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NCT Number: NCT03835312

Sequential Transplantation of UCBSCs and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency T1DM

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.

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Key information

Conditions

Diabetes Mellitus, Type 1 Autoimmune Diseases Diabetes Mellitus Endocrine System Diseases Glucose Metabolism Disorders Immune System Diseases Immunologic Deficiency Syndromes Metabolic Diseases Nutritional and Metabolic Diseases

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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.

Exclusion criteria

  • 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.

Treatment and study plan

Sequential transplantation

Procedure

After successful matching of umbilical cord blood stem cells, patients will receive pretreatment and chemotherapy under protective isolation, followed by thawing and reinfusion of umbilical cord blood stem cells. Immunosuppressive agents will be used for GVHD prevention and anti-infection support will be provided after reinfusion. The status of umbilical cord blood stem cell implantation, immune reconstruction and therapeutic effect will be evaluated. Islet transplantation will be performed in those who meet the conditions. The long-term prognosis will be observed by long-term follow-up.

Primary outcomes

  1. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 3 months

    Islet function (concentration of serum C-peptide)

  2. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 3 years

    Islet function (concentration of serum C-peptide)

Secondary outcomes

  1. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 6 months

    Islet function (concentration of serum C-peptide)

  2. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 9 months

    Islet function (concentration of serum C-peptide)

  3. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 12 months

    Islet function (concentration of serum C-peptide)

  4. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 15 months

    Islet function (concentration of serum C-peptide)

  5. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 18 months

    Islet function (concentration of serum C-peptide)

  6. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 21 months

    Islet function (concentration of serum C-peptide)

  7. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 24 months

    Islet function (concentration of serum C-peptide)

  8. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 27 months

    Islet function (concentration of serum C-peptide)

  9. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 30 months

    Islet function (concentration of serum C-peptide)

  10. Concentration of serum C-peptide

    Time frame: from the completion of treatment to 33 months

    Islet function (concentration of serum C-peptide)

