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)

Plain-language summary

This study evaluates a gene therapy (ARTEGENE) in which the DCLRE1C gene is transferred into the patient's own stem cells, in patients with severe combined immunodeficiency (SCID) due to Artemis (DCLRE1C) deficiency. It enrolls children up to 47 months of age with confirmed biallelic mutations in the DCLRE1C gene who lack a suitable matched donor. It is a Phase 1/Phase 2 study and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Recruiting

Sponsor

Assistance Publique - Hôpitaux de Paris

Intervention

ARTEGENE drug product

Start date

7/19/2023

Estimated completion

11/19/2041

Countries

France

Related genes

Locations

• Paris, France

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.