Friedreich AtaxiaPhase 2NCT02255435

A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

A Phase 2 study testing Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg and Omaveloxolone Capsules, 10 mg in people with Friedreich Ataxia, run by Biogen at 11 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort, movement, coordination, and how participants feel in daily life. Read more about this study

Who is it for?: Ages 16 to 40 years
Conditions the study is for: Friedreich Ataxia
What is being tested?: Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg and Omaveloxolone Capsules, 10 mg
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 172 participants
Where: 11 sitesin 5 countries
Study dates: 31 Jan 2015to 19 Dec 2025
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 to 40 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Have genetically confirmed Friedreich's ataxia
Have a modified FARS score ≥20 and ≤80
Be male or female and ≥16 years of age and ≤40 years of age
Have no changes to exercise regimen within 30 days prior to Study Day 1 and be willing to remain on the same exercise regimen during the 16-week study period
Have the ability to complete maximal exercise testing
Be able to swallow capsules

Who cannot join

Have uncontrolled diabetes (HbA1c >11.0%)
Have B-type natriuretic peptide value >200 pg/mL
Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease
Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C)
Have known or suspected active drug or alcohol abuse
Have clinically significant abnormalities of clinical hematology or biochemistry, including but not limited to elevations greater than 1.5 times the upper limit of normal of aspartate aminotransferase, or alanine aminotransferase

Show all 14Show fewer

Have any abnormal laboratory test value or serious pre-existing medical condition that, in the opinion of the investigator, would put the patient at risk by study enrollment
Have taken any of the following drugs within 7 days prior to Study Day 1 or plan to take any of these drugs during the time of study participation:
Sensitive substrates for cytochrome P450 2C8 or 3A4 (e.g., repaglinide, midazolam, sildenafil)
Moderate or strong inhibitors or inducers of cytochrome P450 3A4 (e.g., carbamazepine, phenytoin, ciprofloxacin, grapefruit juice)
Substrates for p-glycoprotein transporter (e.g., ambrisentan, digoxin)
Have participated in any other interventional clinical study within 30 days prior to Study Day 1
Have a cognitive impairment that may preclude ability to comply with study procedures
Prior participation in a trial with omaveloxolone (RTA 408)

Trial locations and contacts

11 sites in 5 countries.

Country

City, postcode or hospital

11 sites in 5 countries

United States7 sites

Emory University Hospital - Neurology
Atlanta, Georgia, 30329
Ohio State University - Neurology
Columbus, Ohio, 43221
University of Florida - Neurology
Gainesville, Florida, 32610
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242
UCLA
Los Angeles, California, 90095
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
USF Ataxia Research Center
Tampa, Florida, 33612

Other trials in United States

Australia1 site

Murdoch Childrens Research Institute
Parkville, Victoria, 3052

Other trials in Australia

Austria1 site

Medical University Innsbruck
Innsbruck, 6020

Other trials in Austria

Italy1 site

Neurological Institute Carlo Besta
Milan, 20133

Other trials in Italy

United Kingdom1 site

University College of London
London, WC1E 6BT

Other trials in United Kingdom

About this study

Official title: A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)

The main questions researchers want to answer in this study are:

How much physical effort can a participant produce during a cycling test after 12 weeks of treatment?
How do scores on the modified Friedreich's Ataxia Rating Scale (mFARS) change after 48 weeks?

Researchers will use the modified Friedreich's Ataxia Rating Scale (mFARS) to measure how FA affects the nervous system. The mFARS looks at movement ability, balance, coordination, speech, and how well the arms and legs work.

They will also use a cycling test to measure physical effort, along with questionnaires to learn how participants feel and function in daily life.

Safety will also be tested using physical exams, vital sign checks, echocardiograms (ECHO), electrocardiograms (ECG), and blood and urine tests.

