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AzTrials

Diabetes Mellitus, Type 1NCT03835312

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

A study testing Sequential transplantation in people with Diabetes Mellitus, Type 1 and Immunologic Deficiency Syndromes, run by Children's Hospital of Fudan University at 1 site in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages Up to 18 years
What is being tested?
Sequential transplantation
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
1 siteChina
Study dates
20 Feb 2019to 31 Dec 2030
Phase
Interventional
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Luo Feihong contact

+862164931226 ext. +862164931226

luofh@fudan.edu.cn

Xu Zhenran contact

+862164931226 ext. +862164931226

xu_zhenran@163.com

China1 site, 1 recruiting

  • Children's Hospital of Fudan University

    Shanghai, Shanghai Municipality

    Luo Feihong, +862164931226 ext. +862164931226, luofh@fudan.edu.cn

    Xu Zhenran, +862164931226 ext. +862164931226, xu_zhenran@fudan.edu.cn

    Recruiting

Other trials in China

About this study

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

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.

Read the detailed description

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.

Being tested
Sequential transplantation
Keywords
Islets of Langerhans Transplantation, Cord Blood Stem Cell Transplantation
Medical subject headings
Diabetes Mellitus, Type 1, Immunologic Deficiency Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Sequential transplantationProcedure or surgery

    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.

Study groups in NCT03835312
Study groupRoleWhat they receive
InterventionalExperimentalSequential transplantation of umbilical cord blood stem cells and islet cells
Receives: Procedure: Sequential transplantation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 3 months

  2. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 3 years

Secondary outcomes (142)

  1. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 6 months

  2. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 9 months

  3. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 12 months

  4. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 15 months

  5. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 18 months

  6. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 21 months

  7. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 24 months

  8. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 27 months

  9. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 30 months

  10. Concentration of serum C-peptide

    Islet function (concentration of serum C-peptide)

    Measured: from the completion of treatment to 33 months

  11. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 3 months

  12. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 6 months

  13. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 9 months

  14. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 12 months

  15. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 15 months

  16. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 18 months

  17. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 21 months

  18. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 24 months

  19. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 27 months

  20. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 30 months

  21. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 33 months

  22. Concentration of serum insulin

    Concentration of serum insulin

    Measured: from the completion of treatment to 36 months

  23. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 3 months

  24. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 6 months

  25. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 9 months

  26. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 12 months

  27. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 15 months

  28. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 18 months

  29. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 21 months

  30. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 24 months

  31. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 27 months

  32. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 30 months

  33. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 33 months

  34. Fast blood glucose level

    Fast blood glucose level

    Measured: from the completion of treatment to 36 months

  35. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 3 months

  36. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 6 months

  37. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 9 months

  38. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 12 months

  39. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 15 months

  40. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 18 months

  41. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 21 months

  42. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 24 months

  43. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 27 months

  44. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 30 months

  45. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 33 months

  46. HbA1c level

    HbA1c level

    Measured: from the completion of treatment to 36 months

  47. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 3 months

  48. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 6 months

  49. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 9 months

  50. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 12 months

  51. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 15 months

  52. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 18 months

  53. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 21 months

  54. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 24 months

  55. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 27 months

  56. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 30 months

  57. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 33 months

  58. occurrence of infection (number of infections)

    occurrence of infection (number of infections)

    Measured: from the completion of treatment to 36 months

  59. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 3 months

  60. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 6 months

  61. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 9 months

  62. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 12 months

  63. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 15 months

  64. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 18 months

  65. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 21 months

  66. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 24 months

  67. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 27 months

  68. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 30 months

  69. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 33 months

  70. Concentration of blood immunoglobulin

    Concentration of blood immunoglobulin

    Measured: from the completion of treatment to 36 months

  71. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 3 months

  72. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 6 months

  73. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 9 months

  74. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 12 months

  75. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 15 months

  76. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 18 months

  77. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 21 months

  78. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 24 months

  79. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 27 months

  80. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 30 months

  81. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 33 months

  82. Concentration of T lymphocyte subsets

    Concentration of T lymphocyte subsets

    Measured: from the completion of treatment to 36 months

  83. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 3 months

  84. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 6 months

  85. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 9 months

  86. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 12 months

  87. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 15 months

  88. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 18 months

  89. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 21 months

  90. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 24 months

  91. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 27 months

  92. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 30 months

  93. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 33 months

  94. Concentraion of interleukin-2

    Cytokines (concentraion of interleukin-2)

