Autologous CD34+ haematopoietic stem and progenitor cells transduced with a lentiviral vector containing the human DCLRE1C gene for Severe combined immunodeficiency (SCID) due to biallelic DCLRE1C (Artemis) mutation

Inclusion criteria

• {"criterion_text":"-Patient up to 47 months of age\n-SCID patients with confirmed biallelic mutations in the Artemis (DCLRE1C) gene even in the case of leaky forms characterised by a residual activity\n-Absence of an HLA genoidentical donor or without rapidly available HLA-compatible unrelated donor (within six weeks of diagnosis)\n-The patient can be treated by gene therapy without delay in case of 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\n-Beneficiary of a social security scheme\n-Parental, guardian’s patient signed informed consent"}

Exclusion criteria

• {"criterion_text":"-Unwillingness to return for follow-up during the first 2 years study and the long term follow-up\n-HIV-1 or 2 or HTLV1 infections.\n-Hypersensitivity to G-CSF, busulfan or fludarabine\n-Unable to tolerate general anesthesia and/or marrow harvest or peripheral blood stem cell collection (apheresis) or insertion of central venous catheter"}

Primary endpoints

• {"endpoint_text":"-Safety and efficacy of ARTEGENE drug product","definition_or_measurement_approach":"Assessing the initial safety and efficacy of treatment with ARTEGENE drug product, including the mobilization procedure, conditioning regimen and transplantation with ARTEGENE lentiviral vector gene modified autologous hematopoietic stem cells in up to 5 Artemis deficient patients."}

Secondary endpoints

• {"endpoint_text":"-End of ongoing infection before the transplantation\n-Kinetics of immune reconstitution\n-Adverse event will be measured using CTCAE","definition_or_measurement_approach":"End of ongoing infection before transplantation (as stated); kinetics of immune reconstitution (as stated); adverse events measured using CTCAE."}

France

Earliest CTIS Part Ii Submission Date

20-09-2024

Latest Decision Or Authorization Date

27-09-2024

Processing Time Days

7

Number Of Sites

4

Number Of Participants

7

Primary sponsor

Full Name

Assistance Publique Hopitaux De Paris

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France