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

NCT ID: NCT06623890

Last Updated: 2025-04-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

300 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-12-12

Study Completion Date

2029-10-01

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Friedreich Ataxia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Omaveloxolone Naive Cohort

Participants with FA who will initiate omaveloxolone treatment per its approved label will be followed prospectively for up to 5 years.

Omaveloxolone

Intervention Type DRUG

Administered as specified in the treatment arm.

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.

Omaveloxolone

Intervention Type DRUG

Administered as specified in the treatment arm.

Interventions

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Omaveloxolone

Administered as specified in the treatment arm.

Intervention Type DRUG

Other Intervention Names

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SKYCLARYS BIIB141

Eligibility Criteria

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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

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.
Minimum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Biogen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Biogen

Locations

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UCLA Neurology

Los Angeles, California, United States

Site Status NOT_YET_RECRUITING

University of Colorado

Aurora, Colorado, United States

Site Status RECRUITING

University of Florida

Gainesville, Florida, United States

Site Status NOT_YET_RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Medizinische Universität Innsbruck

Innsbruck, , Austria

Site Status RECRUITING

HUB-Hôpital Erasme

Brussels, , Belgium

Site Status NOT_YET_RECRUITING

Center for hereditary ataxias, Motol

Motol, Prague, Czechia

Site Status RECRUITING

Universitätsklinikum Tübingen

Tübingen, Baden-Würtemberg, Germany

Site Status RECRUITING

University Hospital Aachen

Aachen, , Germany

Site Status NOT_YET_RECRUITING

Klinikum der Universität München

Munich, , Germany

Site Status NOT_YET_RECRUITING

Scientific Institute, IRCCS E. Medea

Conegliano, , Italy

Site Status NOT_YET_RECRUITING

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

Milan, , Italy

Site Status NOT_YET_RECRUITING

Ospedale Pediatrico Bambino Gesu

Rome, , Italy

Site Status NOT_YET_RECRUITING

Stichting Radboud universitair medisch centrum

Nijmegen, , Netherlands

Site Status NOT_YET_RECRUITING

Countries

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United States Austria Belgium Czechia Germany Italy Netherlands

Central Contacts

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Study Director

Role: CONTACT

866-633-4636

Global Biogen Clinical Trial Center

Role: CONTACT

Facility Contacts

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Role: primary

310-206-8153

Role: primary

303-724-2642

Role: primary

352-273-5000

Role: primary

215-590-2242

Role: primary

0043 512 504 81196

Role: primary

3225558196

Role: primary

+420-702001921

Role: primary

+4970712980445

Role: primary

+49 241 80 89609

Role: primary

+49 89 4400 57400

Role: primary

+393383065324

Role: primary

+39 02 23942269

Role: primary

+390668593461

Role: primary

+31 (0)24-3613396

Other Identifiers

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EUPAS1000000196

Identifier Type: OTHER

Identifier Source: secondary_id

296FA401

Identifier Type: -

Identifier Source: org_study_id

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