Actively Recruiting
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
Led by Johns Hopkins All Children's Hospital · Updated on 2025-06-24
17
Participants Needed
1
Research Sites
309 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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...
- Diagnosis of severe sickle cell disease with specific complications such as stroke, neuropsychological impairment, frequent pain episodes, or recurrent acute chest syndrome
- Diagnosis of thalassemia major with transfusion dependency or genetic confirmation
- Diagnosis of bone marrow failure syndromes or autoimmune cytopenias refractory to conventional treatments
- Suitable genotypic match with donor at specific HLA genetic loci
- Adequate organ function including heart, lung, kidney, and liver as defined by clinical criteria
- Karnofsky or Lansky performance score of 50 or higher
- Signed written informed consent
You will not qualify if you...
- Presence of an HLA-matched sibling donor able and willing to donate bone marrow
- Pregnant or breastfeeding females
- HIV infection or uncontrolled fungal, bacterial, or viral infections
- Prior solid organ transplant
- Active graft versus host disease (GVHD) grade greater than II or chronic extensive GVHD from previous transplant
- Liver disease including cirrhosis, extensive fibrosis, or active hepatitis in patients with chronic transfusion history and elevated ferritin levels
AI-Screening
AI-Powered Screening
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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
CONTACT
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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