Clinical Trial

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

Recruiting Phase 2
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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.
Protocol Amendment History 8 amendments
This ClinicalTrials.gov record has been amended 8 times since 2020-04-21; most recent amendment 2026-06-29.
Status change: Not Yet Recruiting → Recruiting 2020-07-23
Trial Details
NCT Number NCT04356469
Lead Sponsor Johns Hopkins All Children's Hospital
Conditions Hemoglobinopathy (Disorder), Severe Aplastic Anemia, Bone Marrow Failure Syndrome
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