Testing tafamidis for transthyretin amyloid polyneuropathy
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests whether the drug tafamidis can help people with a specific type of nerve damage caused by abnormal protein buildup. It aims to slow or improve symptoms in adults with this condition.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 80 years old.
- You have a confirmed buildup of abnormal protein (amyloid) in your body, proven by a biopsy within the last 5 years.
- You have a genetic mutation known to cause transthyretin amyloid polyneuropathy, confirmed by a test within the last 5 years.
- You have symptoms of nerve damage (peripheral neuropathy) that started before the screening visit.
- Your overall health and ability to do daily tasks is at least 50% on the Karnofsky scale (able to care for yourself with some help).
- Your disease is in the early stage (stage 1), meaning symptoms are mild and mainly in the limbs.
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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Other trials that look related to this one.
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This study tests a new treatment, NTLA-2001, for people with a type of heart disease called transthyretin amyloidosis with cardiomyopathy. It aims to see if the medicine can help improve heart function and quality of life.
This study follows patients with a specific type of heart amyloidosis (wild-type ATTR) who are taking or could start the drug tafamidis. It aims to learn more about how the condition and treatment affect people over time.
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This trial tests a daily pill called acoramidis to see if it can prevent ATTR amyloidosis in people who carry a gene mutation but have no symptoms yet. It aims to stop the disease before it starts.
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.
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