Gene therapy trial for Artemis SCID using modified blood cells
Part of Genetic & congenital, Hormones & metabolism, Immune system & allergy clinical trials.
This trial tests whether gene therapy can safely and effectively treat children with a specific type of severe combined immunodeficiency (SCID) caused by an Artemis gene problem. It uses the patient’s own stem cells, modifies them to add a missing gene, and then returns them after chemotherapy to help the new cells work.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age up to 47 months (under 4 years old)
- You have SCID from confirmed Artemis (DCLRE1C) gene mutations (even if you still have some leftover function)
- No matching bone marrow donor available, or an unrelated donor can’t be found within 6 weeks of diagnosis
- Your treating team says gene therapy can start without dangerous delay because of a serious, life-threatening infection risk
- You (and a parent/guardian) can agree to long follow-up visits for the first 2 years and beyond
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This early-phase study tests an “autologous” gene therapy, meaning they use the child’s own cells, adjusted with a working gene, to help restore immune function. It is for babies and children with Artemis-deficient SCID, aiming to improve T-cell immunity and reduce the need for lifelong infection risk management.
This trial tests a gene therapy that uses base-edited stem cells to treat X-SCID, a genetic immune disorder. It may help improve immune function and reduce infections in patients who still have low immune cells or severe infections despite previous treatments.
This trial tests a one-time gene therapy approach to help children with X-linked SCID (a serious inherited immune disorder) make better immune cells. It’s for people who don’t have a fully matched sibling donor and need long-term follow-up after treatment.
This trial tests a gene therapy using a lentivirus to give missing immune system instructions in children with X-linked SCID (SCID-X1). It also uses a low dose of targeted chemotherapy (busulfan) to help the new genes work, and it requires long-term follow-up for safety.
This early-stage study tests a one-time lentiviral gene therapy to help children with X-linked severe combined immunodeficiency (SCID-X1) make infection-fighting T cells. It may reduce the need for a matching donor transplant, but it requires long-term follow-up for safety over many years.
This trial tests a gene therapy made to help people with ADA-SCID (an inherited immune condition caused by a faulty ADA gene). The goal is to improve immune function and reduce severe infections by delivering a working ADA gene into the patient’s own cells.
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