The study will be done in 2 main parts, followed by an optional Extension period:

In Part 1, participants will be randomly assigned to take different doses of RTA 408 or a placebo by mouth once a day for 12 weeks. A placebo looks like the study drug but contains no real medicine.
Researchers will compare these doses to decide which one to use in Part 2.
In Part 2, a different group of participants will take either the chosen dose of RTA 408 (150 mg) or placebo once a day for 48 weeks.
Participants who complete Part 1 or Part 2 may be able to join an Extension period, where everyone receives RTA 408.
In the Extension period, participants will continue to receive RTA 408 until the drug becomes commercially available or until they leave the study
Participants in Part 1 will have up to 9 study visits and 2 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 20 weeks.
Participants in Part 2 will have up to 10 study visits and 3 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 61 weeks.
Participants in the Extension period will have 2 visits in the first month, followed by visits every 6 months.

Read the detailed description

Friedreich's ataxia is an autosomal recessive cerebellar ataxia caused by triplet-repeat expansions. The causative mutation is a trinucleotide (GAA) repeat expansion in the first intron of the frataxin gene, leading to impaired transcription of frataxin. The pathological consequences of frataxin deficiency include a severe disruption of iron-sulfur cluster biosynthesis, mitochondrial iron overload coupled to cellular iron dysregulation, and an increased sensitivity to oxidative stress.

A hallmark of Friedreich's ataxia is impairment of antioxidative defense mechanisms, which play a major role in disease progression. Studies have demonstrated that nuclear factor erythroid-derived 2-related factor 2 (Nrf2) signaling is grossly impaired in participants with Friedreich's ataxia. Therefore, the ability of omaveloxolone (RTA 408) to activate Nrf2 and induce antioxidant target genes is hypothesized to be therapeutic in participants with Friedreich's ataxia.

This 2-part study will evaluate the efficacy, safety, and pharmacodynamics of omaveloxolone (RTA 408) in the treatment of participants with Friedreich's ataxia.

Part 1: The first part of this study will be a randomized, placebo-controlled, double-blind, dose-escalation study to evaluate the safety of omaveloxolone (RTA 408) at various doses in participants with Friedreich's ataxia.

Part 2: The second part of this study is a randomized, placebo-controlled, double-blind, parallel-group study to evaluate the safety and efficacy of omaveloxolone (RTA 408) 150 mg in participants with Friedreich's ataxia. Participants enrolled in Part 2 will be randomized 1:1 to receive omaveloxolone (RTA 408) 150 mg or placebo.

Extension: The extension will assess long-term safety and tolerability of omaveloxolone (RTA 408) in qualified participants with Friedreich's ataxia following completion of Part 1 or Part 2. Participants will not be unblinded to study treatment in Part 1 or Part 2 upon entering the extension study. Participants will receive open-label omaveloxolone (RTA 408) at 150 mg once daily.

Conditions: Friedreich Ataxia
Being tested: Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg, Placebo, Omaveloxolone Capsules, 20 mg
Keywords: RTA 408, RTA 408 Capsules, Oxidative Stress, Mitochondrial dysfunction, omaveloxolone
Medical subject headings: Friedreich Ataxia, Mitochondrial Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Omaveloxolone Capsules, 2.5 mgDrug
Also called RTA 408 Capsules 2.5 mg
Omaveloxolone Capsules, 5 mgDrug
Also called RTA 408 capsules, 5 mg
Omaveloxolone Capsules, 10 mgDrug
Also called RTA 408 capsules, 10 mg
PlaceboDrug
Omaveloxolone Capsules, 20 mgDrug
Also called RTA 408 capsules, 20 mg
Omaveloxolone Capsules, 40 mgDrug
Also called RTA 408 capsules, 40 mg
Omaveloxolone Capsules, 80 mgDrug
Also called RTA 408 capsules, 80 mg
Omaveloxolone Capsules, 160 mgDrug
Also called RTA 408 capsules, 160 mg
Omaveloxolone Capsules, 300 mgDrug
Also called RTA 408 capsules, 300 mg
Omaveloxolone Capsules, 150 mgDrug
Also called RTA 408 capsules, 150 mg

