Gene therapy for ADA-SCID using blood and a special treatment shot
Part of Genetic & congenital, Immune system & allergy clinical trials.
This Phase 1/2 trial studies a one-time gene therapy approach for children and adults with ADA-SCID, a rare immune deficiency caused by low ADA enzyme activity. The goal is to use the person’s own cells to deliver a corrected gene and help the immune system work better.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your child’s parents/guardians) must sign consent, and the child must assent when required
- Your child must be at least 30 days old
- You must have ADA-SCID confirmed by lab tests showing very low ADA activity or ADA-related genetic changes, or by signs of severe immune deficiency before immune treatment
- You must not be able to use a matched family bone marrow donor for transplant (no eligible HLA-identical sibling/family donor)
- You and your family must be able to follow study rules and stay available for the full clinic visits and follow-up
- If the participant can become pregnant: a negative pregnancy test is required
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 is a follow-up (no new treatment) study for people who previously received a gene therapy made from their own cells for ADA-SCID. It mainly tracks long-term safety and whether a marker showing the gene change can still be found.
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 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.
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 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 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.
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