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

Not Applicable

Recruiting

• Conditions: Diabetes Mellitus, Type 1; Immunologic Deficiency Syndromes

• Registration Number: NCT03835312
• Lead Sponsor: Children's Hospital of Fudan University

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.

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 Timeline

• Study First Submitted: 2019-01-31
• Study First Submitted QC: 2019-02-06
• Study First Posted: 2019-02-08
• Study Start: 2019-02-20
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-17
• Last Update Posted: 2024-10-21
• Primary Completion: 2025-12-31
• Study Completion: 2025-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Concentration of serum C-peptide	from the completion of treatment to 3 years	Islet function (concentration of serum C-peptide)

Secondary Outcome Measures

Name	Time	Method
occurrence of infection (number of infections)	from the completion of treatment to 36 months	occurrence of infection (number of infections)
Concentration of blood immunoglobulin	from the completion of treatment to 36 months	Concentration of blood immunoglobulin
Body weight	from the completion of treatment to 36 months	Body weight
Concentration of serum insulin	from the completion of treatment to 36 months	Concentration of serum insulin
Concentration of T lymphocyte subsets	from the completion of treatment to 36 months	Concentration of T lymphocyte subsets
Concentraion of interleukin-2	from the completion of treatment to 36 months	Cytokines (concentraion of interleukin-2)
Body height	from the completion of treatment to 36 months	Body height
Tanner stage	from the completion of treatment to 36 months	Puberty change (Tanner stage)
Occurrence of graft versus host disease	from the completion of treatment to 36 months	Occurrence of graft versus host disease
Concentration of serum C-peptide	from the completion of treatment to 33 months	Islet function (concentration of serum C-peptide)
Fast blood glucose level	from the completion of treatment to 36 months	Fast blood glucose level
HbA1c level	from the completion of treatment to 36 months	HbA1c level

Trial Locations

Locations (1)

Children's Hospital of Fudan University; 🇨🇳 Shanghai, Shanghai, China