Actively Recruiting
An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich's Ataxia
Led by Biogen · Updated on 2026-04-28
300
Participants Needed
14
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the long-term safety of a drug called BIIB141, also known as omaveloxolone or SKYCLARYS4, in people with Friedreich's Ataxia (FA). This observational study collects health information from participants without changing their medical care. It involves participants from the Friedreich's Ataxia Global Clinical Consortium (FA GCC) UNIFIED Natural History Study (UNIFAI) to better understand how FA affects patients and to monitor safety events related to the drug. Participants are divided into two groups: those who will start omaveloxolone treatment as prescribed by their doctors during the study, and those who have started treatment less than 12 months before joining. Participants will be followed for up to 5 years. Doctors will decide how often participants visit based on clinical judgment and drug labeling. Data will be collected at multiple time points, including 1, 2, 3, 6, 12, 24, 36, 48, and 60 months. During the study, researchers will monitor serious adverse events, including heart failure and liver damage related to the drug. They will also track reasons for stopping treatment, leaving the study, or taking more of the drug than prescribed. Participants remain under their own doctors' care, and health data from regular visits will be collected throughout the study period.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documented diagnosis of Friedreich's Ataxia confirmed by genetic testing
- Participants aged 16 years and older at the start of omaveloxolone treatment
- For omaveloxolone-naive cohort: starting omaveloxolone treatment as per approved label during study enrollment
- For omaveloxolone-non-naive cohort: started omaveloxolone treatment less than 12 months before enrollment
- No treatment interruption of more than 60 days prior to enrollment
- Actively receiving omaveloxolone treatment at enrollment
- Treating physician is the study site principal investigator or sub-investigator
- Study site can provide required baseline data through medical records or the UNIFAI database
- Enrolled in the UNIFAI study before starting omaveloxolone treatment
You will not qualify if you...
- Received omaveloxolone off-label at any time
- Previously participated in a clinical trial of omaveloxolone
- Participating in a blinded interventional trial at enrollment; can join other trials after baseline data collection
- Other protocol-defined exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 5 years
Participants who are prescribed omaveloxolone by their own doctors are observed to assess the long-term safety of the treatment, including monitoring for serious adverse events, liver injury, and heart failure over time.
Regular visits as per routine care and study assessments
Trial Site Locations
Total: 14 locations
1
UCLA Neurology
Los Angeles, California, United States, 90095
Not Yet Recruiting
2
University of Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
University of Florida
Gainesville, Florida, United States, 32608
Not Yet Recruiting
4
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
5
Medizinische Universität Innsbruck
Innsbruck, Austria, 6020
Actively Recruiting
6
HUB-Hôpital Erasme
Brussels, Belgium, 1070
Not Yet Recruiting
7
Center for hereditary ataxias, Motol
Motol, Prague, Czechia, 5, 15006
Actively Recruiting
8
Universitätsklinikum Tübingen
Tübingen, Baden-Würtemberg, Germany, 72076
Actively Recruiting
9
University Hospital Aachen
Aachen, Germany, 52074
Not Yet Recruiting
10
Klinikum der Universität München
Munich, Germany, 80336
Not Yet Recruiting
11
Scientific Institute, IRCCS E. Medea
Conegliano, Italy, 31015
Not Yet Recruiting
12
Fondazione I.R.C.C.S. Istituto Neurologico C. Besta
Milan, Italy, 20133
Not Yet Recruiting
13
Ospedale Pediatrico Bambino Gesu
Rome, Italy, 50
Not Yet Recruiting
14
Stichting Radboud universitair medisch centrum
Nijmegen, Netherlands, 6525
Not Yet Recruiting
Research Team
S
Study Director
G
Global Biogen Clinical Trial Center
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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