Testing gene therapy for Friedreich’s ataxia heart disease
Treatments studied
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You’re between 12 and 50 years old and can give informed consent (or have a guardian who can).
- You have Friedreich’s ataxia confirmed by genetic testing: a GAA expansion on both copies of the gene, with over 600 repeats in at least one copy.
- Your heart ultrasound/MRI and exercise results show Friedreich’s-related heart disease, and your left pumping function (EF) is about 35% to 75%.
- Your heart MRI shows limited scar/fibrosis (10% or less) and acceptable oxygen levels (resting O2 saturation at least 95%).
- You can have MRI scans, and your blood and organ lab tests are within allowed ranges (including kidney, liver, and blood counts).
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 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.
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 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 study tests an investigational drug given as a daily injection to see if it can improve walking and balance in people with Friedreich's ataxia.
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.
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