NCT06623890 · Biogen
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
What this study is about
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.
View original scientific description
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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.
Where
- Los Angeles, California
- Aurora, Colorado
- Gainesville, Florida
- Philadelphia, Pennsylvania
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 28, 2026 · Source of record for eligibility and locations