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RECRUITING
NCT03835312
NA

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

Sponsor: Children's Hospital of Fudan University

View on ClinicalTrials.gov

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.

Official title: Sequential Transplantation of Umbilical Cord Blood Stem Cells and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency Type 1 Diabetes Mellitus

Key Details

Gender

All

Age Range

Any - 18 Years

Study Type

INTERVENTIONAL

Enrollment

50

Start Date

2019-02-20

Completion Date

2030-12-31

Last Updated

2026-03-17

Healthy Volunteers

No

Interventions

PROCEDURE

Sequential transplantation

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.

Locations (1)

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China