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.
Similar studies
Other trials that look related to this one.
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.
This trial tests a gene therapy called FLT201 for adults with Gaucher disease type 1. It aims to provide a long-term treatment option for people who have been stable on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least two years.
This study tests a drug called vatiquinone to see if it can help slow the progression of Friedreich's ataxia over the long term. You may be able to join if you are at a certain stage of the disease and are still able to walk.
This study looks at the long-term safety of omaveloxolone (BIIB141) for people with Friedreich's ataxia. You can join if you are just starting this medicine or have been taking it for less than a year.
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.
Hear when a new Friedreich's Ataxia (FA) trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.