Creating gene-corrected stem cells for Alpha-1 Antitrypsin Deficiency
Part of Digestive system, Genetic & congenital, Lungs & breathing clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 years or older.
- You must have a confirmed diagnosis of severe Alpha-1 Antitrypsin Deficiency with two specific genetic changes (pathological variants).
- You must not be currently receiving augmentation therapy (infusions of AAT protein).
- You must be willing and able to give informed consent.
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 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.
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 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 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.
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.