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Gene Therapy Trial Report

Summary

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)


NCTID NCT05071222 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency
Disease Ontology Term DOID:0090012
Compound Name ARTEGENE
Compound Description EF1a-hArtemis LV
Sponsor Assistance Publique - Hôpitaux de Paris
Funder Type Other
Recruitment Status
Enrollment Count 5 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant DCLRE1C
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type LV
Editor Type none
Dose 1 Transduced CD34+ cells, range: 1.8-3.7E6 CD34+ cells/kg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2021-08-27
Completion Date 2041-07-19
Last Update 2023-11-27

Participation Criteria


Eligible Age <=47 Months
Standard Ages Child
Sexes Eligible for Study ALL
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations France

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links