Experimental drug aims to halt rare neurological decline
NCT ID NCT07681713
First seen Jul 02, 2026 · Last updated Aug 14, 2026 · Updated 1 time
Summary
This Phase 3 trial tests whether vatiquinone can slow the progression of Friedreich's ataxia, a rare genetic disorder that damages the nervous system and impairs movement. About 120 adults with moderate symptoms will take the drug for 24 months. Researchers will measure changes in movement, balance, and daily activities to see if the treatment preserves function.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- vatiquinone
- What this could lead to
- If successful, vatiquinone could become the first treatment to slow the progression of Friedreich's ataxia, preserving mobility and daily function for longer.
- What could go wrong
- This is a Phase 3 trial, but results may not show a clear benefit. The drug may cause side effects, and what works in this study may not help all patients.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 120 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Aug 2026
An estimate. Start dates often move.
- Expected to finish
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Mar 2029
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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7 to 21 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Key Inclusion Criteria: * mFARS ≥20 to ≤70 at Screening (4 weeks prior to Day 1) and Baseline (Day 1). * Must be ambulatory as defined by a E7 score of 4 or less on the USS at Screening and Baseline. * Documentation that participants reached maximum score on items E4, E5, and E3b on the USS of mFARS at Screening and Baseline. * FA diagnosis (homozygous for guanine-adenine-adenine \[GAA\] repeat expansion in intron-1 of the frataxin gene), confirmed and documented with GAA repeat length for both alleles by clinical genetic testing. * Difference in the mFARS score between Screening and Baseline of no more than 4 points. * Ability to abstain from strong cytochrome P450 (CYP) 3A4 inducers/inhibitors (for example, ketoconazole, rifampin, St. John's wort, grapefruit juice) for at least 4 weeks prior to Baseline and for the duration of the study. Key Exclusion Criteria: * Individuals with clinical diagnosis of FA who have point mutations, deletions, or other non-GAA expansion mutations. * Allergy to vatiquinone, sesame oil, gelatin (bovine and/or porcine), titanium dioxide, or red iron oxide. * Pregnant or lactating participants or those sexually active participants who are unwilling to comply with proper birth control methods; females of childbearing potential must have a negative pregnancy test at Screening and during the Baseline Visit. * Comorbidities that may confound study results (for example, fat malabsorption syndrome, other mitochondrial disorder) in the opinion of the investigator. * Participation in an interventional clinical study or received investigational drug (other than SKYCLARYS® \[omaveloxolone\]) within 60 days prior to Screening. Participants may be screened after the exclusionary period of 60 days has passed. * Current use of omaveloxolone. Previous use of omaveloxolone will be allowed if: 1. Use was less than 3 cumulative months and participants have been off treatment for \>30 days. 2. Use was less than 6 cumulative months and participants have been off treatment for \>90 days * Previous or concurrent use of other investigational treatment for FA. * Participation in any cell or gene therapy-based treatment for FA. * Participation in an ongoing study for vatiquinone or current or previous use of vatiquinone. * Illicit drug use 30 days prior to Screening and during the study. Note: Other protocol-defined inclusion and exclusion criteria may apply.
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Sign up to get updates when this study changes or when new studies for Friedreich's ataxia are added.
Genom att skicka in godkänner du våra Användarvillkor
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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
9 sites in 6 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Email: •••••@•••••
Locations
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Centre Hospitalier de l'Universite de Montreal CHUM
Montreal, Quebec, H2X 0A9, Canada
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Hospital Sant Joan de Deu
Barcelona, 08950, Spain
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Hôpital Érasme (Hôpital Universitaire de Bruxelles)
Brussels, 1070, Belgium
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Institut du Cerveau et de la Moelle Epinière
Paris, 75013, France
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University of California Los Angeles
Los Angeles, California, 90095, United States
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University of Campinas
Campinas, São Paulo, 13083-888, Brazil
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University of Iowa
Iowa City, Iowa, 52242, United States
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University of South Florida
Tampa, Florida, 33612, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a missing protein be replaced to slow Friedreich's ataxia?
- Can advanced heart scans predict the biggest killer in Friedreich's ataxia?
- New brain scans could reveal hidden clues in rare nerve disease
- Genetic clues may predict when Friedreich's ataxia begins
- Can a supplement and exercise improve stamina in Friedreich's ataxia?
- Exercise and drug combo may fight fatigue in rare disease