Study of NTLA-2001 for hereditary ATTR amyloidosis with nerve damage
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study is testing a new gene-silencing treatment called NTLA-2001 for people with hereditary ATTR amyloidosis that causes nerve damage. It works by stopping the body from making a faulty protein that builds up and harms nerves.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of hereditary ATTR amyloidosis with nerve damage (ATTRv-PN).
- You should be able to carry out most daily activities on your own (Karnofsky score of 60 or higher).
- You cannot have any other reason for nerve damage, like diabetes or a different type of amyloidosis.
- You must not have taken any TTR-silencing medicine before (such as patisiran, vutrisiran, or inotersen).
- Your kidney function must be good enough (eGFR of 30 or higher), and you cannot have serious heart failure or liver disease.
- You must be willing to take a daily vitamin A supplement for the entire study.
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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