Gene therapy for alpha-1 antitrypsin deficiency
Part of Digestive system, Genetic & congenital, Lungs & breathing clinical trials.
This trial tests a gene therapy to fix the genetic cause of alpha-1 antitrypsin deficiency, aiming to stop or slow emphysema. It is for people with the most common harmful gene variants who have mild to moderate lung damage and are otherwise healthy enough to take steroids safely.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a specific gene type (ZZ or Z null) that causes alpha-1 antitrypsin deficiency.
- You must have emphysema (damaged air sacs) seen on a lung CT scan.
- Your lung function should be mildly to moderately reduced, not too severe.
- You cannot be using extra oxygen at home or during the day.
- You must not currently be taking steroids for another condition, and you must be able to take steroids for the trial.
- You cannot have had a heart attack or cancer in the last 5 years (except for certain skin cancers).
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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This trial tests whether a new weekly injection form of alpha-1 antitrypsin replacement therapy (a protein your body lacks) works as well as the standard IV infusion you may currently receive. The goal is to offer a more convenient treatment option for people with alpha-1 antitrypsin deficiency (a genetic lung condition).
This trial uses your own cells to create stem cells (iPSCs) that can be genetically corrected to treat severe Alpha-1 Antitrypsin Deficiency. It is an early study exploring a potential cure.
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