ARTEGENE - 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

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-513652-15-00

Acronym

D20180302

Enrollment

Registered

2024-09-27

Start date

2023-07-19

Completion date

Unknown

Last updated

2025-08-13

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

Conditions

Severe Combined Immuno-Deficiency (SCID) caused by biallelic mutations in the Artemis gene (DCLRE1C)

Brief summary

Safety and efficacy of ARTEGENE drug product

Detailed description

End of ongoing infection before the transplantation, Kinetics of immune reconstitution, Adverse event will be measured using CTCAE

Interventions

DRUGARTEGENE

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Safety and efficacy of ARTEGENE drug product	—

Secondary

Measure	Time frame
End of ongoing infection before the transplantation, Kinetics of immune reconstitution, Adverse event will be measured using CTCAE	—

Countries

France

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026