ClinicalTrials.gov record NCT06623890

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 7, 2026

An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich's Ataxia

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age16 years old and older
SexAll
LocationAustria, Belgium, Czechia, Germany, Italy, Netherlands, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This observational registry collects real-world safety information on omaveloxolone (SKYCLARYS) in people with Friedreich's Ataxia who are prescribed the drug by their own doctors. Participants are identified through the FA-GCC UNIFAI network and followed for up to 5 years. The study includes people starting the drug when they join and people who began treatment within the year before joining.

What it is studying. The study is gathering long-term, real-world safety data on omaveloxolone in people with Friedreich's Ataxia. It focuses on counting serious adverse events and specifically tracking liver injury and congestive heart failure events, and it records when and why treatment is interrupted, stopped, or overdosed.

What the researchers hope to learn. To assess the long-term safety of omaveloxolone in real-world use, including characterization of drug-induced liver injury and congestive heart failure adverse events, and to capture reasons and timing of treatment interruptions, discontinuations, and overdose.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This is a drug available for doctors to prescribe for people with Friedreich's Ataxia, also known as FA. This is known as an "observational" study, which collects health information about study participants without changing their medical care. Participants for this study will be found using a group called the Friedreich's Ataxia Global Clinical Consortium (FA GCC) UNIFIED Natural History Study (UNIFAI). The FA-GCC is a group of study research centers that helps provide clinical care for FA patients and also helps researchers learn more about how FA affects patients over a long time. The main objective of this study is to collect safety information in participants with FA from UNIFAI. Some of the participants in this study will be prescribed BIIB141 for the first time by their own doctors. Some of the participants will have started taking BIIB141 after joining UNIFAI, but less than 12 months before joining this study. The main questions researchers want to answer in this study are:

  • How many participants had serious adverse events (SAEs)? An adverse event is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.
  • How many participants had adverse events (AEs) related to heart failure or liver damage caused by the drug?

Researchers will also learn more about :

  • Why and when participants stopped treatment, left the study, or took more of the drug than was prescribed

This study will be done as follows:

  • Participants will be screened to check if they can join the study.
  • After joining the study, the participants who had never started BIIB141 treatment before must start it within 6 months. Otherwise, all participants will take BIIB141 throughout this study as prescribed by their own doctor.
  • During the study, each participant's doctor will decide how often the participant visits the study research center to check on their health. This will be based on the doctor's own clinical judgment and what is recommended by the drug's label.
  • Data from the participants' regular visits to their doctor will be collected at 1 month, 2 months, 3 months, 6 months, 12 months, 24 months, 36 months, 48 months, and 60 months.
  • Each participant will be in the study for up to 5 years.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Omaveloxolone

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

The primary objective of this study is to assess the long-term safety of omaveloxolone as prescribed to participants with FA in the real-world setting, including characterization of all drug-induced liver injury (DILI) and congestive heart failure (CHF) AEs. The secondary objective of this study is to capture the reasons and timing of omaveloxolone treatment interruptions, discontinuations, and drug overdose.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

After screening, participants' routine clinical visits and health information will be collected at specified times (1, 2, 3, 6, 12, 24, 36, 48 and 60 months) for up to 5 years. Non‑naive participants have retrospective baseline data from before enrollment plus prospective follow‑up; naive participants must start omaveloxolone within 6 months of joining and then be followed. Visit timing is determined by the participant's own doctor per clinical judgment and the drug label. The registry record does not list extra invasive procedures beyond collection of data from regular clinical visits.

How the study is structured. This is a prospective observational cohort registry. Participants receive usual clinical care from their own doctors (the study does not assign treatment) and data from routine clinic visits are collected at scheduled intervals over a 5-year period. The study analyzes two groups: people who start omaveloxolone when they enroll and people who began omaveloxolone within the previous 12 months.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in Austria, Belgium, Czechia, Germany, Italy, Netherlands, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Friedreich ataxia

Clinical trials26 trials listed for Friedreich ataxiaEvery trial the registry lists for Friedreich ataxia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Friedreich ataxia researchLatest monthly update: September 2026. Includes: Heart change stages. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Dec 12, 2024
Primary completion
Oct 1, 2029 (estimated)
Study completion
Oct 1, 2029 (estimated)
Target enrollment
300 participants (estimated)
Last updated on registry
Apr 28, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Biogen

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (14)

Terms used on this page · explanations by MedicalUpdates.ai

Natural History Study
A study that follows how a condition changes over time without assigning an experimental treatment. It may involve study visits and tests, and is often used to plan future trials.
serious adverse events
Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
cohort
A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
principal investigator
The doctor or scientist responsible for running the study at a site or overall.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
clinical trial
A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
blinded
Who does not know which intervention a participant is receiving. It can apply to participants, the people giving the treatment, those assessing results, or those analysing the data.
Exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Key Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
primary objective
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
secondary objective
An additional result the study measures alongside the primary outcome, for example quality of life or a laboratory value.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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