Gene therapy trial for Friedreich's ataxia
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a gene therapy called SGT-212 for people with Friedreich's ataxia. It aims to help treat the condition by delivering a working copy of the gene.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have been diagnosed with Friedreich's ataxia based on symptoms starting at age 25 or younger
- Your disease stage must be between 1 and 5 on the FA disability scale
- If you take Skyclarys, you must be on a stable dose for 12 weeks and willing to stop if needed; if not, you must have stopped it at least 12 weeks ago
- You cannot have antibodies against the virus used in this therapy (AAV9)
- Your weight must be over 55 pounds (25 kg) and your BMI under 33
- You must be able to have heart and brain MRIs and heart biopsies
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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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.
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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This trial tests a long-term study medication called vatiquinone for people with Friedreich's ataxia. It is designed to see if this medicine can help slow down the disease over time.
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.
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