Gene therapy for ADA-SCID using a modified virus vector
Part of Immune system & allergy clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have classical ADA-SCID confirmed by DNA testing showing a faulty ADA gene
- You have significant T-cell immune problems (for example, low T-cell count or weak T-cell response in lab tests)
- You have had serious infections such as lung inflammation, long-lasting diarrhea needing IV nutrition, herpes/adenovirus/fungal infections, or widespread BCG infection
- Your leukemia-related chromosome tests are normal (no concerning blood/bone marrow changes)
- You have never had a stem cell transplant from another person (no allogeneic transplant)
- You test negative for HIV and are expected to live at least 2 months
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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