Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Non-Malignant Hematological Disorders in Children
Johns Hopkins All Children's Hospital
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
- Age range
- 0–21 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Severe sickle cell disease (HbSS, HbSC, HbSB0, HbSB+, HbSD, HbSE) with at least one of the following criteria: 1. Cerebrovascular accident lasting longer than 24 hours 2. Impaired neuropsychological function with abnormal brain MRI/MRA 3. Patients with frequent (≥ 3 per year for preceding 2 years) painful vaso-occlusive episodes 4. Recurrent (≥ 3 in lifetime) acute chest syndrome events which have necessitated erythrocyte transfusion therapy 5. Any combination of ≥ 3 acute chest syndrome episodes and vaso-occlusive pain episodes yearly for 3 years and ha…
Interventions
- BiologicalHaploidentical Hematopoietic Cell Transplantation
TCR alpha beta T-cell and CD19 B-cell depleted haploidentical transplantation
Location
- Johns Hopkins All Children's HospitalSt. Petersburg, Florida