(Week 52)