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)

Recruiting Phase 1 Phase 2 Interventional Study
Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
Up to 47 Months
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
7 (estimated)
Sponsor
Assistance Publique - Hôpitaux de Paris · Other
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About This Trial

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.

Trial Locations
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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 (withi…
Contacts

Marina CAVAZZANA, MD, PhD

+33 144495068

m.cavazzana@aphp.fr

CONTACT

Jinmi BAEK, Master

+33 1 42 19 28 49

jinmi.baek@aphp.fr

CONTACT