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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Friedreich Ataxia
• Intervention / Treatment: Drug: Omaveloxolone

Detailed Description

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.

• Study Type: Observational
• Enrollment (Estimated): 300

Contacts and Locations

• Study Contact: Name: Global Biogen Clinical Trial Center; Email: clinicaltrials@biogen.com
• Study Contact Backup: Name: Study Director; Phone Number: 866-633-4636; Email: clinicaltrials@biogen.com

Study Locations

• Austria
  • Innsbruck, Austria, 6020
    • Recruiting
    • Medizinische Universität Innsbruck
    • Contact: Phone Number: 0043 512 504 81196; Email: wolfgang.nachbauer@i-med.ac.at
    • Principal Investigator: Sylvia Boesch
• Belgium
  • Brussels, Belgium, 1070
    • Not yet recruiting
    • HUB-Hôpital Erasme
    • Contact: Phone Number: 3225558196; Email: gilles.naeije@hubruxelles.be
    • Principal Investigator: Naeije Gilles
• Czechia
  • Prague
    • Motol, Prague, Czechia, 5, 15006
      • Recruiting
      • Center for hereditary ataxias, Motol
      • Contact: Phone Number: +420-702001921; Email: martin.vyhnalek@fnmotol.cz
      • Principal Investigator: Martin Vyhnalek
• Germany
  • Aachen, Germany, 52074
    • Not yet recruiting
    • University Hospital Aachen
    • Principal Investigator: Kathrin Reetz
    • Contact: Phone Number: +49 241 80 89609; Email: kreetz@ukaachen.de
  • Munich, Germany, 80336
    • Not yet recruiting
    • Klinikum der Universitat Munchen
    • Contact: Phone Number: +49 89 4400 57400; Email: thomas.klopstock@med.uni-muenchen.de
    • Principal Investigator: Thomas Klopstock
  • Baden-Würtemberg
    • Tübingen, Baden-Würtemberg, Germany, 72076
      • Recruiting
      • Universitätsklinikum Tübingen
      • Contact: Phone Number: +4970712980445
      • Principal Investigator: Ludger Schöls
• Italy
  • Conegliano, Italy, 31015
    • Not yet recruiting
    • Scientific Institute, IRCCS E. Medea
    • Contact: Phone Number: +393383065324; Email: gabriella.paparella@lanostrafamiglia.it
    • Principal Investigator: Gabriella Paparella
  • Milan, Italy, 20133
    • Not yet recruiting
    • Fondazione I.R.C.C.S. Istituto Neurologico C. Besta
    • Principal Investigator: Caterina Mariotti
    • Contact: Phone Number: +39 02 23942269; Email: caterina.mariotti@istituto-besta.it
  • Rome, Italy, 50
    • Not yet recruiting
    • Ospedale Pediatrico Bambino Gesù
    • Contact: Phone Number: +390668593461; Email: gessica.vasco@opbg.net
    • Principal Investigator: Gessica Vasco
• Netherlands
  • Nijmegen, Netherlands, 6525
    • Not yet recruiting
    • Stichting Radboud Universitair Medisch Centrum
    • Principal Investigator: Bart van de Warrenburg
    • Contact: Phone Number: +31 (0)24-3613396; Email: bart.vandewarrenburg@radboudumc.nl
• United States
  • California
    • Los Angeles, California, United States, 90095
      • Not yet recruiting
      • UCLA Neurology
      • Principal Investigator: Susan Perlman
      • Contact: Phone Number: 310-206-8153; Email: sperlman@mednet.ucla.edu
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Recruiting
      • University of Colorado
      • Contact: Phone Number: 303-724-2642; Email: dillan.patel@cuanschutz.edu
      • Principal Investigator: Trevor Hawkins
  • Florida
    • Gainesville, Florida, United States, 32608
      • Not yet recruiting
      • University of Florida
      • Contact: Phone Number: 352-273-5000; Email: s.subramony@neurology.ufl.edu
      • Principal Investigator: Sub Subramony
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Children's Hospital of Philadelphia
      • Principal Investigator: David Lynch
      • Contact: Phone Number: 215-590-2242

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Omaveloxolone Non-Naive Cohort; Participants with FA who initiated omaveloxolone treatment as per the approved label less than 12 months prior to enrollment in this study will be analyzed retrospectively (baseline data) followed by prospective analysis (post-baseline data) for up to 5 years.	Drug: Omaveloxolone; Administered as specified in the treatment arm.; Other Names: SKYCLARYS; BIIB141
Omaveloxolone Naive Participants; Participants with FA who will initiate omaveloxolone treatment per its approved label will be followed prospectively for up to 5 years.	Drug: Omaveloxolone; Administered as specified in the treatment arm.; Other Names: SKYCLARYS; BIIB141

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Omaveloxolone Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)	From the start of the treatment up to end of the study (up to 5 years)
Omaveloxolone Non-Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)	From enrolment in the current study up to end of the study (up to 5 years)
Omaveloxolone Naive Cohort: Number of Participants With DILI and CHF AEs	From the start of the treatment up to end of the study (up to 5 years)
Omaveloxolone Non-Naive Cohort: Number of Participants With DILI and CHF AE	From enrolment in the current study up to end of the study (up to 5 years)

Secondary Outcome Measures

Outcome Measure	Time Frame
Time to Omaveloxolone Treatment Interruption	Up to 5 years
Time to Omaveloxolone Treatment Discontinuation	Up to 5 years
Time to Omaveloxolone Drug Overdose	Up to 5 years
Number of Participants With Reasons for Omaveloxolone Treatment Interruption, Treatment Discontinuation and Overdose	Up to 5 years

Collaborators and Investigators

• Sponsor: Biogen
• Investigators: Study Director: Medical Director, Biogen

Study record dates

Study Major Dates

• Study Start (Actual): December 12, 2024
• Primary Completion (Estimated): October 1, 2029
• Study Completion (Estimated): October 1, 2029

Study Registration Dates

• First Submitted: September 30, 2024
• First Submitted That Met QC Criteria: September 30, 2024
• First Posted (Actual): October 2, 2024

Study Record Updates

• Last Update Posted (Actual): April 28, 2026
• Last Update Submitted That Met QC Criteria: April 23, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolic Diseases; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Spinal Cord Diseases; Mitochondrial Diseases; Cerebellar Diseases; Spinocerebellar Degenerations; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Friedreich Ataxia; omaveloxolone
• Other Study ID Numbers: 296FA401; EUPAS1000000196 (Other Identifier: HMA-EMA Catalogues)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No