RecruitingPhase 2NCT04414046

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children

Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children

Sponsor

Johns Hopkins All Children's Hospital

Enrollment

17 participants

Start Date

Jul 22, 2020

Study Type

INTERVENTIONAL

Conditions

Metabolic Disease Primary Immune Deficiency Disorders

Summary

This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.

Eligibility

Max Age: 21 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called Haploidentical Hematopoietic Cell Transplantation for people with metabolic disease and primary immune deficiency disorders. The study is currently recruiting participants at 1 location. People eligible for this study include up to age 21 Years.

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.