Status:
RECRUITING
Sequential Transplantation of UCBSCs and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency T1DM
Lead Sponsor:
Children's Hospital of Fudan University
Conditions:
Diabetes Mellitus, Type 1
Immunologic Deficiency Syndromes
Eligibility:
All Genders
Up to 18 years
Phase:
NA
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 ...
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, hemo...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
February 20 2019
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2025
Estimated Enrollment :
50 Patients enrolled
Trial Details
Trial ID
NCT03835312
Start Date
February 20 2019
End Date
December 31 2025
Last Update
October 21 2024
Active Locations (1)
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1
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China