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

Официальное название

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

Обзор

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.

Подробное описание

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.

Вмешательства

• Процедура 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 t

Первичные конечные точки

• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 3 months]
• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 3 years]

Вторичные конечные точки (12)

• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 6 months]
• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 9 months]
• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 12 months]
• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 15 months]
• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 18 months]
• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 21 months]
• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 24 months]
• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 27 months]
• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 30 months]
• Concentration of serum C-peptide [Срок оценки: from the completion of treatment to 33 months]
• Concentration of serum insulin [Срок оценки: from the completion of treatment to 3 months]
• Concentration of serum insulin [Срок оценки: from the completion of treatment to 6 months]

Критерии участия

Критерии включения

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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Children's Hospital of Fudan University — Шанхай

Публикации

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• 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. PMID 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. PMID 12161590

Идентификаторы

NCT: NCT03835312 · SeqTranUCBC&IC-001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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