BridgeMD
Tests treatment safety and results for Artemis (DCLRE1C ) Deficient Severe Combined...
Official title 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)
ClinicalTrials.gov ID: NCT05071222
What this study is testing
What is ARTEGENE drug product?
ARTEGENE drug product is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for artemis (dclre1c ) deficient severe combined immunodeficiency.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 47
You may be able to join if
- 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...
- Beneficiary of a social security scheme
You likely can't join if
- 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...
See the full eligibility 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...
- Beneficiary of a social security scheme
- Parental, guardian's patient signed informed consent.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Paris, France
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 47 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Paris, France. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.