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Clinical Trial Report

Gene Therapy Trial Report

Summary

Gene Therapy for X-linked Severe Combined Immunodeficiency


NCTID NCT01410019 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication X-linked Severe Combined Immunodeficiency (XSCID)
Disease Ontology Term DOID:0060013
Compound Name Autologous CD34+ cells transduced with the self-inactivating (SIN) GAMMARETROVIRAL vector pSRS11.EFS.IL2RG.pre
Sponsor Assistance Publique - Hôpitaux de Paris
Funder Type Other
Recruitment Status
Completed
Enrollment Count 5
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 RV
Editor Type
Dose 1 Median dose: 7.58E6 transduced CD34+ cells/kg (n=11)
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2011-06-21
Completion Date 2015-06-16
Last Update 2021-09-28

Participation Criteria


Eligible Age <=12 Months
Standard Ages Child
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 1
Locations France

Regulatory Information


Has US IND
FDA Designations
Recent Updates

Resources/Links