Study groups in NCT02255435
Study group	Role	What they receive
Part 1: Omaveloxolone 2.5 and 5 mg	Experimental	Participants received omaveloxolone 2.5 mg, oral capsule, QD for 2 weeks, followed by 5 mg Omaveloxolone, oral capsule, QD for 10 weeks. Receives: Drug: Omaveloxolone Capsules, 2.5 mg; Drug: Omaveloxolone Capsules, 5 mg
Part 1: Omaveloxolone 10 mg	Experimental	Participants received omaveloxolone 10 mg, oral capsule, QD for 12 weeks. Receives: Drug: Omaveloxolone Capsules, 10 mg
Part 1: Omaveloxolone 20 mg	Experimental	Participants received omaveloxolone 20 mg, oral capsule, QD for 12 weeks. Receives: Drug: Omaveloxolone Capsules, 20 mg
Part 1: Omaveloxolone 40 mg	Experimental	Participants received omaveloxolone 40 mg, oral capsule, QD for 12 weeks. Receives: Drug: Omaveloxolone Capsules, 40 mg
Part 1: Omaveloxolone 80 mg	Experimental	Participants received omaveloxolone 80 mg, oral capsule, QD for 12 weeks. Receives: Drug: Omaveloxolone Capsules, 80 mg
Part 1: Omaveloxolone 160 mg	Experimental	Participants received omaveloxolone 160 mg, oral capsule, QD for 12 weeks. Receives: Drug: Omaveloxolone Capsules, 160 mg
Part 1: Omaveloxolone 300 mg	Experimental	Participants received omaveloxolone 300 mg, oral capsule, QD for 12 weeks. Receives: Drug: Omaveloxolone Capsules, 300 mg
Part 1: Placebo	Placebo comparator	Participants received placebo, oral capsule, QD for 12 weeks. Receives: Drug: Placebo
Part 2: Omaveloxolone 150 mg	Experimental	Participants received omaveloxolone 150 mg, oral capsule, QD for 48 weeks. Receives: Drug: Omaveloxolone Capsules, 150 mg
Part 2: Placebo	Placebo comparator	Participants received placebo, oral capsule, QD for 48 weeks. Receives: Drug: Placebo
OLE: Placebo/ Omaveloxolone	Experimental	All eligible participants who had received either omaveloxone or placebo in Part 1 and eligible participants who received placebo in Part 2 received omaveloxone, 150 mg, oral capsule, QD, until the drug was available through commercial channels or alternate post-trial access mechanisms, or until participant withdrawal, whichever was sooner (up to approximately 370 weeks). Receives: Drug: Omaveloxolone Capsules, 150 mg
OLE: Omaveloxolone/ Omaveloxolone	Experimental	All eligible participants who had received omaveloxone in Part 2 continued to receive omaveloxolone, 150 mg, oral capsule, QD until the drug was available through commercial channels or alternate post-trial access mechanisms, or until participant withdrawal, whichever was sooner (up to approximately 370 weeks). Receives: Drug: Omaveloxolone Capsules, 150 mg

What is being measured

The primary outcome is the main question the study is designed to answer.

Part 1: Change From Baseline in Peak Work During Maximal Exercise Testing at Week 12
Cycle ergometry using a recumbent stationary bicycle was used to conduct maximal exercise testing and workload was increased incrementally. Peak work is defined as the workload at which participants reach maximal volition (defined as an inability to continue to exercise due to exhaustion). A positive change from baseline suggests an improvement.
Measured: Baseline, Week 12
Part 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
An adverse event (AE) was any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug whether or not it is considered to be study drug related. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. TEAEs were defined as any AEs, regardless of relationship to study drug, that had an onset or worsened in severity on or after the first dose of study drug.
Measured: From first dose of study drug up to end of Part 1 of the study (up to Week 16)
Part 2: Change From Baseline in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48
The Friedreich Ataxia Rating Scale (FARS) is a neurological-exam-based rating scale with five sections: Bulbar (section A), Upper Limb Coordination (section B), Lower Limb Coordination (section C), Peripheral Nervous System (section D), and Upright Stability (section E). mFARS is the sum of sections A (score 0 to 11), B (score 0 to 36), C (score 0 to 16), and E (score 0 to 36). The minimum score is 0 and the maximum score is 99. A lower score indicates better neurological function.
Measured: Baseline, Week 48
Part 2: Number of Participants With TEAEs and TESAEs
An AE was any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug whether or not it is considered to be study drug related. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. TEAEs were defined as any AEs, regardless of relationship to study drug, that had an onset or worsened in severity on or after the first dose of study drug.
Measured: From first dose of study drug up to end of Part 2 of the study (up to Week 52)

