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Experimental drug aims to halt rare neurological decline

NCT ID NCT07681713

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 120 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Aug 2026

An estimate. Start dates often move.

Expected to finish

Mar 2029

An estimate. End dates often move.

Lead sponsor

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

7 to 21 years

Sex

Anyone

Healthy volunteers

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

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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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.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    9 sites in 6 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Centre Hospitalier de l'Universite de Montreal CHUM

    Montreal, Quebec, H2X 0A9, Canada

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Hospital Sant Joan de Deu

    Barcelona, 08950, Spain

  • Hôpital Érasme (Hôpital Universitaire de Bruxelles)

    Brussels, 1070, Belgium

  • Institut du Cerveau et de la Moelle Epinière

    Paris, 75013, France

  • University of California Los Angeles

    Los Angeles, California, 90095, United States

  • University of Campinas

    Campinas, São Paulo, 13083-888, Brazil

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of South Florida

    Tampa, Florida, 33612, United States

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