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

NCT03835312 | Recruiting

• 1 other identifier: SeqTranUCBC&IC-001
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

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.

Conditions

Diabetes Mellitus, Type 1; Immunologic Deficiency Syndromes

Keywords

Islets of Langerhans Transplantation; Cord Blood Stem Cell Transplantation

Outcome Measures

Primary Outcomes (2)

• Concentration of serum C-peptide

  Islet function (concentration of serum C-peptide)

  from the completion of treatment to 3 months

• Concentration of serum C-peptide

  Islet function (concentration of serum C-peptide)

  from the completion of treatment to 3 years

Secondary Outcomes (142)

• Concentration of serum C-peptide

  from the completion of treatment to 6 months

• Concentration of serum C-peptide

  from the completion of treatment to 9 months

• Concentration of serum C-peptide

  from the completion of treatment to 12 months

• Concentration of serum C-peptide

  from the completion of treatment to 15 months

• Concentration of serum C-peptide

  from the completion of treatment to 18 months

Study Arms (1)

Interventional

EXPERIMENTAL

Sequential transplantation of umbilical cord blood stem cells and islet cells

Procedure: Sequential transplantation

Interventions

Sequential transplantation PROCEDURE

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.

Interventional

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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.

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China

RECRUITING

Related Publications (17)

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MeSH Terms

Conditions

Diabetes Mellitus, Type 1; Immunologic Deficiency Syndromes

Condition Hierarchy (Ancestors)

Diabetes Mellitus; Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Luo Feihong

  Children's Hospital of Fudan University

  STUDY CHAIR

Central Study Contacts

Luo Feihong

CONTACT

+862164931226; luofh@fudan.edu.cn

Xu Zhenran

CONTACT

+862164931226; xu_zhenran@163.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 31, 2019
• First Posted: February 8, 2019
• Study Start: February 20, 2019
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2030
• Last Updated: March 17, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)