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Clinical Trial Report

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 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
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
Dose 1 Dose: 30E6 transduced CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

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

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