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
Last Updated: 2023-11-27
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
5 participants
INTERVENTIONAL
2023-07-19
2041-07-19
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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ARTEGENE drug product
Autologous purified CD34+ cells transduced with a self-inactivated lentiviral vector, expressing the DCLRE1C gene (alias Artemis)
ARTEGENE drug product
Each patient will receive a single intravenous infusion of ARTEGENE drug product at D0.
Interventions
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ARTEGENE drug product
Each patient will receive a single intravenous infusion of ARTEGENE drug product at D0.
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
47 Months
ALL
No
Sponsors
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Assistance Publique - Hôpitaux de Paris
OTHER
Responsible Party
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Principal Investigators
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Alessandra MAGNANI, MD, PhD
Role: STUDY_DIRECTOR
Department of Biotherapy,LTCG, Necker-Enfants Malades Hospital
Chantal Lagresle-Peyrou, MD
Role: STUDY_DIRECTOR
IMAGINE
Locations
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Department of Pediatric Immunology, Hematology and Rheumatology UIHR, Necker-Enfants Malades Hospital
Paris, , France
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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2019-003555-11
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
D20180302
Identifier Type: -
Identifier Source: org_study_id