Status: Recruiting
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.
Participants needed: 17
Trial details
Phase: Phase 2Age: Up to 21Biological sex: AllType: InterventionalSponsor: Johns Hopkins All Children's HospitalUpdated: Jul 1, 2026Locations: 1
Eligibility criteria
Histiocytic disorders including hemophagocytic lymphohistiocytosis (familial HLH... [+8]
Participants who have an HLA-matched sibling who is able and willing to donate b... [+4]