Weekly treatment for alpha-1 related lung disease
Part of Digestive system, Genetic & congenital, Lungs & breathing clinical trials.
This study tests a weekly treatment for adults with alpha-1 antitrypsin deficiency (AATD) who have emphysema. The treatment aims to slow down the loss of lung density over three years, which may help preserve lung function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 65 years old.
- You have a confirmed diagnosis of emphysema related to alpha-1 antitrypsin deficiency (AATD).
- You have a specific genetic type (PiZZ, PiZ(null), or Pi(null/null)) and low AAT levels in your blood.
- You have not had a recent lung flare-up or acute illness in the last 6 weeks.
- You have not had gene therapy for AATD before.
- You do not have liver disease caused by AATD.
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 study looks at how alpha-1 antitrypsin deficiency affects the liver in adults. It aims to learn more about the liver problems caused by this condition and may help find better ways to manage them.
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.
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 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 study checks how safe a prescribed blood protein medicine (alpha-1 proteinase inhibitor) is over the long term in people with Alpha-1 antitrypsin deficiency. You may join if you already finished an earlier study or if your lung function has been declining.
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).
Hear when a new Alpha1-Antitrypsin Deficiency trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.