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

• Sponsor: Children's Hospital of Fudan University (Other)
• Overall Status: Recruiting
• CT.gov ID: NCT03835312
• Collaborator: (none)
• Enrollment: 50
• Location: 1
• Arm: 1
• Anticipated Duration (Months): 70.3
• Patients Per Site Per Month: 0.7

Study Details

Study Description

Brief 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. Children with stable immune reconstruction will than receive islet cell transplantation.

Condition or Disease	Intervention/Treatment	Phase
Diabetes Mellitus, Type 1; Immunologic Deficiency Syndromes	Procedure: Sequential transplantation	N/A

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.

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 50 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Sequential Transplantation of Umbilical Cord Blood Stem Cells and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency Type 1 Diabetes Mellitus
• Actual Study Start Date: Feb 20, 2019
• Anticipated Primary Completion Date: Dec 31, 2024
• Anticipated Study Completion Date: Dec 31, 2024

Arms and Interventions

Arm

Intervention/Treatment

Experimental: Interventional Sequential transplantation of umbilical cord blood stem cells and islet cells

Procedure: Sequential transplantation; 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.

Outcome Measures

Primary Outcome Measures

1. Concentration of serum C-peptide [from the completion of treatment to 3 months]

   Islet function (concentration of serum C-peptide)

2. Concentration of serum C-peptide [from the completion of treatment to 3 years]

   Islet function (concentration of serum C-peptide)

Secondary Outcome Measures

1. Concentration of serum C-peptide [from the completion of treatment to 6 months]

   Islet function (concentration of serum C-peptide)

2. Concentration of serum C-peptide [from the completion of treatment to 9 months]

   Islet function (concentration of serum C-peptide)

3. Concentration of serum C-peptide [from the completion of treatment to 12 months]

   Islet function (concentration of serum C-peptide)

4. Concentration of serum C-peptide [from the completion of treatment to 15 months]

   Islet function (concentration of serum C-peptide)

5. Concentration of serum C-peptide [from the completion of treatment to 18 months]

   Islet function (concentration of serum C-peptide)

6. Concentration of serum C-peptide [from the completion of treatment to 21 months]

   Islet function (concentration of serum C-peptide)

7. Concentration of serum C-peptide [from the completion of treatment to 24 months]

   Islet function (concentration of serum C-peptide)

8. Concentration of serum C-peptide [from the completion of treatment to 27 months]

   Islet function (concentration of serum C-peptide)

9. Concentration of serum C-peptide [from the completion of treatment to 30 months]

   Islet function (concentration of serum C-peptide)

10. Concentration of serum C-peptide [from the completion of treatment to 33 months]

    Islet function (concentration of serum C-peptide)

