Gene therapy trial for Friedreich ataxia heart disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This trial tests a one-time gene therapy called LX2006 for heart problems caused by Friedreich ataxia. The goal is to see if it can improve heart function and structure.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 6 years old.
- You must have a confirmed diagnosis of Friedreich ataxia with symptoms starting by age 25.
- Your heart must show thickening (left ventricular hypertrophy) and abnormal mass.
- Your heart's pumping ability must be at least 30%.
- You cannot have other causes of heart failure or have had gene therapy before.
- You must not have poorly controlled diabetes or active cancer.
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 one-time gene therapy that delivers a corrected copy of the frataxin gene directly to the heart. It aims to improve or stabilize heart function in people with Friedreich's ataxia who have early or advanced heart involvement.
This study looks at how heart disease progresses in people with Friedreich's ataxia, a genetic condition that affects coordination and heart health. By taking part, you help researchers better understand the condition, which may guide future treatments.
This trial tests an investigational drug called DT-216P2 in people with Friedreich's ataxia. It aims to find the right dose and see how safe and tolerable it is.
This study follows people with Friedreich's ataxia (FA) over time to learn more about the disease. It helps researchers understand how FA affects people of all ages and may lead to better treatments.
This study tests a new medicine called DT-216P2 in healthy people to find a safe dose. It is a first step in developing a treatment for Friedreich ataxia, a genetic condition that affects movement and balance.
This trial tests an injectable medicine called CTI-1601 for Friedreich's ataxia (FRDA). It is an open-label study, meaning everyone knows they are getting the drug. The study is for people who have taken CTI-1601 before (without serious side effects) or are new to the drug.
Hear when a new Friedreich Ataxia trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.