Sequential Transplantation of UCBSCs and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency T1DM
Recruiting now · Not applicable
Conditions studied: Diabetes Mellitus, Type 1, Immunologic Deficiency Syndromes
In brief
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.
Key facts
- Study ID
- NCT03835312
- Run by
- Children's Hospital of Fudan University
- People needed
- 50
- Starts
- 2019-02-20
- Expected to finish
- 2030-12-31
- Last updated by the study team
- 2026-03-17
Who can join
Age: any, up to 18. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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.
Where it is running
- Children's Hospital of Fudan University — Shanghai, Shanghai Municipality, China (enrolling)
Full record on ClinicalTrials.gov
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