11. Concentration of serum insulin [from the completion of treatment to 3 months]

    Concentration of serum insulin

12. Concentration of serum insulin [from the completion of treatment to 6 months]

    Concentration of serum insulin

13. Concentration of serum insulin [from the completion of treatment to 9 months]

    Concentration of serum insulin

14. Concentration of serum insulin [from the completion of treatment to 12 months]

    Concentration of serum insulin

15. Concentration of serum insulin [from the completion of treatment to 15 months]

    Concentration of serum insulin

16. Concentration of serum insulin [from the completion of treatment to 18 months]

    Concentration of serum insulin

17. Concentration of serum insulin [from the completion of treatment to 21 months]

    Concentration of serum insulin

18. Concentration of serum insulin [from the completion of treatment to 24 months]

    Concentration of serum insulin

19. Concentration of serum insulin [from the completion of treatment to 27 months]

    Concentration of serum insulin

20. Concentration of serum insulin [from the completion of treatment to 30 months]

    Concentration of serum insulin

21. Concentration of serum insulin [from the completion of treatment to 33 months]

    Concentration of serum insulin

22. Concentration of serum insulin [from the completion of treatment to 36 months]

    Concentration of serum insulin

23. Fast blood glucose level [from the completion of treatment to 3 months]

    Fast blood glucose level

24. Fast blood glucose level [from the completion of treatment to 6 months]

    Fast blood glucose level

25. Fast blood glucose level [from the completion of treatment to 9 months]

    Fast blood glucose level

26. Fast blood glucose level [from the completion of treatment to 12 months]

    Fast blood glucose level

27. Fast blood glucose level [from the completion of treatment to 15 months]

    Fast blood glucose level

28. Fast blood glucose level [from the completion of treatment to 18 months]

    Fast blood glucose level

29. Fast blood glucose level [from the completion of treatment to 21 months]

    Fast blood glucose level

30. Fast blood glucose level [from the completion of treatment to 24 months]

    Fast blood glucose level

31. Fast blood glucose level [from the completion of treatment to 27 months]

    Fast blood glucose level

32. Fast blood glucose level [from the completion of treatment to 30 months]

    Fast blood glucose level

33. Fast blood glucose level [from the completion of treatment to 33 months]

    Fast blood glucose level

34. Fast blood glucose level [from the completion of treatment to 36 months]

    Fast blood glucose level

35. HbA1c level [from the completion of treatment to 3 months]

    HbA1c level

36. HbA1c level [from the completion of treatment to 6 months]

    HbA1c level

37. HbA1c level [from the completion of treatment to 9 months]

    HbA1c level

38. HbA1c level [from the completion of treatment to 12 months]

    HbA1c level

39. HbA1c level [from the completion of treatment to 15 months]

    HbA1c level

40. HbA1c level [from the completion of treatment to 18 months]

    HbA1c level

41. HbA1c level [from the completion of treatment to 21 months]

    HbA1c level

42. HbA1c level [from the completion of treatment to 24 months]

    HbA1c level

43. HbA1c level [from the completion of treatment to 27 months]

    HbA1c level

44. HbA1c level [from the completion of treatment to 30 months]

    HbA1c level

45. HbA1c level [from the completion of treatment to 33 months]

    HbA1c level

46. HbA1c level [from the completion of treatment to 36 months]

    HbA1c level

47. occurrence of infection (number of infections) [from the completion of treatment to 3 months]

    occurrence of infection (number of infections)

48. occurrence of infection (number of infections) [from the completion of treatment to 6 months]

    occurrence of infection (number of infections)

49. occurrence of infection (number of infections) [from the completion of treatment to 9 months]

    occurrence of infection (number of infections)

50. occurrence of infection (number of infections) [from the completion of treatment to 12 months]

    occurrence of infection (number of infections)

51. occurrence of infection (number of infections) [from the completion of treatment to 15 months]

    occurrence of infection (number of infections)

52. occurrence of infection (number of infections) [from the completion of treatment to 18 months]

    occurrence of infection (number of infections)

53. occurrence of infection (number of infections) [from the completion of treatment to 21 months]

    occurrence of infection (number of infections)

54. occurrence of infection (number of infections) [from the completion of treatment to 24 months]

    occurrence of infection (number of infections)

55. occurrence of infection (number of infections) [from the completion of treatment to 27 months]

    occurrence of infection (number of infections)

56. occurrence of infection (number of infections) [from the completion of treatment to 30 months]

    occurrence of infection (number of infections)

57. occurrence of infection (number of infections) [from the completion of treatment to 33 months]

    occurrence of infection (number of infections)

58. occurrence of infection (number of infections) [from the completion of treatment to 36 months]

    occurrence of infection (number of infections)

