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) — ARTEGENE  

Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency Clinical Trial

Official title: A Phase 1/2 Open Label Non Randomized Study, Multicentric, Single Arm Evaluating 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

Status Recruiting

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

n/a

Related Conditions & MeSH terms

• Immunologic Deficiency Syndromes
• Severe Combined Immunodeficiency

• NCT number: NCT05071222
• Study type: Interventional
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: Marina CAVAZZANA, MD, PhD
• Phone: +33 144495068
• Email: m.cavazzana@aphp.fr
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: July 19, 2023
• Completion date: July 19, 2041