Secondary outcomes (8)

Part 1: Change From Baseline in the mFARS at Week 12
The FARS is a neurological-exam-based rating scale with five sections: Bulbar (section A), Upper Limb Coordination (section B), Lower Limb Coordination (section C), Peripheral Nervous System (section D), and Upright Stability (section E). mFARS is the sum of sections A (score 0 to 11), B (score 0 to 36), C (score 0 to 16), and E (score 0 to 36). The minimum score is 0 and the maximum score is 99. A lower score indicates better neurological function.
Measured: Baseline, Week 12
Part 2: Number of Participants With Patient Global Impression of Change (PGI-C)
The PGI-C is a 7-point scale that requires participants to assess how much their illness has improved or worsened relative to their baseline state at the beginning of the trial. Participants self-rated their perceived change by completing the following statement: "Since I began trial treatment, my overall status is:" 1=Very much improved, 2=Much improved, 3=Minimally improved, 4=No change, 5=Minimally worse, 6=Much worse and 7=Very much worse. Lower score indicates better improvement. The categories with at least one participant having a PGI-C score are reported.
Measured: Week 48
Part 2: Number of Participants With Clinical Global Impression of Change (CGI-C)
The CGIC scale is a 7 point scale that requires the clinician to assess how much the participant's illness has improved or worsened relative to a baseline state at the beginning of an intervention. The CGIC was assessed by completing the following statement "Compared to the participant's condition at the start of the trial, this participant's overall status is", where 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse. Lower score indicates better improvement. The categories with at least one participant having a CGIC score are reported.
Measured: Week 48
Part 2: Change From Baseline in Performance on a 9-Hole Peg Test (9-HPT) at Week 48
The 9-HPT is a brief, standardized, quantitative test of upper extremity (arm and hand) function. Both the dominant and nondominant hands were tested twice (2 consecutive trials of the dominant hand, followed immediately by 2 consecutive trials of the nondominant hand). The participant picks up pegs 1 at a time (9 in total), using 1 hand only, and places them into holes on the board as quickly as possible, in any order until all holes are filled. Then, without pausing, the participant removes the pegs 1 at a time and returns them as quickly as possible. The times recorded for the two trials for each hand are averaged, and the reciprocal average value for each hand was used in analyses. Longer test times reflect more impairment on the participant's upper extremity function, thus a negative change from baseline suggests an improvement.
Measured: Baseline, Week 48
Part 2: Change From Baseline in Performance on a 25-Foot Timed Walk Test (T25-FWT) at Week 48
The T25-FWT is a quantitative mobility and leg function performance test based on time (in seconds) to complete a 25-foot walk. Participants were instructed to attempt two T25-FWT trials at each visit. Both walk times were averaged, and the reciprocal average value was used in analyses. Longer test times reflect more impairment on the participant's ability to walk, thus a negative change from baseline suggests an improvement.
Measured: Baseline, Week 48
Part 2: Total Number of Falls
A fall was defined as "the participant unintentionally coming to rest on the ground or at a lower level." The total number of falls before and after study drug administration has been reported.
Measured: Up to Week 48
Part 2: Change From Baseline in Peak Work During Maximal Exercise Testing at Week 48
Cycle ergometry using a recumbent stationary bicycle was used to conduct maximal exercise testing and workload was increased incrementally. Peak work is defined as the workload at which participants reach maximal volition (defined as an inability to continue to exercise due to exhaustion). A positive change from baseline suggests an improvement.
Measured: Baseline, Week 48
Part 2: Change From Baseline in the Activities of Daily Living (ADL) Score at Week 48
The ADL scale examines the ability of participants to complete everyday tasks (e.g., holding a fork, dressing). The ADL is a 9-question assessment, with the total score being the sum of 9 questions. The ADL survey assesses 9 concepts: (1) speech; (2) swallowing; (3) cutting food and handling utensils; (4) dressing; (5) personal hygiene; (6) falling; (7) walking; (8) quality of sitting position; and (9) bladder function. Each of these is rated on a 5-point scale where 0= normal and 4= severe disability/inability to carry out activity independently, for a total possible score of 0 to 36, with higher scores representing greater disability/dependency. A negative change from baseline indicates improvement.
Measured: Baseline, Week 48

