Gene therapy for the heart problems in Friedreich's ataxia
Treatments studied
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 12 and 50 years old.
- You need a confirmed diagnosis of Friedreich's ataxia with a specific genetic marker (more than 600 repeats of a DNA sequence).
- Your heart function (measured by a special heart MRI) must be between 35% and 75%.
- You must have some sign of heart disease linked to Friedreich's ataxia, such as a positive blood test for troponin (a heart damage marker) or a thickened heart muscle.
- You cannot have severe heart failure, uncontrolled diabetes, or a history of cancer in the last 5 years (except treated skin cancer).
- You must not be taking immunosuppressive drugs like corticosteroids, and you must not have had gene 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 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 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 tests a new medicine called GV101 to see if it can help people with Friedreich's ataxia, a condition that affects movement and balance. The trial will check if GV101 is safe and if it improves symptoms.
This study uses heart tests (including ultrasound and MRI) to better understand how Friedreich’s ataxia affects the heart. You might benefit by getting detailed heart evaluation and contributing to research that could guide future care.
This trial tests a gene therapy for people with alpha-1 antitrypsin deficiency who have mild to moderate lung disease. The aim is to see if the treatment can improve lung health and is safe.
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.