Actively Recruiting
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)
Led by Assistance Publique - Hôpitaux de Paris · Updated on 2026-03-27
7
Participants Needed
1
Research Sites
956 weeks
Total Duration
On this page
Sponsors
A
Assistance Publique - Hôpitaux de Paris
Lead Sponsor
U
URC-CIC Paris Descartes Necker Cochin
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient age up to 47 months
- Confirmed SCID with biallelic mutations in the Artemis (DCLRE1C) gene, including leaky forms
- Absence of an HLA genoidentical donor or no rapidly available compatible unrelated donor within 6 weeks
- Patient can receive gene therapy immediately despite active life-threatening infections
- Beneficiary of a social security scheme
- Signed informed consent from parent or guardian
You will not qualify if you...
- Unwillingness to return for follow-up during the first 2 years and long-term monitoring
- Infection with HIV-1, HIV-2, or HTLV1
- Hypersensitivity to G-CSF, busulfan, or fludarabine
- Unable to tolerate general anesthesia, marrow harvest, peripheral blood stem cell collection, or central venous catheter insertion
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Department of Pediatric Immunology, Hematology and Rheumatology UIHR, Necker-Enfants Malades Hospital
Paris, France, 75015
Actively Recruiting
Research Team
M
Marina CAVAZZANA, MD, PhD
CONTACT
J
Jinmi BAEK, Master
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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