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

31 Jan 201519 Dec 2025

First posted2 Oct 2014 (estimated)
Study start31 Jan 2015
Primary completion31 Oct 2019
Study completion19 Dec 2025

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (3)Papers the sponsor has linked to this study.
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: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 172 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02255435
Sponsor's protocol ID: RTA 408-C-1402
Other IDs: 2024-517436-22
Status last verified: Aug 2026
First submitted: 30 Sep 2014
Last updated: 3 Sep 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Publications

Perlman S, Zhang S, Setyawan J, Yang H, Jiang A, Hua Q, Boudreau J, Khan S, Bajaj A, Lynch DR, Lawson R. The clinical burden of Friedreich ataxia in the United States: A retrospective claims database analysis. J Neurol Sci. 2025 Aug 15;475:123594. doi: 10.1016/j.jns.2025.123594. Epub 2025 Jun 25.
PubMed 40614535
Lynch DR, Chin MP, Delatycki MB, Subramony SH, Corti M, Hoyle JC, Boesch S, Nachbauer W, Mariotti C, Mathews KD, Giunti P, Wilmot G, Zesiewicz T, Perlman S, Goldsberry A, O'Grady M, Meyer CJ. Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study). Ann Neurol. 2021 Feb;89(2):212-225. doi: 10.1002/ana.25934. Epub 2020 Nov 5.
PubMed 33068037
Lynch DR, Farmer J, Hauser L, Blair IA, Wang QQ, Mesaros C, Snyder N, Boesch S, Chin M, Delatycki MB, Giunti P, Goldsberry A, Hoyle C, McBride MG, Nachbauer W, O'Grady M, Perlman S, Subramony SH, Wilmot GR, Zesiewicz T, Meyer C. Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia. Ann Clin Transl Neurol. 2018 Nov 10;6(1):15-26. doi: 10.1002/acn3.660. eCollection 2019 Jan.
PubMed 30656180

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
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RecruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
Friedreich Ataxia, Friedreich's Ataxia

All Friedreich Ataxia trials

Study at a glance

From the registry record, last updated by the sponsor on 3 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe)
ClinicalTrials.gov ID: NCT02255435
Acronym / sponsor ID: RTA 408-C-1402
Conditions: Friedreich Ataxia
Keywords: RTA 408, RTA 408 Capsules, Oxidative Stress, Mitochondrial dysfunction, omaveloxolone
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 172 participants (actual)
Lead sponsor: BiogenIndustry (pharma, biotech, devices)
Collaborators: AbbVie, Friedreich's Ataxia Research Alliance
Study start: 31 Jan 2015 (actual)
Primary completion: 31 Oct 2019 (actual)
Study completion: 19 Dec 2025 (actual)
First posted: 2 Oct 2014 (estimated)
Results: Posted 29 Nov 2022

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The main questions researchers want to answer in this study are:

How much physical effort can a participant produce during a cycling test after 12 weeks of treatment?
How do scores on the modified Friedreich's Ataxia Rating Scale (mFARS) change after 48 weeks?

They will also use a cycling test to measure physical effort, along with questionnaires to learn how participants feel and function in daily life.

Safety will also be tested using physical exams, vital sign checks, echocardiograms (ECHO), electrocardiograms (ECG), and blood and urine tests.

The study will be done in 2 main parts, followed by an optional Extension period:

In Part 1, participants will be randomly assigned to take different doses of RTA 408 or a placebo by mouth once a day for 12 weeks. A placebo looks like the study drug but contains no real medicine.
Researchers will compare these doses to decide which one to use in Part 2.
In Part 2, a different group of participants will take either the chosen dose of RTA 408 (150 mg) or placebo once a day for 48 weeks.
Participants who complete Part 1 or Part 2 may be able to join an Extension period, where everyone receives RTA 408.
In the Extension period, participants will continue to receive RTA 408 until the drug becomes commercially available or until they leave the study
Participants in Part 1 will have up to 9 study visits and 2 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 20 weeks.
Participants in Part 2 will have up to 10 study visits and 3 phone calls. If they do not move onto the Extension period, they will stay in the study for up to 61 weeks.
Participants in the Extension period will have 2 visits in the first month, followed by visits every 6 months.

