Testing BEAM-302 for alpha-1 antitrypsin deficiency
Part of Digestive system, Genetic & congenital, Lungs & breathing clinical trials.
This trial tests a new treatment called BEAM-302, which aims to correct the genetic defect that causes alpha-1 antitrypsin deficiency. It may help prevent further lung and liver damage in people with the PiZZ mutation.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 70 years old.
- You have been diagnosed with alpha-1 antitrypsin deficiency and have the PiZZ genetic mutation.
- Your blood level of alpha-1 antitrypsin is below a certain threshold (less than 11 micromoles per liter).
- Your lung function is at least 40% of the expected value (FEV1) and shows signs of COPD (low FEV1/FVC ratio).
- You have emphysema seen on a CT scan or a low lung diffusion capacity (DLCO) on a breathing test.
- You do not have severe liver disease, a history of liver cirrhosis, or a body mass index over 30.
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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