59. Concentration of blood immunoglobulin [from the completion of treatment to 3 months]

    Concentration of blood immunoglobulin

60. Concentration of blood immunoglobulin [from the completion of treatment to 6 months]

    Concentration of blood immunoglobulin

61. Concentration of blood immunoglobulin [from the completion of treatment to 9 months]

    Concentration of blood immunoglobulin

62. Concentration of blood immunoglobulin [from the completion of treatment to 12 months]

    Concentration of blood immunoglobulin

63. Concentration of blood immunoglobulin [from the completion of treatment to 15 months]

    Concentration of blood immunoglobulin

64. Concentration of blood immunoglobulin [from the completion of treatment to 18 months]

    Concentration of blood immunoglobulin

65. Concentration of blood immunoglobulin [from the completion of treatment to 21 months]

    Concentration of blood immunoglobulin

66. Concentration of blood immunoglobulin [from the completion of treatment to 24 months]

    Concentration of blood immunoglobulin

67. Concentration of blood immunoglobulin [from the completion of treatment to 27 months]

    Concentration of blood immunoglobulin

68. Concentration of blood immunoglobulin [from the completion of treatment to 30 months]

    Concentration of blood immunoglobulin

69. Concentration of blood immunoglobulin [from the completion of treatment to 33 months]

    Concentration of blood immunoglobulin

70. Concentration of blood immunoglobulin [from the completion of treatment to 36 months]

    Concentration of blood immunoglobulin

71. Concentration of T lymphocyte subsets [from the completion of treatment to 3 months]

    Concentration of T lymphocyte subsets

72. Concentration of T lymphocyte subsets [from the completion of treatment to 6 months]

    Concentration of T lymphocyte subsets

73. Concentration of T lymphocyte subsets [from the completion of treatment to 9 months]

    Concentration of T lymphocyte subsets

74. Concentration of T lymphocyte subsets [from the completion of treatment to 12 months]

    Concentration of T lymphocyte subsets

75. Concentration of T lymphocyte subsets [from the completion of treatment to 15 months]

    Concentration of T lymphocyte subsets

76. Concentration of T lymphocyte subsets [from the completion of treatment to 18 months]

    Concentration of T lymphocyte subsets

77. Concentration of T lymphocyte subsets [from the completion of treatment to 21 months]

    Concentration of T lymphocyte subsets

78. Concentration of T lymphocyte subsets [from the completion of treatment to 24 months]

    Concentration of T lymphocyte subsets

79. Concentration of T lymphocyte subsets [from the completion of treatment to 27 months]

    Concentration of T lymphocyte subsets

80. Concentration of T lymphocyte subsets [from the completion of treatment to 30 months]

    Concentration of T lymphocyte subsets

81. Concentration of T lymphocyte subsets [from the completion of treatment to 33 months]

    Concentration of T lymphocyte subsets

82. Concentration of T lymphocyte subsets [from the completion of treatment to 36 months]

    Concentration of T lymphocyte subsets

83. Concentraion of interleukin-2 [from the completion of treatment to 3 months]

    Cytokines (concentraion of interleukin-2)

84. Concentraion of interleukin-2 [from the completion of treatment to 6 months]

    Cytokines (concentraion of interleukin-2)

85. Concentraion of interleukin-2 [from the completion of treatment to 9 months]

    Cytokines (concentraion of interleukin-2)

86. Concentraion of interleukin-2 [from the completion of treatment to 12 months]

    Cytokines (concentraion of interleukin-2)

87. Concentraion of interleukin-2 [from the completion of treatment to 15 months]

    Cytokines (concentraion of interleukin-2)

88. Concentraion of interleukin-2 [from the completion of treatment to 18 months]

    Cytokines (concentraion of interleukin-2)

89. Concentraion of interleukin-2 [from the completion of treatment to 21 months]

    Cytokines (concentraion of interleukin-2)

90. Concentraion of interleukin-2 [from the completion of treatment to 24 months]

    Cytokines (concentraion of interleukin-2)

91. Concentraion of interleukin-2 [from the completion of treatment to 27 months]

    Cytokines (concentraion of interleukin-2)

92. Concentraion of interleukin-2 [from the completion of treatment to 30 months]

    Cytokines (concentraion of interleukin-2)

93. Concentraion of interleukin-2 [from the completion of treatment to 33 months]

    Cytokines (concentraion of interleukin-2)

94. Concentraion of interleukin-2 [from the completion of treatment to 36 months]

    Cytokines (concentraion of interleukin-2)

