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

Primary Purpose

Diabetes Mellitus, Type 1, Immunologic Deficiency Syndromes

Status
Recruiting
Phase
Not Applicable
Locations
China
Study Type
Interventional
Intervention
Sequential transplantation
Sponsored by
Children's Hospital of Fudan University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Diabetes Mellitus, Type 1 focused on measuring Islets of Langerhans Transplantation, Cord Blood Stem Cell Transplantation

Eligibility Criteria

undefined - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's Hospital of Fudan UniversityRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Interventional

Arm Description

Sequential transplantation of umbilical cord blood stem cells and islet cells

Outcomes

Primary Outcome Measures

Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)

Secondary Outcome Measures

Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Concentration of serum C-peptide
Islet function (concentration of serum C-peptide)
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Concentration of serum insulin
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
Fast blood glucose level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
HbA1c level
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
occurrence of infection (number of infections)
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of blood immunoglobulin
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentration of T lymphocyte subsets
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Concentraion of interleukin-2
Cytokines (concentraion of interleukin-2)
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body height
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Body weight
Tanner stage
Puberty change (Tanner stage)
Tanner stage
Puberty change (Tanner stage)
Tanner stage
Puberty change (Tanner stage)
Tanner stage
Puberty change (Tanner stage)
Tanner stage
Puberty change (Tanner stage)
Tanner stage
Puberty change (Tanner stage)
Tanner stage
Puberty change (Tanner stage)
Tanner stage
Puberty change (Tanner stage)
Tanner stage
Puberty change (Tanner stage)
Tanner stage
Puberty change (Tanner stage)
Tanner stage
Puberty change (Tanner stage)
Tanner stage
Puberty change (Tanner stage)
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease
Occurrence of graft versus host disease

Full Information

First Posted
January 31, 2019
Last Updated
August 29, 2023
Sponsor
Children's Hospital of Fudan University
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1. Study Identification

Unique Protocol Identification Number
NCT03835312
Brief Title
Sequential Transplantation of UCBSCs and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency T1DM
Official Title
Sequential Transplantation of Umbilical Cord Blood Stem Cells and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency Type 1 Diabetes Mellitus
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 20, 2019 (Actual)
Primary Completion Date
December 31, 2025 (Anticipated)
Study Completion Date
December 31, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital of Fudan University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Diabetes Mellitus, Type 1, Immunologic Deficiency Syndromes
Keywords
Islets of Langerhans Transplantation, Cord Blood Stem Cell Transplantation

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Interventional
Arm Type
Experimental
Arm Description
Sequential transplantation of umbilical cord blood stem cells and islet cells
Intervention Type
Procedure
Intervention Name(s)
Sequential transplantation
Intervention Description
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.
Primary Outcome Measure Information:
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 3 months
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 3 years
Secondary Outcome Measure Information:
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 6 months
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 9 months
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 12 months
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 15 months
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 18 months
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 21 months
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 24 months
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 27 months
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 30 months
Title
Concentration of serum C-peptide
Description
Islet function (concentration of serum C-peptide)
Time Frame
from the completion of treatment to 33 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 3 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 6 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 9 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 12 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 15 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 18 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 21 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 24 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 27 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 30 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 33 months
Title
Concentration of serum insulin
Description
Concentration of serum insulin
Time Frame
from the completion of treatment to 36 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 3 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 6 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 9 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 12 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 15 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 18 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 21 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 24 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 27 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 30 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 33 months
Title
Fast blood glucose level
Description
Fast blood glucose level
Time Frame
from the completion of treatment to 36 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 3 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 6 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 9 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 12 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 15 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 18 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 21 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 24 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 27 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 30 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 33 months
Title
HbA1c level
Description
HbA1c level
Time Frame
from the completion of treatment to 36 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 3 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 6 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 9 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 12 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 15 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 18 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 21 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 24 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 27 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 30 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 33 months
Title
occurrence of infection (number of infections)
Description
occurrence of infection (number of infections)
Time Frame
from the completion of treatment to 36 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 3 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 6 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 9 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 12 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 15 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 18 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 21 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 24 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 27 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 30 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 33 months
Title
Concentration of blood immunoglobulin
Description
Concentration of blood immunoglobulin
Time Frame
from the completion of treatment to 36 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 3 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 6 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 9 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 12 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 15 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 18 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 21 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 24 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 27 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 30 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 33 months
Title
Concentration of T lymphocyte subsets
Description
Concentration of T lymphocyte subsets
Time Frame
from the completion of treatment to 36 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 3 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 6 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 9 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 12 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 15 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 18 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 21 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 24 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 27 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 30 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 33 months
Title
Concentraion of interleukin-2
Description
Cytokines (concentraion of interleukin-2)
Time Frame
from the completion of treatment to 36 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 3 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 6 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 9 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 12 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 15 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 18 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 21 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 24 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 27 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 30 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 33 months
Title
Body height
Description
Body height
Time Frame
from the completion of treatment to 36 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 3 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 6 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 9 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 12 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 15 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 18 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 21 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 24 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 27 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 30 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 33 months
Title
Body weight
Description
Body weight
Time Frame
from the completion of treatment to 36 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 3 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 6 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 9 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 12 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 15 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 18 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 21 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 24 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 27 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 30 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 33 months
Title
Tanner stage
Description
Puberty change (Tanner stage)
Time Frame
from the completion of treatment to 36 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 3 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 6 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 9 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 12 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 15 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 18 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 21 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 24 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 27 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 30 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 33 months
Title
Occurrence of graft versus host disease
Description
Occurrence of graft versus host disease
Time Frame
from the completion of treatment to 36 months

10. Eligibility

Sex
All
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Luo Feihong
Phone
+862164931226
Ext
+862164931226
Email
luofh@fudan.edu.cn
First Name & Middle Initial & Last Name or Official Title & Degree
Xu Zhenran
Phone
+862164931226
Ext
+862164931226
Email
xu_zhenran@163.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Luo Feihong
Organizational Affiliation
Children's Hospital of Fudan University
Official's Role
Study Chair
Facility Information:
Facility Name
Children's Hospital of Fudan University
City
Shanghai
State/Province
Shanghai
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Luo Feihong
Phone
+862164931226
Ext
+862164931226
Email
luofh@fudan.edu.cn
First Name & Middle Initial & Last Name & Degree
Xu Zhenran
Phone
+862164931226
Ext
+862164931226
Email
xu_zhenran@fudan.edu.cn

12. IPD Sharing Statement

Plan to Share IPD
No
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Sequential Transplantation of UCBSCs and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency T1DM

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