  11. Concentration of serum insulin

    Time frame: from the completion of treatment to 3 months

    Concentration of serum insulin

  12. Concentration of serum insulin

    Time frame: from the completion of treatment to 6 months

    Concentration of serum insulin

  13. Concentration of serum insulin

    Time frame: from the completion of treatment to 9 months

    Concentration of serum insulin

  14. Concentration of serum insulin

    Time frame: from the completion of treatment to 12 months

    Concentration of serum insulin

  15. Concentration of serum insulin

    Time frame: from the completion of treatment to 15 months

    Concentration of serum insulin

  16. Concentration of serum insulin

    Time frame: from the completion of treatment to 18 months

    Concentration of serum insulin

  17. Concentration of serum insulin

    Time frame: from the completion of treatment to 21 months

    Concentration of serum insulin

  18. Concentration of serum insulin

    Time frame: from the completion of treatment to 24 months

    Concentration of serum insulin

  19. Concentration of serum insulin

    Time frame: from the completion of treatment to 27 months

    Concentration of serum insulin

  20. Concentration of serum insulin

    Time frame: from the completion of treatment to 30 months

    Concentration of serum insulin

  21. Concentration of serum insulin

    Time frame: from the completion of treatment to 33 months

    Concentration of serum insulin

  22. Concentration of serum insulin

    Time frame: from the completion of treatment to 36 months

    Concentration of serum insulin

  23. Fast blood glucose level

    Time frame: from the completion of treatment to 3 months

    Fast blood glucose level

  24. Fast blood glucose level

    Time frame: from the completion of treatment to 6 months

    Fast blood glucose level

  25. Fast blood glucose level

    Time frame: from the completion of treatment to 9 months

    Fast blood glucose level

  26. Fast blood glucose level

    Time frame: from the completion of treatment to 12 months

    Fast blood glucose level

  27. Fast blood glucose level

    Time frame: from the completion of treatment to 15 months

    Fast blood glucose level

  28. Fast blood glucose level

    Time frame: from the completion of treatment to 18 months

    Fast blood glucose level

  29. Fast blood glucose level

    Time frame: from the completion of treatment to 21 months

    Fast blood glucose level

  30. Fast blood glucose level

    Time frame: from the completion of treatment to 24 months

    Fast blood glucose level

  31. Fast blood glucose level

    Time frame: from the completion of treatment to 27 months

    Fast blood glucose level

  32. Fast blood glucose level

    Time frame: from the completion of treatment to 30 months

    Fast blood glucose level

  33. Fast blood glucose level

    Time frame: from the completion of treatment to 33 months

    Fast blood glucose level

  34. Fast blood glucose level

    Time frame: from the completion of treatment to 36 months

    Fast blood glucose level

  35. HbA1c level

    Time frame: from the completion of treatment to 3 months

    HbA1c level

  36. HbA1c level

    Time frame: from the completion of treatment to 6 months

    HbA1c level

  37. HbA1c level

    Time frame: from the completion of treatment to 9 months

    HbA1c level

  38. HbA1c level

    Time frame: from the completion of treatment to 12 months

    HbA1c level

  39. HbA1c level

    Time frame: from the completion of treatment to 15 months

    HbA1c level

  40. HbA1c level

    Time frame: from the completion of treatment to 18 months

    HbA1c level

  41. HbA1c level

    Time frame: from the completion of treatment to 21 months

    HbA1c level

  42. HbA1c level

    Time frame: from the completion of treatment to 24 months

    HbA1c level

  43. HbA1c level

    Time frame: from the completion of treatment to 27 months

    HbA1c level

  44. HbA1c level

    Time frame: from the completion of treatment to 30 months

    HbA1c level

  45. HbA1c level

    Time frame: from the completion of treatment to 33 months

    HbA1c level

  46. HbA1c level

    Time frame: from the completion of treatment to 36 months

    HbA1c level

  47. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 3 months

    occurrence of infection (number of infections)

  48. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 6 months

    occurrence of infection (number of infections)

  49. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 9 months

    occurrence of infection (number of infections)

  50. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 12 months

    occurrence of infection (number of infections)

  51. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 15 months

    occurrence of infection (number of infections)

  52. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 18 months

    occurrence of infection (number of infections)

  53. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 21 months

    occurrence of infection (number of infections)

  54. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 24 months

    occurrence of infection (number of infections)

  55. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 27 months

    occurrence of infection (number of infections)

  56. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 30 months

    occurrence of infection (number of infections)

  57. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 33 months

    occurrence of infection (number of infections)

  58. occurrence of infection (number of infections)

    Time frame: from the completion of treatment to 36 months

    occurrence of infection (number of infections)

  59. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 3 months

    Concentration of blood immunoglobulin

  60. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 6 months

    Concentration of blood immunoglobulin

  61. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 9 months

    Concentration of blood immunoglobulin

  62. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 12 months

    Concentration of blood immunoglobulin

  63. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 15 months

    Concentration of blood immunoglobulin

  64. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 18 months

    Concentration of blood immunoglobulin

  65. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 21 months

    Concentration of blood immunoglobulin

  66. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 24 months

    Concentration of blood immunoglobulin

  67. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 27 months

    Concentration of blood immunoglobulin

  68. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 30 months

    Concentration of blood immunoglobulin

  69. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 33 months

    Concentration of blood immunoglobulin

  70. Concentration of blood immunoglobulin

    Time frame: from the completion of treatment to 36 months

    Concentration of blood immunoglobulin

  71. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 3 months

    Concentration of T lymphocyte subsets

  72. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 6 months

    Concentration of T lymphocyte subsets

  73. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 9 months

    Concentration of T lymphocyte subsets

  74. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 12 months

    Concentration of T lymphocyte subsets

  75. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 15 months

    Concentration of T lymphocyte subsets

  76. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 18 months

    Concentration of T lymphocyte subsets

  77. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 21 months

    Concentration of T lymphocyte subsets

  78. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 24 months

    Concentration of T lymphocyte subsets

  79. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 27 months

    Concentration of T lymphocyte subsets

  80. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 30 months

    Concentration of T lymphocyte subsets

  81. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 33 months

    Concentration of T lymphocyte subsets

  82. Concentration of T lymphocyte subsets

    Time frame: from the completion of treatment to 36 months

    Concentration of T lymphocyte subsets

  83. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 3 months

    Cytokines (concentraion of interleukin-2)

  84. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 6 months

    Cytokines (concentraion of interleukin-2)

  85. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 9 months

    Cytokines (concentraion of interleukin-2)

  86. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 12 months

    Cytokines (concentraion of interleukin-2)

  87. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 15 months

    Cytokines (concentraion of interleukin-2)

  88. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 18 months

    Cytokines (concentraion of interleukin-2)

  89. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 21 months

    Cytokines (concentraion of interleukin-2)

  90. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 24 months

    Cytokines (concentraion of interleukin-2)

  91. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 27 months

    Cytokines (concentraion of interleukin-2)

  92. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 30 months

    Cytokines (concentraion of interleukin-2)

  93. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 33 months

    Cytokines (concentraion of interleukin-2)

  94. Concentraion of interleukin-2

    Time frame: from the completion of treatment to 36 months

    Cytokines (concentraion of interleukin-2)

