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New registry tracks Real-World safety of FA drug over 5 years

NCT ID NCT06623890

Knowledge-focused Sponsor: Biogen Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows about 300 people with Friedreich's ataxia who are taking the prescribed drug omaveloxolone (SKYCLARYS®). Researchers will collect safety information from regular doctor visits for up to 5 years. The goal is to see how many participants have serious side effects, especially heart or liver problems. No experimental treatment is given—this is an observational registry.

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.

Participants

About 300 people

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

Started

Dec 2024

Expected to finish

Oct 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.

Who is studied

All participants for this study will be identified and enrolled via the FA-Global Clinical Consortium (FA-GCC) UNIFIED Natural History Study (UNIFAI study).

Ages

16 years and older

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: * Documented diagnosis of FA, including confirmation via genetic testing. * Participants aged 16 years and older at initiation of omaveloxolone treatment. For the omaveloxolone-naive cohort \- Initiating omaveloxolone treatment as per an approved label concurrent with enrolling in this study. For the omaveloxolone-non-naive cohort * Initiated omaveloxolone treatment as per an approved label less than 12 months prior to enrollment in this study * Prior to enrollment, maintained omaveloxolone treatment with no discontinuation of more than 60 days * Actively on treatment at the time of enrollment in this study * Treating physician is the study site principal investigator or sub-investigator * Study site confirms ability to provide required baseline data through medical record review, UNIFAI database, or other site-collected data * Enrolled in the UNIFAI study prior to initiation of omaveloxolone treatment Key Exclusion Criteria: * Received off-label prescription of omaveloxolone at any time. * Previously enrolled in a clinical trial of omaveloxolone. * Participating in a blinded interventional trial at the time of enrollment in the study; participants may participate in other clinical trials after baseline data are collected. Note: Other protocol-defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

Friedreich ataxia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Friedreich ataxia

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

    14 sites in 7 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

  • Center for hereditary ataxias, Motol

    RECRUITING

    Motol, Prague, 5, 15006, Czechia

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Fondazione I.R.C.C.S. Istituto Neurologico C. Besta

    NOT_YET_RECRUITING

    Milan, 20133, Italy

  • HUB-Hôpital Erasme

    NOT_YET_RECRUITING

    Brussels, 1070, Belgium

  • Klinikum der Universität München

    NOT_YET_RECRUITING

    Munich, 80336, Germany

  • Medizinische Universität Innsbruck

    RECRUITING

    Innsbruck, 6020, Austria

  • Ospedale Pediatrico Bambino Gesu

    NOT_YET_RECRUITING

    Rome, 50, Italy

  • Scientific Institute, IRCCS E. Medea

    NOT_YET_RECRUITING

    Conegliano, 31015, Italy

  • Stichting Radboud universitair medisch centrum

    NOT_YET_RECRUITING

    Nijmegen, 6525, Netherlands

  • UCLA Neurology

    NOT_YET_RECRUITING

    Los Angeles, California, 90095, United States

  • University Hospital Aachen

    NOT_YET_RECRUITING

    Aachen, 52074, Germany

  • University of Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • University of Florida

    NOT_YET_RECRUITING

    Gainesville, Florida, 32608, United States

  • Universitätsklinikum Tübingen

    RECRUITING

    Tübingen, Baden-Würtemberg, 72076, Germany

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