Gene therapy for alpha-1 antitrypsin deficiency
Part of Digestive system, Genetic & congenital, Lungs & breathing clinical trials.
This trial tests a new gene therapy that may fix the genetic cause of alpha-1 antitrypsin deficiency (AATD). It might help protect your lungs and liver from damage.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 or older.
- You must have a confirmed diagnosis of AATD with the specific genetic change (Pi*Z) in both genes.
- Your blood level of AAT protein must be below a certain threshold, or you must be willing to stop augmentation therapy for a while.
- You must be willing to avoid alcohol, smoking, and strenuous exercise during the study.
- You must agree to long-term follow-up for 15 years 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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This trial tests a gene therapy for people with alpha-1 antitrypsin deficiency who have mild to moderate lung disease. The aim is to see if the treatment can improve lung health and is safe.
This study tests a one-time gene therapy called BEAM-302 that aims to fix the faulty gene causing alpha-1 antitrypsin deficiency (AATD), a condition that damages the lungs and liver. The goal is to improve AAT levels and slow or stop disease progression.
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.
This study tests a new gene therapy called KB408 for people with alpha-1 antitrypsin deficiency (AATD). It aims to see if KB408 can help your body produce enough of the missing protein to protect your lungs and liver.
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This trial tests a new medicine called TSRA-196 for people with Alpha-1 Antitrypsin Deficiency who have the PiZZ genetic type. The study looks at whether TSRA-196 can help with lung or liver problems related to this condition.
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