FRIEDREICH ATAXIA
Clinical trials for FRIEDREICH ATAXIA explained in plain language.
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Hope for young FA patients: major trial tests first approved drug in kids
Disease control Recruiting nowThis study is testing a drug called omaveloxolone (SKYCLARYS®) in children and teenagers with Friedreich's ataxia (FA), a rare genetic disorder that affects movement and coordination. The drug is already approved for adults, but this trial aims to see if it is safe and effective …
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE3 • Sponsor: Biogen • Aim: Disease control
Last updated Apr 02, 2026 14:57 UTC
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First trial for young kids with rare neurological disease
Disease control Recruiting nowThis study aims to find the right dose and check the safety of the drug omaveloxolone (SKYCLARYS®) in children and teenagers aged 2 to 15 with Friedreich's ataxia. The drug is already approved for people 16 and older, but researchers need to understand how younger bodies process …
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE1 • Sponsor: Biogen • Aim: Disease control
Last updated Mar 31, 2026 12:12 UTC
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First-in-Human gene therapy trial targets fatal heart damage in rare disease
Disease control Recruiting nowThis early-stage study is testing the safety of a new gene therapy for the serious heart muscle damage that occurs in people with Friedreich's ataxia. The therapy, called AAVrh.10hFXN, is given through an IV and aims to deliver a working copy of the frataxin gene directly to the …
Matched conditions: FRIEDREICH ATAXIA
Phase: PHASE1 • Sponsor: Weill Medical College of Cornell University • Aim: Disease control
Last updated Mar 23, 2026 15:18 UTC
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Scientists track Real-Time impact of rare neurological disease
Knowledge-focused Recruiting nowThis study aims to better understand how Friedreich Ataxia affects people's daily lives, quality of life, and creates financial burdens. It will observe 200 patients in Germany, Austria, and France. Participants will complete an initial interview and then use a smartphone app to …
Matched conditions: FRIEDREICH ATAXIA
Sponsor: German Center for Neurodegenerative Diseases (DZNE) • Aim: Knowledge-focused
Last updated Apr 03, 2026 14:41 UTC
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Scientists track rare Disease's hidden heart damage
Knowledge-focused Recruiting nowThis study aims to understand how heart disease develops in people with Friedreich Ataxia, a rare genetic disorder. Researchers will follow 65 participants over time to track changes in heart structure and function. The goal is to gather detailed information that could help desig…
Matched conditions: FRIEDREICH ATAXIA
Sponsor: Lexeo Therapeutics • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:34 UTC