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

NCT06623890 | Recruiting FDA Drug

• 2 other identifiers: 296FA401EUPAS1000000196
• Study Type: observational
• Target: 300
• Locations: 7 countries
• Sites: 14

Brief Summary

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.

Conditions

Friedreich Ataxia

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (4)

• 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

Study Arms (2)

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

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

Interventions

Omaveloxolone DRUG

Administered as specified in the treatment arm.

Also known as: SKYCLARYS, BIIB141

Omaveloxolone Naive Participants; Omaveloxolone Non-Naive Cohort

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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).

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Biogen: lead

Study Sites (14)

UCLA Neurology

Los Angeles, California, 90095, United States

NOT YET RECRUITING

University of Colorado

Aurora, Colorado, 80045, United States

RECRUITING

University of Florida

Gainesville, Florida, 32608, United States

NOT YET RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Medizinische Universität Innsbruck

Innsbruck, 6020, Austria

RECRUITING

HUB-Hôpital Erasme

Brussels, 1070, Belgium

NOT YET RECRUITING

Center for hereditary ataxias, Motol

Motol, Prague, 5, 15006, Czechia

RECRUITING

Universitätsklinikum Tübingen

Tübingen, Baden-Würtemberg, 72076, Germany

RECRUITING

University Hospital Aachen

Aachen, 52074, Germany

NOT YET RECRUITING

Klinikum der Universität München

Munich, 80336, Germany

NOT YET RECRUITING

Scientific Institute, IRCCS E. Medea

Conegliano, 31015, Italy

NOT YET RECRUITING

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

Milan, 20133, Italy

NOT YET RECRUITING

Ospedale Pediatrico Bambino Gesu

Rome, 50, Italy

NOT YET RECRUITING

Stichting Radboud universitair medisch centrum

Nijmegen, 6525, Netherlands

NOT YET RECRUITING

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

omaveloxolone

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Central Study Contacts

Study Director

CONTACT

866-633-4636; clinicaltrials@biogen.com

Global Biogen Clinical Trial Center

CONTACT

clinicaltrials@biogen.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 30, 2024
• First Posted: October 2, 2024
• Study Start: December 12, 2024
• Primary Completion (Estimated): October 1, 2029
• Study Completion (Estimated): October 1, 2029
• Last Updated: April 28, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (4); CZ (1); NL (1); DE (3); IT (3); AU (1); BE (1)