TCR Alpha Beta T-cell Depleted Haploidentical HCT for Non-Malignant Blood Disorders in Children
This study is investigating a new type of stem cell transplant called TCR alpha beta T-cell and CD19 B-cell depleted haploidentical transplantation for children and young adults (ages 0-21) with certain non-cancerous blood disorders like severe sickle cell disease, severe aplastic anemia, or bone marrow failure syndrome. This treatment uses stem cells from a parent or half-matched family member. These cells are specially processed using a device called CliniMACS, which is still being studied. The main goal is to see if the donor stem cells successfully engraft (start working) in the patient's body by Day 100 after the transplant. The study is currently recruiting up to 17 participants, but its overall status is unclear.
- Study design
- This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 17 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary endpoint is measured at Day 100 after transplantation, focusing on successful donor engraftment.
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TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Deepak Chellapandian, MD · PRINCIPAL_INVESTIGATOR · Johns Hopkins All Children's Hospital
Who to contact
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What this trial measures
- Incidence of successful donor engraftmentDay 100 after transplantation
The incidence of engraftment at day 100 will be described based on donor chimerism in the whole blood and or fractions sorted for T-cell and myeloid subsets. The donor chimerism will be scored as autologous reconstitution (\< 5% donor), mixed chimerism (5-49%=low mixed, 50-95%=high mixed), \> 95%=full donor chimerism.