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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)

NCT05071222 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2026-03-27

No results posted yet for this study

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.

Conditions

  • Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency

Interventions

GENETIC

ARTEGENE drug product

Each patient will receive a single intravenous infusion of ARTEGENE drug product at D0.

Sponsors & Collaborators

  • URC-CIC Paris Descartes Necker Cochin

    collaborator OTHER

  • Assistance Publique - Hôpitaux de Paris

    lead OTHER

Principal Investigators

  • Alessandra MAGNANI, MD, PhD · Department of Biotherapy,LTCG, Necker-Enfants Malades Hospital

  • Chantal Lagresle-Peyrou, MD · Imagine

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
47 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-07-19
Primary Completion
2041-11-19
Completion
2041-11-19

Countries

  • France

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05071222 on ClinicalTrials.gov