    Measured: from the completion of treatment to 36 months

  95. Body height

    Body height

    Measured: from the completion of treatment to 3 months

  96. Body height

    Body height

    Measured: from the completion of treatment to 6 months

  97. Body height

    Body height

    Measured: from the completion of treatment to 9 months

  98. Body height

    Body height

    Measured: from the completion of treatment to 12 months

  99. Body height

    Body height

    Measured: from the completion of treatment to 15 months

  100. Body height

    Body height

    Measured: from the completion of treatment to 18 months

  101. Body height

    Body height

    Measured: from the completion of treatment to 21 months

  102. Body height

    Body height

    Measured: from the completion of treatment to 24 months

  103. Body height

    Body height

    Measured: from the completion of treatment to 27 months

  104. Body height

    Body height

    Measured: from the completion of treatment to 30 months

  105. Body height

    Body height

    Measured: from the completion of treatment to 33 months

  106. Body height

    Body height

    Measured: from the completion of treatment to 36 months

  107. Body weight

    Body weight

    Measured: from the completion of treatment to 3 months

  108. Body weight

    Body weight

    Measured: from the completion of treatment to 6 months

  109. Body weight

    Body weight

    Measured: from the completion of treatment to 9 months

  110. Body weight

    Body weight

    Measured: from the completion of treatment to 12 months

  111. Body weight

    Body weight

    Measured: from the completion of treatment to 15 months

  112. Body weight

    Body weight

    Measured: from the completion of treatment to 18 months

  113. Body weight

    Body weight

    Measured: from the completion of treatment to 21 months

  114. Body weight

    Body weight

    Measured: from the completion of treatment to 24 months

  115. Body weight

    Body weight

    Measured: from the completion of treatment to 27 months

  116. Body weight

    Body weight

    Measured: from the completion of treatment to 30 months

  117. Body weight

    Body weight

    Measured: from the completion of treatment to 33 months

  118. Body weight

    Body weight

    Measured: from the completion of treatment to 36 months

  119. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 3 months

  120. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 6 months

  121. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 9 months

  122. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 12 months

  123. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 15 months

  124. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 18 months

  125. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 21 months

  126. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 24 months

  127. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 27 months

  128. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 30 months

  129. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 33 months

  130. Tanner stage

    Puberty change (Tanner stage)

    Measured: from the completion of treatment to 36 months

  131. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 3 months

  132. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 6 months

  133. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 9 months

  134. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 12 months

  135. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 15 months

  136. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 18 months

  137. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 21 months

  138. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 24 months

  139. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 27 months

  140. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 30 months

  141. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 33 months

  142. Occurrence of graft versus host disease

    Occurrence of graft versus host disease

    Measured: from the completion of treatment to 36 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Feb 2019
  2. Study start20 Feb 2019
  3. Primary completion31 Dec 2028 (estimated)
  4. Study completion31 Dec 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03835312
Sponsor's protocol ID
SeqTranUCBC&IC-001
Status last verified
Mar 2026
First submitted
31 Jan 2019
Last updated
17 Mar 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Cho NH, Shaw JE, Karuranga S, Huang Y, da Rocha Fernandes JD, Ohlrogge AW, Malanda B. IDF Diabetes Atlas: Global estimates of diabetes prevalence for 2017 and projections for 2045. Diabetes Res Clin Pract. 2018 Apr;138:271-281. doi: 10.1016/j.diabres.2018.02.023. Epub 2018 Feb 26.

    PubMed 29496507

  • Fu H, Shen SX, Chen ZW, Wang JJ, Ye TT, LaPorte RE, Tajima N. Shanghai, China, has the lowest confirmed incidence of childhood diabetes in the world. Diabetes Care. 1994 Oct;17(10):1206-8. doi: 10.2337/diacare.17.10.1206.

    PubMed 7821146

  • Zhao Z, Sun C, Wang C, Li P, Wang W, Ye J, Gu X, Wang X, Shen S, Zhi D, Lu Z, Ye R, Cheng R, Xi L, Li X, Zheng Z, Zhang M, Luo F. Rapidly rising incidence of childhood type 1 diabetes in Chinese population: epidemiology in Shanghai during 1997-2011. Acta Diabetol. 2014 Dec;51(6):947-53. doi: 10.1007/s00592-014-0590-2. Epub 2014 Apr 29.