95. Body height [from the completion of treatment to 3 months]

    Body height

96. Body height [from the completion of treatment to 6 months]

    Body height

97. Body height [from the completion of treatment to 9 months]

    Body height

98. Body height [from the completion of treatment to 12 months]

    Body height

99. Body height [from the completion of treatment to 15 months]

    Body height

100. Body height [from the completion of treatment to 18 months]

     Body height

101. Body height [from the completion of treatment to 21 months]

     Body height

102. Body height [from the completion of treatment to 24 months]

     Body height

103. Body height [from the completion of treatment to 27 months]

     Body height

104. Body height [from the completion of treatment to 30 months]

     Body height

105. Body height [from the completion of treatment to 33 months]

     Body height

106. Body height [from the completion of treatment to 36 months]

     Body height

107. Body weight [from the completion of treatment to 3 months]

     Body weight

108. Body weight [from the completion of treatment to 6 months]

     Body weight

109. Body weight [from the completion of treatment to 9 months]

     Body weight

110. Body weight [from the completion of treatment to 12 months]

     Body weight

111. Body weight [from the completion of treatment to 15 months]

     Body weight

112. Body weight [from the completion of treatment to 18 months]

     Body weight

113. Body weight [from the completion of treatment to 21 months]

     Body weight

114. Body weight [from the completion of treatment to 24 months]

     Body weight

115. Body weight [from the completion of treatment to 27 months]

     Body weight

116. Body weight [from the completion of treatment to 30 months]

     Body weight

117. Body weight [from the completion of treatment to 33 months]

     Body weight

118. Body weight [from the completion of treatment to 36 months]

     Body weight

119. Tanner stage [from the completion of treatment to 3 months]

     Puberty change (Tanner stage)

120. Tanner stage [from the completion of treatment to 6 months]

     Puberty change (Tanner stage)

121. Tanner stage [from the completion of treatment to 9 months]

     Puberty change (Tanner stage)

122. Tanner stage [from the completion of treatment to 12 months]

     Puberty change (Tanner stage)

123. Tanner stage [from the completion of treatment to 15 months]

     Puberty change (Tanner stage)

124. Tanner stage [from the completion of treatment to 18 months]

     Puberty change (Tanner stage)

125. Tanner stage [from the completion of treatment to 21 months]

     Puberty change (Tanner stage)

126. Tanner stage [from the completion of treatment to 24 months]

     Puberty change (Tanner stage)

127. Tanner stage [from the completion of treatment to 27 months]

     Puberty change (Tanner stage)

128. Tanner stage [from the completion of treatment to 30 months]

     Puberty change (Tanner stage)

129. Tanner stage [from the completion of treatment to 33 months]

     Puberty change (Tanner stage)

130. Tanner stage [from the completion of treatment to 36 months]

     Puberty change (Tanner stage)

131. Occurrence of graft versus host disease [from the completion of treatment to 3 months]

     Occurrence of graft versus host disease

132. Occurrence of graft versus host disease [from the completion of treatment to 6 months]

     Occurrence of graft versus host disease

133. Occurrence of graft versus host disease [from the completion of treatment to 9 months]

     Occurrence of graft versus host disease

134. Occurrence of graft versus host disease [from the completion of treatment to 12 months]

     Occurrence of graft versus host disease

135. Occurrence of graft versus host disease [from the completion of treatment to 15 months]

     Occurrence of graft versus host disease

136. Occurrence of graft versus host disease [from the completion of treatment to 18 months]

     Occurrence of graft versus host disease

137. Occurrence of graft versus host disease [from the completion of treatment to 21 months]

     Occurrence of graft versus host disease

138. Occurrence of graft versus host disease [from the completion of treatment to 24 months]

     Occurrence of graft versus host disease

139. Occurrence of graft versus host disease [from the completion of treatment to 27 months]

     Occurrence of graft versus host disease

140. Occurrence of graft versus host disease [from the completion of treatment to 30 months]

     Occurrence of graft versus host disease

141. Occurrence of graft versus host disease [from the completion of treatment to 33 months]

     Occurrence of graft versus host disease

142. Occurrence of graft versus host disease [from the completion of treatment to 36 months]

     Occurrence of graft versus host disease

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 18 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1.Type 1 diabetes mellitus children with genetic immunodeficiency

1. 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.

2. 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.

