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Clinical Trial Report

Gene Therapy Trial Report

Summary

Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)


NCTID NCT05071222 (View at clinicaltrials.gov)
Description
Indication Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency
Compound Name EF1a-hArtemis LV (modified EF1a promotor) ARTEGENE
Sponsor Assistance Publique - Hôpitaux de Paris
Funder Type Other
Status
Recruiting
Enrollment Count 5

Therapy Information


Target Gene/Variant DCLRE1C
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 LV
Editor Type none
Dose 1 Transduced CD34+ cells, range: 1.8-3.7E6 CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2021-08-27
Completion Date 2041-07-19
Last Update 2023-11-27

Participation Criteria


Eligible Age <=47 Months
Standard Ages Child
Eligible Sex ALL

Locations


No.of Trial Sites 1
Locations France

Regulatory Information


Has US IND False
Recent Updates

Resources/Links