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)

Assistance Publique - Hôpitaux de Paris

Status and phase

Enrolling

Phase 2

Phase 1

Conditions

Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency

Treatments

Genetic: ARTEGENE drug product

Study type

Interventional

Funder types

Other

Identifiers

NCT05071222

2019-003555-11 (EudraCT Number)

D20180302

Details and patient eligibility

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.

Enrollment

5 estimated patients

Sex

All

Ages

Under 47 months old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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.