Follow-up study for people treated with gene therapy for ADA-SCID
Part of Genetic & congenital, Hormones & metabolism, Immune system & allergy clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You previously received ADA-SCID gene therapy using your own cells (EFS-ADA LV/OTL-101 program).
- Your study doctor can still detect a lasting “gene marking” signal in you.
- You (or your parent/legal guardian) can understand and sign informed consent.
- This is observational, meaning no extra study treatment is added.
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 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 study monitors the long-term health of people with Glycogen Storage Disease Type Ia who have already received the gene therapy DTX401, either in a previous clinical trial or after it was approved. The goal is to gather more information about their condition over time.
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 study is a long-term follow-up for people who already received gene therapy for sickle cell disease in a specific sponsor’s clinical trial. It helps researchers watch for long-term safety and outcomes over time.
This study follows people long-term after receiving a specific gene-cell treatment called AGT103-T. It helps researchers check long-term safety and outcomes, which can guide future care for others.
This trial is a follow-up study testing whether AVTX-803 continues to be safe and effective for people with Leukocyte Adhesion Deficiency type II (LAD II). If you already took part in an earlier AVTX-803 LAD II study, this may be the next step while researchers monitor benefits and side effects.
Hear when a new Severe Combined Immunodeficiency (SCID) trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.