Gene therapy for emphysema caused by alpha-1 antitrypsin deficiency
Part of Digestive system, Genetic & congenital, Lungs & breathing clinical trials.
This trial tests a new treatment called CTX460, which is a type of gene therapy that may correct the genetic defect causing alpha-1 antitrypsin deficiency (AATD) and help slow or improve emphysema. It's designed for adults with severe AATD and emphysema who have not had a liver or lung transplant.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 75 years old.
- You have been diagnosed with alpha-1 antitrypsin deficiency (AATD) with the PiZZ genetic type.
- Your blood level of AAT protein is low (below 11 micromolar) without using augmentation therapy (infusions).
- You have emphysema confirmed by a CT scan, and your lung function (FEV1) is at least 30% of the expected value.
- You weigh at least 50 kg (about 110 pounds).
- You have not smoked or vaped for at least 12 months before the study.
- You are willing to avoid alcohol for 2 weeks before and 2 weeks after the treatment infusion.
- You can use highly effective birth control (if applicable) for up to 6 months after the infusion.
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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