Study of Friedreich's Ataxia With Cardiomyopathy
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This study follows people with Friedreich's ataxia (FA) who also have a heart condition called cardiomyopathy. By tracking how the disease progresses over time, researchers hope to better understand FA-related heart problems.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 6 years old or older.
- You have Friedreich's ataxia (FA) diagnosed before age 25.
- Your heart's main pumping chamber is thickened (left ventricular hypertrophy).
- Your heart's pumping strength (ejection fraction) is 40% or higher.
- You have not had gene therapy or cell therapy before.
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 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 drug called omaveloxolone (BIIB141) for children and teens with Friedreich’s Ataxia. It looks at how well the drug works and how safe it is over a longer time.
This Phase 1 trial tests an AAV gene therapy that aims to improve heart problems caused by Friedreich’s ataxia. You may be eligible if you have genetically confirmed Friedreich’s ataxia with measurable heart weakening but still enough heart function to safely receive treatment.
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.
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 looks at a condition where an abnormal heart rhythm can weaken the heart muscle (arrhythmia-induced cardiomyopathy). You may be asked questions, have heart tests reviewed, and help researchers better describe who is affected and how it develops.
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.