Study of YOLT-201 for transthyretin amyloidosis with nerve or heart damage
Part of Brain & nervous system, Heart & circulation clinical trials.
This trial tests an experimental gene-editing medicine called YOLT-201 for people with a type of amyloidosis that damages nerves or the heart. It aims to see if one or two doses can safely stop the disease from getting worse.
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 TTR gene mutation from genetic testing.
- Your weight is between 88 and 198 pounds (40 to 90 kg).
- You have either nerve damage (ATTR-PN) or heart damage (ATTR-CM) from the disease, with specific test results showing moderate to severe symptoms.
- You have not been able to get or benefit from approved treatments like tafamidis, patisiran, or inotersen, or your disease has worsened despite them.
- You are willing to avoid alcohol and to use birth control if needed, and you agree to stop other ATTR treatments during the study.
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 trial tests a new intravenous medicine called YOLT-201 for people with a heart condition caused by buildup of a protein called transthyretin (ATTR-CM). The goal is to see if it can slow or improve the disease.
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 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.
This study is testing an experimental treatment called YOLT-204 for people with sickle cell disease (SCD) or beta-thalassemia. The goal is to see if YOLT-204 is safe and effective, potentially reducing the need for blood transfusions and improving symptoms.
This trial tests a new gene therapy called YOLT-101 for people with familial hypercholesterolemia, a genetic condition that causes very high cholesterol. The treatment aims to lower cholesterol levels and reduce heart risks.
This study tests an experimental drug called YOLT-204 to see if it is safe and can help people with transfusion-dependent beta-thalassemia need fewer blood transfusions. It is for adults aged 18 to 35 who have a specific type of thalassemia and are otherwise healthy enough for the trial.
Hear when a new Transthyretin Amyloidosis Polyneuropathy trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.