    PubMed 24777734

  • Daniels M, DuBose SN, Maahs DM, Beck RW, Fox LA, Gubitosi-Klug R, Laffel LM, Miller KM, Speer H, Tamborlane WV, Tansey MJ; T1D Exchange Clinic Network. Factors associated with microalbuminuria in 7,549 children and adolescents with type 1 diabetes in the T1D Exchange clinic registry. Diabetes Care. 2013 Sep;36(9):2639-45. doi: 10.2337/dc12-2192. Epub 2013 Apr 22.

    PubMed 23610082

  • Amin R, Widmer B, Prevost AT, Schwarze P, Cooper J, Edge J, Marcovecchio L, Neil A, Dalton RN, Dunger DB. Risk of microalbuminuria and progression to macroalbuminuria in a cohort with childhood onset type 1 diabetes: prospective observational study. BMJ. 2008 Mar 29;336(7646):697-701. doi: 10.1136/bmj.39478.378241.BE. Epub 2008 Mar 18.

    PubMed 18349042

  • Mollsten A, Svensson M, Waernbaum I, Berhan Y, Schon S, Nystrom L, Arnqvist HJ, Dahlquist G; Swedish Childhood Diabetes Study Group; Diabetes Incidence Study in Sweden; Swedish Renal Registry. Cumulative risk, age at onset, and sex-specific differences for developing end-stage renal disease in young patients with type 1 diabetes: a nationwide population-based cohort study. Diabetes. 2010 Jul;59(7):1803-8. doi: 10.2337/db09-1744. Epub 2010 Apr 27.

    PubMed 20424230

  • American Diabetes Association. (2) Classification and diagnosis of diabetes. Diabetes Care. 2015 Jan;38 Suppl:S8-S16. doi: 10.2337/dc15-S005. No abstract available.

    PubMed 25537714

  • Wildin RS, Smyk-Pearson S, Filipovich AH. Clinical and molecular features of the immunodysregulation, polyendocrinopathy, enteropathy, X linked (IPEX) syndrome. J Med Genet. 2002 Aug;39(8):537-45. doi: 10.1136/jmg.39.8.537.

    PubMed 12161590

  • Uzel G, Sampaio EP, Lawrence MG, Hsu AP, Hackett M, Dorsey MJ, Noel RJ, Verbsky JW, Freeman AF, Janssen E, Bonilla FA, Pechacek J, Chandrasekaran P, Browne SK, Agharahimi A, Gharib AM, Mannurita SC, Yim JJ, Gambineri E, Torgerson T, Tran DQ, Milner JD, Holland SM. Dominant gain-of-function STAT1 mutations in FOXP3 wild-type immune dysregulation-polyendocrinopathy-enteropathy-X-linked-like syndrome. J Allergy Clin Immunol. 2013 Jun;131(6):1611-23. doi: 10.1016/j.jaci.2012.11.054. Epub 2013 Mar 25.

    PubMed 23534974

  • Bennett CL, Christie J, Ramsdell F, Brunkow ME, Ferguson PJ, Whitesell L, Kelly TE, Saulsbury FT, Chance PF, Ochs HD. The immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome (IPEX) is caused by mutations of FOXP3. Nat Genet. 2001 Jan;27(1):20-1. doi: 10.1038/83713.

    PubMed 11137993

  • Rao A, Kamani N, Filipovich A, Lee SM, Davies SM, Dalal J, Shenoy S. Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning. Blood. 2007 Jan 1;109(1):383-5. doi: 10.1182/blood-2006-05-025072. Epub 2006 Sep 21.

    PubMed 16990602

  • van de Veerdonk FL, Plantinga TS, Hoischen A, Smeekens SP, Joosten LA, Gilissen C, Arts P, Rosentul DC, Carmichael AJ, Smits-van der Graaf CA, Kullberg BJ, van der Meer JW, Lilic D, Veltman JA, Netea MG. STAT1 mutations in autosomal dominant chronic mucocutaneous candidiasis. N Engl J Med. 2011 Jul 7;365(1):54-61. doi: 10.1056/NEJMoa1100102. Epub 2011 Jun 29.

    PubMed 21714643

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Questions about this trial

What is this clinical trial studying?

A study testing Sequential transplantation in people with Diabetes Mellitus, Type 1 and Immunologic Deficiency Syndromes, run by Children's Hospital of Fudan University at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 17 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Luo Feihong (+862164931226 ext. +862164931226, luofh@fudan.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Concentration of serum C-peptide (time frame: from the completion of treatment to 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03835312, which also lists 17 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2026; AzTrials last refreshed its copy on 8 Nov 2026.

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