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临床试验/NCT03835312
NCT03835312招募中不适用

Sequential Transplantation of Umbilical Cord Blood Stem Cells and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency Type 1 Diabetes Mellitus

Children's Hospital of Fudan University1 个研究点 分布在 1 个国家目标入组 50 人开始时间: 2019年2月20日最近更新:
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适应症
干预措施

试验速览

阶段
不适用
状态
招募中
入组人数
50
试验地点
1
主要终点
Concentration of serum C-peptide
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

This study evaluates the efficacy of sequential transplantation of umbilical cord blood stem cells and islet cells in children with monogenic immunodeficiency type 1 diabetes mellitus. Umbilical cord blood stem cell transplantation will be performed first. Children with stable immune reconstruction will than receive islet cell transplantation.

详细描述

Monogenic immunodeficiency type 1 diabetes mellitus (T1DM) usually onsets in early age and has a long course of treatment. Because of T cell deficiency, patients are prone to recurrent infection, hemorrhage, sepsis, colitis or complications of diabetes mellitus, which lead to early death.

New clinical treatment schemes have been explored and introduced around the world. Sequential transplantation of umbilical cord blood stem cells and islet cells is the latest treatment method for these children. Early treatment of monogenic immunodeficiency T1DM children can avoid disease-related organ toxicity, infection risk associated with chronic immunosuppression, and possible prevention of autoimmune endocrine organ damage. Thus, sequential transplantation of umbilical cord blood stem cells and islet cells is the only possible cure for those patients currently.

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
— 至 18 Years（Child, Adult）
性别
All
接受健康志愿者
否

入选标准

  • •1.Type 1 diabetes mellitus children with genetic immunodeficiency
  • •Meet the diagnostic criteria of type 1 diabetes mellitus: clinical manifestations of typical diabetes mellitus include polyphagia, polyuria, weight loss, or diabetic ketoacidosis, confirmed by blood sugar level, islet function and autoimmune antibody.
  • •Existence of extrapancreatic organ damage: (1) inflammatory bowel disease, (2) impairment of renal function, (3) repeated infection of mouth, skin, anus or whole body, (4) immune hepatitis, (5) persistent chronic immune iridocyclitis, (6) immune adrenalinitis leading to adrenocortical dysfunction, (7) pituitary inflammation leading to hypophysis, (8) rheumatoid disease, (9) immune vasculitis, (10) systemic lupus erythematosus, (11) other organs besides thyroid function damage. Suffering from one or more of above diseases. Recurrence after receiving regular clinical treatment, including symptomatic treatment of organ protective drugs.
  • •Gene mutation was found according to gene diagnosis: gene mutation was found by gene sequencing. Literature searches at home and abroad confirmed that the defect of the gene resulted in autoimmune or immune dysfunction, resulting in multiple organ dysfunction and poor prognosis.

排除标准

  • •Mature and effective treatment methods are available.
  • •HIV, HBV and HCV were positive.
  • •A the active period of infection.
  • •At the active stage of malignant tumors.
  • •Combination of other fatal diseases.
  • •Existence of mental and psychological diseases.

研究组 & 干预措施

Interventional

Experimental

Sequential transplantation of umbilical cord blood stem cells and islet cells

干预措施: Sequential transplantation (Procedure)

结局指标

主要结局

Concentration of serum C-peptide

时间窗: from the completion of treatment to 3 years

Islet function (concentration of serum C-peptide)

Concentration of serum C-peptide

时间窗: from the completion of treatment to 3 months

Islet function (concentration of serum C-peptide)

