Hope for Friedreich's ataxia: new drug trial aims to slow disease progression
NCT ID NCT06447025
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing the long-term safety and effectiveness of an experimental drug called CTI-1601 for people with Friedreich's ataxia, a rare genetic disease that affects movement and heart function. It includes about 85 adolescents and adults who have or have not taken the drug before. Participants receive daily injections, and researchers will monitor side effects, heart health, motor skills, and daily living abilities over time.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
Locations
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Children's Hospital of the University of Pennsylvania (CHOP)
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Clinilabs Drug Development, Corp.
RECRUITINGEatontown, New Jersey, 07724, United States
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Fixel Institute for Neurological Disease, University of Florida Health
ACTIVE_NOT_RECRUITINGGainesville, Florida, 32608, United States
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Morsani Center for Advanced Health Care, University of South Florida Health
RECRUITINGTampa, Florida, 33612, United States
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Ohio State University United States
ACTIVE_NOT_RECRUITINGColumbus, Ohio, 43210, United States
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Uncommon Cures
RECRUITINGChevy Chase, Maryland, 20815, United States
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University of California Los Angeles
RECRUITINGLos Angeles, California, 90095, United States
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can voice and hearing tests reveal hidden clues to Friedreich's ataxia progression?
- Video games and AI join the fight against a rare movement disorder
- Can a single gene fix a fatal heart condition? a trial aims to find out
- Brain function in Friedreich's ataxia: new clues from genetic testing
- Pregnancy safety of friedreich ataxia drug under scrutiny
- New brain scans could reveal hidden clues in rare nerve disease