Status:
RECRUITING
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
Lead Sponsor:
David Williams
Conditions:
Severe Combined Immunodeficiency, X Linked
Gene Therapy
Eligibility:
MALE
Up to 5 years
Phase:
PHASE1
PHASE2
Brief Summary
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...
Detailed Description
This is an open labeled, multi-center, phase I/II, cohort study involving a single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) lentiviral vector G2SCID in up to 10 p...
Eligibility Criteria
Inclusion
- \- 1. Diagnosis of SCID-X1 based on immunophenotype and lack of T cell function (proliferation to PHA \<10% of the lower limit of normal for the laboratory) AND confirmed by a mutation in IL2RG 2. Lack of an HLA identical (A, B, C, DR, DQ) related donor 3. Age 5 years old or younger 4. Signed informed consent 5. Documentation of willingness to follow up for 15 years post-infusion as currently required by the FDA 6. If the patient has previously undergone allogeneic transplant, lack of donor T cell engraftment must be documented.
- 7\. Age at least 8 weeks by the time of busulfan administration
Exclusion
- Patients with an active, therapy-resistant infection. Infections that are known to be highly morbid in SCID patients will be considered active and therapy-resistant if the infectious agent is repeatedly isolated despite a minimum of 2 weeks of appropriate therapy and is associated with significant organ dysfunction (including but not limited to abnormalities listed below).
- Mechanical ventilation including continuous positive airway pressure
- Abnormal liver function defined by AST and ALT \>10 times the upper range of normal OR Bilirubin \>2 mg/dL
- Shortening fraction on echocardiogram \<25% or ejection fraction \<50%
- Renal failure defined as glomerular filtration rate \<30 ml/min/1.73 m2 or dialysis dependence
- Uncontrolled seizure disorder
- Encephalopathy
- Documented coexistence of any disorder known to affect DNA repair
- Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease
- Patients with evidence of infection with HIV-1
- Major (life-threatening) congenital anomalies. Examples of "major (life-threatening) congenital anomalies" include, but are not limited to: unrepaired cyanotic heart disease, hypoplastic lungs, anencephaly or other major central nervous system malformations, other severe non-repairable malformations of the gastrointestinal or genitourinary tracts that significantly impair organ function.
- Other conditions which in the opinion of the P.I. or co-investigators, contra-indicate collection and/or infusion of transduced cells or indicate patient's inability to follow the protocol. These may include for example clinical ineligibility to receive anesthesia, severe deterioriation of clinical condition of the patient after collection of bone marrow but before infusion of transduced cells, or documented refusal or inability of the family to return for scheduled visits. There may be other unforeseen rare circumstances that would result in exclusion of the patient, such as sudden loss of legal guardianship
- \-
Key Trial Info
Start Date :
February 26 2018
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
January 1 2028
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT03311503
Start Date
February 26 2018
End Date
January 1 2028
Last Update
December 11 2025
Active Locations (4)
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1
Mattel Children's Hospital - UCLA
Los Angeles, California, United States, 90095
2
Emory University/Childrens Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
3
Boston Childrens Hospital
Boston, Massachusetts, United States, 02115
4
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229