safety and efficacity of gene thérapie of the severe combined immunodeficiency (SCID)

A Phase 1/2 Open Label non randomized Study, multicentric, single arm evaluating 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 - ARTEGENE

Status

Active, not recruiting

Phases

Phase 1Phase 2Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-003555-11-FR

Enrollment

Unknown

Registered

2020-03-25

Start date

2021-04-19

Completion date

Unknown

Last updated

2024-10-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis)

Interventions

Product Name: CD34+modifiées et transduites avec le vecteur lentiviral G2ARTE exprimant le DCLRE1CcDNA Pharmaceutical Form: Concentrate and solvent for concentrate for solution for infusion

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patient with a RS-SCID caused by mutation of the Artemis gene and a clinical diagnosis of SCID documented in the medical record - Absence of an HLA genoidentical donor - Age = 1 year - The patient can be treated by gene therapy without delay in case of life threatening infections compromising the short-term prognosis. 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 . - Parental, guardian's patient signed informed consent Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study: - Unwillingness to return for follow-up during the 2 year study and lifelong for off study review - - HIV-1 or 2 or HTLV1 infection French

Design outcomes

Primary

Measure	Time frame
Primary end point(s): (repeat as necessary)26 English Safety of ARTEGENE drug product, is assessed by: • Incidence of transplant related mortality up to 100 days post treatment • Frequency and severity of clinical AEs and laboratory parameters throughout the whole period of the research • Incidence of RCL measured at 3 months The efficacy of successful transduction of CD34+ by the vector and the injection of autologous gene-modified hematopoietic stem cells is evaluated by the assessment of T and B cells reconstitution in Artemis deficient patients after transplantation.;Main Objective: The primary objective of the study consists in 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 in up to 5 Artemis deficient patients.;Secondary Objective: • The clearing of on going infections present before the transplantation • . • The evaluation of the functional performance of this novel lentiviral vector • The evaluation of the molecular characteristics of vector integration.;Timepoint(s) of evaluation of this end point: 3 months	—

Measure

Time frame

Primary end point(s): (repeat as necessary)26 English Safety of ARTEGENE drug product, is assessed by: • Incidence of transplant related mortality up to 100 days post treatment • Frequency and severity of clinical AEs and laboratory parameters throughout the whole period of the research • Incidence of RCL measured at 3 months The efficacy of successful transduction of CD34+ by the vector and the injection of autologous gene-modified hematopoietic stem cells is evaluated by the assessment of T and B cells reconstitution in Artemis deficient patients after transplantation.;Main Objective: The primary objective of the study consists in 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 in up to 5 Artemis deficient patients.;Secondary Objective: • The clearing of on going infections present before the transplantation • . • The evaluation of the functional performance of this novel lentiviral vector • The evaluation of the molecular characteristics of vector integration.;Timepoint(s) of evaluation of this end point: 3 months

—

Secondary

Measure	Time frame
Secondary end point(s): The assessment of the long-term safety and efficacy of treatment with the ARTEGENE drug product;Timepoint(s) of evaluation of this end point: 100 days	—

Countries

France

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026