Detailed description

This 2-part study will evaluate the efficacy, safety, and pharmacodynamics of omaveloxolone (RTA 408) in the treatment of participants with Friedreich's ataxia.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 12

Arm	Type	Receives
Part 1: Omaveloxolone 2.5 and 5 mg	Experimental	Drug: Omaveloxolone Capsules, 2.5 mg, Drug: Omaveloxolone Capsules, 5 mgParticipants received omaveloxolone 2.5 mg, oral capsule, QD for 2 weeks, followed by 5 mg Omaveloxolone, oral capsule, QD for 10 weeks.
Part 1: Omaveloxolone 10 mg	Experimental	Drug: Omaveloxolone Capsules, 10 mgParticipants received omaveloxolone 10 mg, oral capsule, QD for 12 weeks.
Part 1: Omaveloxolone 20 mg	Experimental	Drug: Omaveloxolone Capsules, 20 mgParticipants received omaveloxolone 20 mg, oral capsule, QD for 12 weeks.
Part 1: Omaveloxolone 40 mg	Experimental	Drug: Omaveloxolone Capsules, 40 mgParticipants received omaveloxolone 40 mg, oral capsule, QD for 12 weeks.
Part 1: Omaveloxolone 80 mg	Experimental	Drug: Omaveloxolone Capsules, 80 mgParticipants received omaveloxolone 80 mg, oral capsule, QD for 12 weeks.
Part 1: Omaveloxolone 160 mg	Experimental	Drug: Omaveloxolone Capsules, 160 mgParticipants received omaveloxolone 160 mg, oral capsule, QD for 12 weeks.
Part 1: Omaveloxolone 300 mg	Experimental	Drug: Omaveloxolone Capsules, 300 mgParticipants received omaveloxolone 300 mg, oral capsule, QD for 12 weeks.
Part 1: Placebo	Placebo comparator	Drug: PlaceboParticipants received placebo, oral capsule, QD for 12 weeks.
Part 2: Omaveloxolone 150 mg	Experimental	Drug: Omaveloxolone Capsules, 150 mgParticipants received omaveloxolone 150 mg, oral capsule, QD for 48 weeks.
Part 2: Placebo	Placebo comparator	Drug: PlaceboParticipants received placebo, oral capsule, QD for 48 weeks.
OLE: Placebo/ Omaveloxolone	Experimental	Drug: Omaveloxolone Capsules, 150 mgAll eligible participants who had received either omaveloxone or placebo in Part 1 and eligible participants who received placebo in Part 2 received omaveloxone, 150 mg, oral capsule, QD, until the drug was available through commercial channels or alternate post-trial access mechanisms, or until participant withdrawal, whichever was sooner (up to approximately 370 weeks).
OLE: Omaveloxolone/ Omaveloxolone	Experimental	Drug: Omaveloxolone Capsules, 150 mgAll eligible participants who had received omaveloxone in Part 2 continued to receive omaveloxolone, 150 mg, oral capsule, QD until the drug was available through commercial channels or alternate post-trial access mechanisms, or until participant withdrawal, whichever was sooner (up to approximately 370 weeks).

Who may qualify

Ages: 16 to 40 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (6 listed)

Have genetically confirmed Friedreich's ataxia
Have a modified FARS score ≥20 and ≤80
Be male or female and ≥16 years of age and ≤40 years of age
Have no changes to exercise regimen within 30 days prior to Study Day 1 and be willing to remain on the same exercise regimen during the 16-week study period
Have the ability to complete maximal exercise testing

Key exclusion criteria (14 listed)

Have uncontrolled diabetes (HbA1c >11.0%)
Have B-type natriuretic peptide value >200 pg/mL
Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease
Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C)
Have known or suspected active drug or alcohol abuse

