Actively Recruiting
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic 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 Primary Immunodeficiency and Inherited Metabolic Disorders in Children
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient has a primary immune deficiency or immune dysregulatory disorder, including but not limited to disorders of phagocytes, defects of cellular and humoral immunity, or immune dysregulation syndromes
- Patient has histiocytic disorders such as familial or secondary hemophagocytic lymphohistiocytosis or multisystem refractory Langerhans cell histiocytosis
- Patient has metabolic disorders that may improve or stabilize after stem cell transplantation
- Patient has a suitable genotypic identical match of 5/10 at specific HLA genetic loci with the donor
- Patient has adequate organ function: cardiac (LVEF ≥ 40% or SF ≥ 26%), pulmonary (DLCO ≥ 40% predicted or pulse oximetry ≥ 92%), renal (Creatinine clearance or GFR > 50 mL/min/1.73 m2), hepatic (bilirubin < 2.0 x ULN, AST and ALT < 5.0 x ULN)
- Patient has a Karnofsky or Lansky performance score of at least 50
- Signed written informed consent is provided
You will not qualify if you...
- Patient has an HLA-matched sibling donor able and willing to donate bone marrow
- Patient is pregnant or breastfeeding
- Patient has HIV or uncontrolled fungal, bacterial, or viral infections
- Patient has received a prior solid organ transplant
- Patient has active graft-versus-host disease greater than grade II or chronic extensive graft-versus-host disease from a previous allograft at inclusion
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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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