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Clinical Trial Summary

The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the severe combined immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis) by transplantation of a single dose of autologous CD34+ cells transduced ex vivo with the G2ARTE lentiviral vector expressing the DCLRE1C cDNA.

Clinical Trial Description


Study Design

Related Conditions & MeSH terms

NCT number NCT05071222
Study type Interventional
Source Assistance Publique - Hôpitaux de Paris
Contact Marina CAVAZZANA, MD, PhD
Phone +33 144495068
Email [email protected]
Status Not yet recruiting
Phase Phase 1/Phase 2
Start date October 2021
Completion date October 2039