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

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.

NCT Number	NCT04414046
Lead Sponsor	Johns Hopkins All Children's Hospital
Conditions	Primary Immune Deficiency Disorders, Metabolic Disease
Enrollment	17 participants
Start Date	2020-07-22
Primary Completion	2027-06-30 (estimated)
Study Completion	2027-06-30 (estimated)
Updated on ClinicalTrials.gov	2026-07-01