Haploidentical Transplant for Immune and Metabolic Disorders in Children
This study is testing a new type of stem cell transplant called TCR alpha beta T-cell and CD19 B-cell depleted haploidentical transplantation. This treatment uses stem cells from a parent or half-matched family member, processed with a special device called CliniMACS. The goal is to see if this transplant is safe and works well for children up to 21 years old who have primary immune deficiency disorders (problems with the body's defense system) or inherited metabolic disorders (problems with how the body uses energy). The main way success will be measured is by checking if the donor cells have successfully grown in the patient's body by 100 days after the transplant. The study plans to enroll 17 participants, and its current status is unclear.
- Study design
- This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 17 children and young adults.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- The study will check for successful donor cell growth 100 days after the transplant.
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TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic 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.