Contact SCGE




Clinical Trial Report

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
Indication Severe Combined Immunodeficiency, X Linked, Gene Therapy
Compound Name G2SCID lentiviral vector transduced CD34+ cells (rHIV_IL2RGcoG2SCID)
Sponsor David Williams
Funder Type Other
Status
Recruiting
Enrollment Count 12

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)
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2017-10-12
Completion Date 2028-10-01
Last Update 2025-01-06

Participation Criteria


Eligible Age 0 Years - 5 Years
Standard Ages Child
Eligible Sex MALE

Locations


No.of Trial Sites 4
Locations United States

Regulatory Information


Has US IND True
Recent Updates

Resources/Links