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Gene Therapy Trial Report

Summary

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning


NCTID NCT03311503 (View at clinicaltrials.gov)
Description
Development Status Active
Indication X-linked Severe Combined Immunodeficiency (XSCID)
Disease Ontology Term DOID:0060013
Compound Name G2SCID lentiviral vector transduced CD34+ cells
Compound Description rHIV_IL2RGcoG2SCID
Sponsor David Williams
Funder Type Other
Recruitment Status
Enrollment Count 12 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant IL2RG
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type HIV
Editor Type none
Dose 1 Transduced CD34+ cells (minimum dose: 2.5E6 cells/kg)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2017-10-12
Completion Date 2028-01-01
Last Update 2025-12-11

Participation Criteria


Eligible Age 0 Years - 5 Years
Standard Ages Child
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion Criteria: \- 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 Criteria: 1. 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). 1. Mechanical ventilation including continuous positive airway pressure 2. Abnormal liver function defined by AST and ALT \>10 times the upper range of normal OR Bilirubin \>2 mg/dL 3. Shortening fraction on echocardiogram \<25% or ejection fraction \<50% 4. Renal failure defined as glomerular filtration rate \<30 ml/min/1.73 m2 or dialysis dependence 2. Uncontrolled seizure disorder 3. Encephalopathy 4. Documented coexistence of any disorder known to affect DNA repair 5. Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease 6. Patients with evidence of infection with HIV-1 7. 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. 8. 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 \-
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates

Resources/Links