NCT05071222 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)
| NCT ID | NCT05071222 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Assistance Publique - Hôpitaux de Paris |
| Condition | Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency |
| Study Type | INTERVENTIONAL |
| Enrollment | 7 participants |
| Start Date | 2023-07-19 |
| Primary Completion | 2041-11-19 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 7 participants in total. It began in 2023-07-19 with a primary completion date of 2041-11-19.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
Inclusion Criteria: * Patient to 47 months * SCID patients with confirmed biallelic mutations in the Artemis (DCLRE1C) gene even in the case of leaky forms characterised by a residual activity * Absence of an HLA genoidentical donor or without rapidly available HLA-compatible unrelated donor (within six weeks of diagnosis) * The patient can be treated by gene therapy without delay in case of active life threatening infections compromising the short-term prognosis and for which the delay in finding a phenoidentical donor is incompatible with the patient's condition of health. Active life threatening infections are defined as: viral respiratory infection, CMV infection, adenovirus infection, disseminated BCGitis or other infections grade ≥ 4 according to CTCAE scale * Beneficiary of a social security scheme * Parental, guardian's patient signed informed consent. Exclusion Criteria * Unwillingness to return for follow-up during the first 2 years study and the long term follow-up * HIV-1 or 2 or HTLV1 infections * Hypersensitivity to G-CSF, busulfan or Fludarabine * Unable to tolerate general anesthesia and/or marrow harvest or peripheral blood stem cell collection (apheresis) or insertion of central venous catheter.
Contact & Investigator
Alessandra MAGNANI, MD, PhD
STUDY DIRECTOR
Department of Biotherapy,LTCG, Necker-Enfants Malades Hospital
Frequently Asked Questions
Who can join the NCT05071222 clinical trial?
This trial is open to participants of all sexes, up to 47 Months, studying Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05071222 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05071222 currently recruiting?
Yes, NCT05071222 is actively recruiting participants. Contact the research team at m.cavazzana@aphp.fr for enrollment information.
Where is the NCT05071222 trial being conducted?
This trial is being conducted at Paris, France.
Who is sponsoring the NCT05071222 clinical trial?
NCT05071222 is sponsored by Assistance Publique - Hôpitaux de Paris. The principal investigator is Alessandra MAGNANI, MD, PhD at Department of Biotherapy,LTCG, Necker-Enfants Malades Hospital. The trial plans to enroll 7 participants.