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Gene Therapy Trial Report

Summary

Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)


NCTID NCT00028236 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication X-linked Severe Combined Immunodeficiency (XSCID)
Disease Ontology Term DOID:0060013
Compound Name GALV MFGS-gc retrovirus
Sponsor National Institute of Allergy and Infectious Diseases (NIAID)
Funder Type NIH
Recruitment Status
Completed
Enrollment Count 3 (ACTUAL)
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 MFGS/GALV
Editor Type none
Dose 1 Dose: 30E6 transduced CD34+ cells/kg (n=3)

Study Record Dates


Current Stage Phase1
Submit Date 2001-12-17
Completion Date 2011-07-25
Last Update 2017-07-02

Participation Criteria


Eligible Age 18 Months - 20 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL
Eligibility Criteria
* INCLUSION CRITERIA: Patients must have XSCID as defined by either a deleterious mutation in IL2RG, the absence of or less than 5% of normal detectable gc protein, or evidence of functionally defective gc protein. Patients must be between 1.5 and 20 years of age. Patients must weigh at least 12 kg. Patients will have evidence of combined B-cell and T-cell immune deficiency over at least a 6 month period despite previous allogeneic BMT at least 12 months prior to study entry. T-cell immune deficiency is defined as one or more of the following: Total T-cell count less than 500/ul; less than 50% of normal value for in vitro mitogen stimulation; or absent proliferation in vitro to antigens. B-cell immune deficiency is defined as one or more of the following: IgM, IgA or IgE values which are 2 or more standard deviations below the established value for normal, IgG values falling to less than 30% of normal during unintended interruptions or delay in the periodic administration of IVIG; or documented failure to respond to a specific antigen challenge. Patients must have less than or equal to 3% of their mobilized CD34+ cells deriving from their allogeneic bone marrow donor. Willingness to remain hospitalized for several days to several weeks. Have a primary care physician at home. Consent to permit blood and/or tissue samples for storage. EXCLUSION CRITERIA: Any current or preexisting hematologic malignancy. Current treatment with any chemotherapeutic agent. Current treatment with any immunosuppressive agent, excluding corticosteroids. Documented HIV-1 infection. Documented Hepatitis B infection. Childhood malignancy (occurring before 18 years of age) in the patient or a first degree relative, or known genotype of the subject conferring a predisposition to cancer.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Enrolled 3/8 planned patients

Resources/Links