Friedreich AtaxiaNCT06623890
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 study of Friedreich Ataxia, run by Biogen at 14 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- Omaveloxolone
- How many people
- 300 participants
- Where
- 14 sitesin 7 countries
- Study dates
- 12 Dec 2024to 1 Oct 2029
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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).
Who may be able to join
- 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
Show all 11Show fewer
- 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
Who cannot join
- 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.
Trial locations and contacts
14 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
Global Biogen Clinical Trial Center contact
14 sites in 7 countries
United States4 sites, 4 recruiting
- Recruiting
University of Colorado
Aurora, Colorado, 80045
303-724-2642, dillan.patel@cuanschutz.edu
Trevor Hawkins
- Not yet recruiting
University of Florida
Gainesville, Florida, 32608
352-273-5000, s.subramony@neurology.ufl.edu
Sub Subramony
- Not yet recruiting
UCLA Neurology
Los Angeles, California, 90095
310-206-8153, sperlman@mednet.ucla.edu
Susan Perlman
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
215-590-2242
David Lynch
Germany3 sites, 3 recruiting
- Not yet recruiting
University Hospital Aachen
Aachen, 52074
+49 241 80 89609, kreetz@ukaachen.de
Kathrin Reetz
- Not yet recruiting
Klinikum der Universität München
Munich, 80336
+49 89 4400 57400, thomas.klopstock@med.uni-muenchen.de
Thomas Klopstock
- Recruiting
Universitätsklinikum Tübingen
Tübingen, Baden-Würtemberg, 72076
+4970712980445
Ludger Schöls
Italy3 sites, 3 recruiting
- Not yet recruiting
Scientific Institute, IRCCS E. Medea
Conegliano, 31015
+393383065324, gabriella.paparella@lanostrafamiglia.it
Gabriella Paparella
- Not yet recruiting
Fondazione I.R.C.C.S. Istituto Neurologico C. Besta
Milan, 20133
+39 02 23942269, caterina.mariotti@istituto-besta.it
Caterina Mariotti
- Not yet recruiting
Ospedale Pediatrico Bambino Gesu
Rome, 50
+390668593461, gessica.vasco@opbg.net
Gessica Vasco
Austria1 site, 1 recruiting
- Recruiting
Medizinische Universität Innsbruck
Innsbruck, 6020
0043 512 504 81196, wolfgang.nachbauer@i-med.ac.at
Sylvia Boesch
Belgium1 site, 1 recruiting
- Not yet recruiting
HUB-Hôpital Erasme
Brussels, 1070
3225558196, gilles.naeije@hubruxelles.be
Naeije Gilles
Czechia1 site, 1 recruiting
- Recruiting
Center for hereditary ataxias, Motol
Motol, Prague, 5, 15006
+420-702001921, martin.vyhnalek@fnmotol.cz
Martin Vyhnalek
Netherlands1 site, 1 recruiting
- Not yet recruiting
Stichting Radboud universitair medisch centrum
Nijmegen, 6525
+31 (0)24-3613396, bart.vandewarrenburg@radboudumc.nl
Bart van de Warrenburg
About this study
Official title: 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.
Read more
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.
Read the 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.
- Conditions
- Friedreich Ataxia
- Being tested
- Omaveloxolone
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OmaveloxoloneDrug
Also called SKYCLARYS, BIIB141
Administered as specified in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| Omaveloxolone Naive Participants | Participants with FA who will initiate omaveloxolone treatment per its approved label will be followed prospectively for up to 5 years. Receives: 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. Receives: Drug: Omaveloxolone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Omaveloxolone Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)
Measured: 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)
Measured: 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
Measured: 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
Measured: From enrolment in the current study up to end of the study (up to 5 years)
Secondary outcomes (4)
Time to Omaveloxolone Treatment Interruption
Measured: Up to 5 years
Time to Omaveloxolone Treatment Discontinuation
Measured: Up to 5 years
Time to Omaveloxolone Drug Overdose
Measured: Up to 5 years
Number of Participants With Reasons for Omaveloxolone Treatment Interruption, Treatment Discontinuation and Overdose
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2024
- Study start12 Dec 2024
- Primary completion1 Oct 2029 (estimated)
- Study completion1 Oct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06623890
- Sponsor's protocol ID
- 296FA401
- Other IDs
- EUPAS1000000196
- Status last verified
- Apr 2026
- First submitted
- 30 Sep 2024
- Last updated
- 28 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational, Multinational, Post-Marketing Registry of Omaveloxolone-Treated Patients With Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT06623890
- Acronym / sponsor ID
- 296FA401
- Conditions
- Friedreich Ataxia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 12 Dec 2024 (actual)
- Primary completion
- 1 Oct 2029 (estimated)
- Study completion
- 1 Oct 2029 (estimated)
- First posted
- 2 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Omaveloxolone Naive Participants | – | Drug: OmaveloxoloneParticipants with FA who will initiate omaveloxolone treatment per its approved label will be followed prospectively for up to 5 years. |
| Omaveloxolone Non-Naive Cohort | – | Drug: OmaveloxoloneParticipants 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. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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).
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OmaveloxoloneAlso: SKYCLARYS, BIIB141Administered as specified in the treatment arm. | Drug | Omaveloxolone Naive Participants, Omaveloxolone Non-Naive Cohort |
Primary outcome
Omaveloxolone Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs)
Time frame: 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)
Time frame: 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
Time frame: 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
Time frame: From enrolment in the current study up to end of the study (up to 5 years)
4 secondary outcomes
Time to Omaveloxolone Treatment Interruption
Time frame: Up to 5 years
Time to Omaveloxolone Treatment Discontinuation
Time frame: Up to 5 years
Time to Omaveloxolone Drug Overdose
Time frame: Up to 5 years
Number of Participants With Reasons for Omaveloxolone Treatment Interruption, Treatment Discontinuation and Overdose
Time frame: Up to 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2026.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Global Biogen Clinical Trial Center contact
Medical Director study director
Biogen
14 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Friedreich Ataxia, run by Biogen at 14 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 28 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Germany, Italy, Austria, Belgium and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Study Director (866-633-4636, clinicaltrials@biogen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Omaveloxolone Naive Cohort: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs) (time frame: From the start of the treatment up to end of the study (up to 5 years)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Apr 2026; AzTrials last refreshed its copy on 30 Oct 2026.