  95. Body height

    Time frame: from the completion of treatment to 3 months

    Body height

  96. Body height

    Time frame: from the completion of treatment to 6 months

    Body height

  97. Body height

    Time frame: from the completion of treatment to 9 months

    Body height

  98. Body height

    Time frame: from the completion of treatment to 12 months

    Body height

  99. Body height

    Time frame: from the completion of treatment to 15 months

    Body height

  100. Body height

    Time frame: from the completion of treatment to 18 months

    Body height

  101. Body height

    Time frame: from the completion of treatment to 21 months

    Body height

  102. Body height

    Time frame: from the completion of treatment to 24 months

    Body height

  103. Body height

    Time frame: from the completion of treatment to 27 months

    Body height

  104. Body height

    Time frame: from the completion of treatment to 30 months

    Body height

  105. Body height

    Time frame: from the completion of treatment to 33 months

    Body height

  106. Body height

    Time frame: from the completion of treatment to 36 months

    Body height

  107. Body weight

    Time frame: from the completion of treatment to 3 months

    Body weight

  108. Body weight

    Time frame: from the completion of treatment to 6 months

    Body weight

  109. Body weight

    Time frame: from the completion of treatment to 9 months

    Body weight

  110. Body weight

    Time frame: from the completion of treatment to 12 months

    Body weight

  111. Body weight

    Time frame: from the completion of treatment to 15 months

    Body weight

  112. Body weight

    Time frame: from the completion of treatment to 18 months

    Body weight

  113. Body weight

    Time frame: from the completion of treatment to 21 months

    Body weight

  114. Body weight

    Time frame: from the completion of treatment to 24 months

    Body weight

  115. Body weight

    Time frame: from the completion of treatment to 27 months

    Body weight

  116. Body weight

    Time frame: from the completion of treatment to 30 months

    Body weight

  117. Body weight

    Time frame: from the completion of treatment to 33 months

    Body weight

  118. Body weight

    Time frame: from the completion of treatment to 36 months

    Body weight

  119. Tanner stage

    Time frame: from the completion of treatment to 3 months

    Puberty change (Tanner stage)

  120. Tanner stage

    Time frame: from the completion of treatment to 6 months

    Puberty change (Tanner stage)

  121. Tanner stage

    Time frame: from the completion of treatment to 9 months

    Puberty change (Tanner stage)

  122. Tanner stage

    Time frame: from the completion of treatment to 12 months

    Puberty change (Tanner stage)

  123. Tanner stage

    Time frame: from the completion of treatment to 15 months

    Puberty change (Tanner stage)

  124. Tanner stage

    Time frame: from the completion of treatment to 18 months

    Puberty change (Tanner stage)

  125. Tanner stage

    Time frame: from the completion of treatment to 21 months

    Puberty change (Tanner stage)

  126. Tanner stage

    Time frame: from the completion of treatment to 24 months

    Puberty change (Tanner stage)

  127. Tanner stage

    Time frame: from the completion of treatment to 27 months

    Puberty change (Tanner stage)

  128. Tanner stage

    Time frame: from the completion of treatment to 30 months

    Puberty change (Tanner stage)

  129. Tanner stage

    Time frame: from the completion of treatment to 33 months

    Puberty change (Tanner stage)

  130. Tanner stage

    Time frame: from the completion of treatment to 36 months

    Puberty change (Tanner stage)

  131. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 3 months

    Occurrence of graft versus host disease

  132. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 6 months

    Occurrence of graft versus host disease

  133. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 9 months

    Occurrence of graft versus host disease

  134. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 12 months

    Occurrence of graft versus host disease

  135. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 15 months

    Occurrence of graft versus host disease

  136. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 18 months

    Occurrence of graft versus host disease

  137. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 21 months

    Occurrence of graft versus host disease

  138. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 24 months

    Occurrence of graft versus host disease

  139. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 27 months

    Occurrence of graft versus host disease

  140. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 30 months

    Occurrence of graft versus host disease

  141. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 33 months

    Occurrence of graft versus host disease

  142. Occurrence of graft versus host disease

    Time frame: from the completion of treatment to 36 months

    Occurrence of graft versus host disease

Study contacts

Contact information is provided by the study sponsor or research team.

Luo Feihong

CONTACT

[email protected]

+862164931226 ext. +862164931226

Xu Zhenran

CONTACT

[email protected]

+862164931226 ext. +862164931226

Sponsors and collaborators

Lead sponsor

Children's Hospital of Fudan University

Other

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2028
Study completion
2030
First posted
Feb 8, 2019
Registry last updated
Mar 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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