3. 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:

1. Mature and effective treatment methods are available.

2. HIV, HBV and HCV were positive.

3. A the active period of infection.

4. At the active stage of malignant tumors.

5. Combination of other fatal diseases.

6. Existence of mental and psychological diseases.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital of Fudan University	Shanghai	Shanghai	China

Sponsors and Collaborators

• Children's Hospital of Fudan University

Investigators

• Study Chair: Luo Feihong, Children's Hospital of Fudan University

Study Documents (Full-Text)

• Study Protocol - Oct 16, 2018
• Statistical Analysis Plan - Oct 16, 2018
• Informed Consent Form - Oct 16, 2018

More Information

Publications

• Ahearn AJ, Parekh JR, Posselt AM. Islet transplantation for Type 1 diabetes: where are we now? Expert Rev Clin Immunol. 2015 Jan;11(1):59-68. doi: 10.1586/1744666X.2015.978291. Epub 2014 Dec 2. Review.
• American Diabetes Association. (2) Classification and diagnosis of diabetes. Diabetes Care. 2015 Jan;38 Suppl:S8-S16. doi: 10.2337/dc15-S005. Review.
• 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.
• 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.
• 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.
• 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.
• 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.
• Kopan C, Tucker T, Alexander M, Mohammadi MR, Pone EJ, Lakey JRT. Approaches in Immunotherapy, Regenerative Medicine, and Bioengineering for Type 1 Diabetes. Front Immunol. 2018 Jun 12;9:1354. doi: 10.3389/fimmu.2018.01354. eCollection 2018. Review.
• Leiding JW, Okada S, Hagin D, Abinun M, Shcherbina A, Balashov DN, Kim VHD, Ovadia A, Guthery SL, Pulsipher M, Lilic D, Devlin LA, Christie S, Depner M, Fuchs S, van Royen-Kerkhof A, Lindemans C, Petrovic A, Sullivan KE, Bunin N, Kilic SS, Arpaci F, Calle-Martin O, Martinez-Martinez L, Aldave JC, Kobayashi M, Ohkawa T, Imai K, Iguchi A, Roifman CM, Gennery AR, Slatter M, Ochs HD, Morio T, Torgerson TR; Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the Primary Immune Deficiency Treatment Consortium. Hematopoietic stem cell transplantation in patients with gain-of-function signal transducer and activator of transcription 1 mutations. J Allergy Clin Immunol. 2018 Feb;141(2):704-717.e5. doi: 10.1016/j.jaci.2017.03.049. Epub 2017 Jun 7.
• Möllsten A, Svensson M, Waernbaum I, Berhan Y, Schön S, Nyström 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.
• Niclauss N, Morel P, Berney T. Has the gap between pancreas and islet transplantation closed? Transplantation. 2014 Sep 27;98(6):593-9. doi: 10.1097/TP.0000000000000288. Review.
• 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. Epub 2006 Sep 21.
• Toubiana J, Okada S, Hiller J, Oleastro M, Lagos Gomez M, Aldave Becerra JC, Ouachée-Chardin M, Fouyssac F, Girisha KM, Etzioni A, Van Montfrans J, Camcioglu Y, Kerns LA, Belohradsky B, Blanche S, Bousfiha A, Rodriguez-Gallego C, Meyts I, Kisand K, Reichenbach J, Renner ED, Rosenzweig S, Grimbacher B, van de Veerdonk FL, Traidl-Hoffmann C, Picard C, Marodi L, Morio T, Kobayashi M, Lilic D, Milner JD, Holland S, Casanova JL, Puel A; International STAT1 Gain-of-Function Study Group. Heterozygous STAT1 gain-of-function mutations underlie an unexpectedly broad clinical phenotype. Blood. 2016 Jun 23;127(25):3154-64. doi: 10.1182/blood-2015-11-679902. Epub 2016 Apr 25.
• 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.
• 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.
• 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. Review.
• 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.

• Responsible Party: Children's Hospital of Fudan University
• ClinicalTrials.gov Identifier: NCT03835312
• Other Study ID Numbers: SeqTranUCBC&IC-001
• First Posted: Feb 8, 2019
• Last Update Posted: Aug 5, 2022
• Last Verified: Aug 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Children's Hospital of Fudan University: Islets of Langerhans Transplantation; Cord Blood Stem Cell Transplantation
• Additional relevant MeSH terms: Diabetes Mellitus; Diabetes Mellitus, Type 1; Immunologic Deficiency Syndromes

Study Results

No Results Posted as of Aug 5, 2022