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

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age0-21
SponsorJohns Hopkins All Children's Hospital

About this trial

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 criteria

Qualifiers

Cerebrovascular accident lasting longer than 24 hours

Impaired neuropsychological function with abnormal brain MRI/MRA

Patients with frequent (≥ 3 per year for preceding 2 years) painful vaso-occlusive episodes

Recurrent (≥ 3 in lifetime) acute chest syndrome events which have necessitated erythrocyte transfusion therapy

Disqualifiers

Participants who have an HLA-matched sibling who is able and willing to donate bone marrow. Patients with a HLA-matched unrelated donors are not excluded.

Pregnant or breastfeeding females.

Patient has HIV or uncontrolled fungal, bacterial or viral infections.

Patient has received prior solid organ transplant.

Trial design

Treatments tested in this trial

  • Haploidentical Hematopoietic Cell Transplantation

Treatment groups

17 Participants
are divided into 1 treatment group