RecruitingNot ApplicableNCT03835312

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

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


Sponsor

Children's Hospital of Fudan University

Enrollment

50 participants

Start Date

Feb 20, 2019

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Max Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a treatment that combines two types of transplants — donated cord blood stem cells and donated insulin-producing islet cells from the pancreas — to help children with Type 1 diabetes who also have a rare genetic immune system condition that causes damage to multiple organs. **You may be eligible if...** - You are a child diagnosed with Type 1 diabetes AND a genetic immune deficiency (confirmed by gene testing) that is causing damage to multiple organs (such as the gut, kidneys, liver, or joints) - Your symptoms keep returning despite standard medical treatment - You do not have a well-established, effective treatment option currently available **You may NOT be eligible if...** - A proven, effective treatment already exists for your condition - You are HIV positive, or have active hepatitis B or C - You currently have an active infection - You have an active malignant (cancerous) tumor - You have another life-threatening disease alongside this one Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

PROCEDURESequential 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

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT03835312


Related Trials