次要结局

  • HbA1c level(from the completion of treatment to 12 months)
  • HbA1c level(from the completion of treatment to 15 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 36 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 36 months)
  • Body weight(from the completion of treatment to 36 months)
  • Concentration of serum insulin(from the completion of treatment to 36 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 36 months)
  • Concentraion of interleukin-2(from the completion of treatment to 36 months)
  • Body height(from the completion of treatment to 36 months)
  • Tanner stage(from the completion of treatment to 36 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 36 months)
  • Concentration of serum C-peptide(from the completion of treatment to 33 months)
  • Fast blood glucose level(from the completion of treatment to 36 months)
  • HbA1c level(from the completion of treatment to 36 months)
  • Fast blood glucose level(from the completion of treatment to 12 months)
  • Fast blood glucose level(from the completion of treatment to 15 months)
  • Fast blood glucose level(from the completion of treatment to 18 months)
  • Fast blood glucose level(from the completion of treatment to 21 months)
  • Fast blood glucose level(from the completion of treatment to 24 months)
  • Fast blood glucose level(from the completion of treatment to 27 months)
  • Fast blood glucose level(from the completion of treatment to 30 months)
  • Fast blood glucose level(from the completion of treatment to 33 months)
  • HbA1c level(from the completion of treatment to 3 months)
  • HbA1c level(from the completion of treatment to 6 months)
  • HbA1c level(from the completion of treatment to 9 months)
  • Concentration of serum C-peptide(from the completion of treatment to 6 months)
  • Concentration of serum C-peptide(from the completion of treatment to 9 months)
  • Concentration of serum C-peptide(from the completion of treatment to 12 months)
  • Concentration of serum C-peptide(from the completion of treatment to 15 months)
  • Concentration of serum C-peptide(from the completion of treatment to 18 months)
  • Concentration of serum C-peptide(from the completion of treatment to 21 months)
  • Concentration of serum C-peptide(from the completion of treatment to 24 months)
  • Concentration of serum C-peptide(from the completion of treatment to 27 months)
  • Concentration of serum C-peptide(from the completion of treatment to 30 months)
  • Concentration of serum insulin(from the completion of treatment to 3 months)
  • Concentration of serum insulin(from the completion of treatment to 6 months)
  • Concentration of serum insulin(from the completion of treatment to 9 months)
  • Concentration of serum insulin(from the completion of treatment to 12 months)
  • Concentration of serum insulin(from the completion of treatment to 15 months)
  • Concentration of serum insulin(from the completion of treatment to 18 months)
  • Concentration of serum insulin(from the completion of treatment to 21 months)
  • Concentration of serum insulin(from the completion of treatment to 24 months)
  • Concentration of serum insulin(from the completion of treatment to 27 months)
  • Concentration of serum insulin(from the completion of treatment to 30 months)
  • Concentration of serum insulin(from the completion of treatment to 33 months)
  • Fast blood glucose level(from the completion of treatment to 3 months)
  • Fast blood glucose level(from the completion of treatment to 6 months)
  • Fast blood glucose level(from the completion of treatment to 9 months)
  • HbA1c level(from the completion of treatment to 18 months)
  • HbA1c level(from the completion of treatment to 21 months)
  • HbA1c level(from the completion of treatment to 24 months)
  • HbA1c level(from the completion of treatment to 27 months)
  • HbA1c level(from the completion of treatment to 30 months)
  • HbA1c level(from the completion of treatment to 33 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 3 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 6 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 9 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 12 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 15 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 18 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 18 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 21 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 24 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 27 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 30 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 21 months)
  • occurrence of infection (number of infections)(from the completion of treatment to 33 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 3 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 6 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 9 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 12 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 15 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 24 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 27 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 30 months)
  • Concentration of blood immunoglobulin(from the completion of treatment to 33 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 3 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 6 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 9 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 12 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 15 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 18 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 21 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 24 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 27 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 30 months)
  • Concentration of T lymphocyte subsets(from the completion of treatment to 33 months)
  • Concentraion of interleukin-2(from the completion of treatment to 3 months)
  • Concentraion of interleukin-2(from the completion of treatment to 6 months)
  • Concentraion of interleukin-2(from the completion of treatment to 9 months)
  • Concentraion of interleukin-2(from the completion of treatment to 12 months)
  • Concentraion of interleukin-2(from the completion of treatment to 15 months)
  • Concentraion of interleukin-2(from the completion of treatment to 18 months)
  • Concentraion of interleukin-2(from the completion of treatment to 21 months)
  • Concentraion of interleukin-2(from the completion of treatment to 24 months)
  • Concentraion of interleukin-2(from the completion of treatment to 27 months)
  • Concentraion of interleukin-2(from the completion of treatment to 30 months)
  • Concentraion of interleukin-2(from the completion of treatment to 33 months)
  • Body height(from the completion of treatment to 3 months)
  • Body height(from the completion of treatment to 6 months)
  • Body height(from the completion of treatment to 9 months)
  • Body height(from the completion of treatment to 12 months)
  • Body height(from the completion of treatment to 15 months)
  • Body height(from the completion of treatment to 18 months)
  • Body height(from the completion of treatment to 21 months)
  • Body height(from the completion of treatment to 24 months)
  • Body height(from the completion of treatment to 27 months)
  • Body height(from the completion of treatment to 30 months)
  • Body height(from the completion of treatment to 33 months)
  • Body weight(from the completion of treatment to 3 months)
  • Body weight(from the completion of treatment to 6 months)
  • Body weight(from the completion of treatment to 9 months)
  • Body weight(from the completion of treatment to 12 months)
  • Body weight(from the completion of treatment to 15 months)
  • Body weight(from the completion of treatment to 18 months)
  • Body weight(from the completion of treatment to 21 months)
  • Body weight(from the completion of treatment to 24 months)
  • Body weight(from the completion of treatment to 27 months)
  • Body weight(from the completion of treatment to 30 months)
  • Body weight(from the completion of treatment to 33 months)
  • Tanner stage(from the completion of treatment to 3 months)
  • Tanner stage(from the completion of treatment to 6 months)
  • Tanner stage(from the completion of treatment to 9 months)
  • Tanner stage(from the completion of treatment to 12 months)
  • Tanner stage(from the completion of treatment to 15 months)
  • Tanner stage(from the completion of treatment to 18 months)
  • Tanner stage(from the completion of treatment to 21 months)
  • Tanner stage(from the completion of treatment to 24 months)
  • Tanner stage(from the completion of treatment to 27 months)
  • Tanner stage(from the completion of treatment to 30 months)
  • Tanner stage(from the completion of treatment to 33 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 3 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 6 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 9 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 12 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 15 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 18 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 21 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 24 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 27 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 30 months)
  • Occurrence of graft versus host disease(from the completion of treatment to 33 months)

研究者

申办方类型
Other
责任方
Sponsor

研究点 (1)

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