Actively Recruiting
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
Led by David Williams · Updated on 2025-12-11
12
Participants Needed
4
Research Sites
513 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol, then followed long-term on a separate long-term follow-up protocol. Enrollment of subjects will be agreed upon by representatives of both sites. Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized. Harvest, cellular manufacturing and infusion will occur at each site using the same SOPs. Key aspects of cellular product characterization will be centralized
CONDITIONS
Official Title
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of SCID-X1 confirmed by immunophenotype, lack of T cell function, and mutation in IL2RG
- No HLA identical related donor available
- Age 5 years old or younger
- Signed informed consent
- Willingness to follow up for 15 years post-infusion
- If previously had allogeneic transplant, must have no donor T cell engraftment
- Age at least 8 weeks at time of busulfan administration
You will not qualify if you...
- Active, therapy-resistant infection causing significant organ dysfunction
- Mechanical ventilation including continuous positive airway pressure
- Abnormal liver function with AST and ALT >10 times normal or bilirubin >2 mg/dL
- Low heart function (shortening fraction <25% or ejection fraction <50%)
- Renal failure with glomerular filtration rate <30 ml/min/1.73 m2 or dialysis dependence
- Uncontrolled seizure disorder
- Encephalopathy
- Disorders affecting DNA repair
- Active malignant disease except EBV-associated lymphoproliferative disease
- HIV-1 infection
- Major life-threatening congenital anomalies impairing organ function
- Other conditions preventing collection/infusion of transduced cells or inability to follow protocol, including refusal or inability to return for visits
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Mattel Children's Hospital - UCLA
Los Angeles, California, United States, 90095
Actively Recruiting
2
Emory University/Childrens Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
Actively Recruiting
3
Boston Childrens Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
4
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
Research Team
C
Colleen Dansereau
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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