Actively Recruiting
Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Haploidentical Hematopoietic Cell Transplantation in Treating Non-Malignant Blood Disorders in Children
Led by Johns Hopkins All Children's Hospital · Updated on 2025-06-24
17
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a new type of haploidentical stem cell transplantation that uses TCR alpha beta and CD19 depleted stem cell grafts from a half-matched family donor to treat children with non-malignant blood disorders such as severe sickle cell disease, thalassemia major, and bone marrow failure syndromes. This phase 2 study aims to learn if this transplantation approach is safe and effective. The investigational process involves a device called CliniMACS to prepare the stem cell grafts. The treatment uses stem cells collected from the donor's peripheral blood or bone marrow, which then undergo a special processing called TCR alpha beta negative selection following a standardized protocol. The donor must be a parent or other half-matched family member with a suitable genetic match. This single-arm study focuses on the TCR alpha beta T-cell depletion method as the experimental intervention. Participants will be closely monitored through various assessments including blood counts, immune recovery, and transplant complications for up to two years after transplantation. The main outcome measured is successful donor engraftment by day 100 post-transplant. Secondary outcomes include survival rates, graft failure, graft-versus-host disease, infections, and immune function. This study requires informed consent and includes regular follow-ups to evaluate safety and effectiveness.
CONDITIONS
Brief Title
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Severe sickle cell disease with significant complications or failure of hydroxyurea treatment
- Thalassemia major with transfusion dependency or genotypic confirmation
- Bone marrow failure syndromes including severe aplastic anemia and autoimmune cytopenias
- Suitable genotypic match of at least 5/10 at key HLA loci with donor
- Adequate organ function: heart, lung, kidney, liver within specified limits
- Karnofsky or Lansky performance score of 50 or above
- Signed informed consent
You will not qualify if you...
- Having an HLA-matched sibling donor available for bone marrow donation
- Pregnancy or breastfeeding
- HIV infection or uncontrolled infections
- Prior solid organ transplant
- Active graft-versus-host disease above grade II or chronic extensive GVHD
- Significant liver disease including cirrhosis or active hepatitis in patients with chronic transfusions
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 100 days post transplantation
Participants receive TCR alpha beta T-cell and CD19 B-cell depleted haploidentical hematopoietic cell transplantation as the active intervention.
Multiple visits during hospitalization and follow-up until Day 100
Duration - Up to 2 years post transplant
Long-term monitoring of overall survival, event-free survival, graft function, complications, and immune reconstitution after transplantation.
Periodic visits for assessments up to 2 years
Trial Site Locations
Total: 1 location
1
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States, 33701
Actively Recruiting
Research Team
J
Jade Hanson, MSN
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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