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
Omaveloxolone Capsules, 2.5 mgAlso: RTA 408 Capsules 2.5 mg	Drug	Part 1: Omaveloxolone 2.5 and 5 mg
Omaveloxolone Capsules, 5 mgAlso: RTA 408 capsules, 5 mg	Drug	Part 1: Omaveloxolone 2.5 and 5 mg
Omaveloxolone Capsules, 10 mgAlso: RTA 408 capsules, 10 mg	Drug	Part 1: Omaveloxolone 10 mg
Placebo	Drug	Part 1: Placebo, Part 2: Placebo
Omaveloxolone Capsules, 20 mgAlso: RTA 408 capsules, 20 mg	Drug	Part 1: Omaveloxolone 20 mg
Omaveloxolone Capsules, 40 mgAlso: RTA 408 capsules, 40 mg	Drug	Part 1: Omaveloxolone 40 mg
Omaveloxolone Capsules, 80 mgAlso: RTA 408 capsules, 80 mg	Drug	Part 1: Omaveloxolone 80 mg
Omaveloxolone Capsules, 160 mgAlso: RTA 408 capsules, 160 mg	Drug	Part 1: Omaveloxolone 160 mg
Omaveloxolone Capsules, 300 mgAlso: RTA 408 capsules, 300 mg	Drug	Part 1: Omaveloxolone 300 mg
Omaveloxolone Capsules, 150 mgAlso: RTA 408 capsules, 150 mg	Drug	OLE: Omaveloxolone/ Omaveloxolone, OLE: Placebo/ Omaveloxolone, Part 2: Omaveloxolone 150 mg

Primary outcome

Part 1: Change From Baseline in Peak Work During Maximal Exercise Testing at Week 12
Time frame: Baseline, Week 12
Cycle ergometry using a recumbent stationary bicycle was used to conduct maximal exercise testing and workload was increased incrementally. Peak work is defined as the workload at which participants reach maximal volition (defined as an inability to continue to exercise due to exhaustion). A positive change from baseline suggests an improvement.
Part 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
Time frame: From first dose of study drug up to end of Part 1 of the study (up to Week 16)
An adverse event (AE) was any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug whether or not it is considered to be study drug related. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. TEAEs were defined as any AEs, regardless of relationship to study drug, that had an onset or worsened in severity on or after the first dose of study drug.
Part 2: Change From Baseline in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48
Time frame: Baseline, Week 48
The Friedreich Ataxia Rating Scale (FARS) is a neurological-exam-based rating scale with five sections: Bulbar (section A), Upper Limb Coordination (section B), Lower Limb Coordination (section C), Peripheral Nervous System (section D), and Upright Stability (section E). mFARS is the sum of sections A (score 0 to 11), B (score 0 to 36), C (score 0 to 16), and E (score 0 to 36). The minimum score is 0 and the maximum score is 99. A lower score indicates better neurological function.
Part 2: Number of Participants With TEAEs and TESAEs
Time frame: From first dose of study drug up to end of Part 2 of the study (up to Week 52)
An AE was any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug whether or not it is considered to be study drug related. An SAE was any untoward medical occurrence that at any dose resulted in death, in the view of the Investigator, placed the participant at immediate risk of death, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, resulted in a birth defect. TEAEs were defined as any AEs, regardless of relationship to study drug, that had an onset or worsened in severity on or after the first dose of study drug.

8 secondary outcomes

Part 1: Change From Baseline in the mFARS at Week 12
Time frame: Baseline, Week 12
Part 2: Number of Participants With Patient Global Impression of Change (PGI-C)
Time frame: Week 48
Part 2: Number of Participants With Clinical Global Impression of Change (CGI-C)
Time frame: Week 48
Part 2: Change From Baseline in Performance on a 9-Hole Peg Test (9-HPT) at Week 48
Time frame: Baseline, Week 48
Part 2: Change From Baseline in Performance on a 25-Foot Timed Walk Test (T25-FWT) at Week 48
Time frame: Baseline, Week 48
Part 2: Total Number of Falls
Time frame: Up to Week 48
Part 2: Change From Baseline in Peak Work During Maximal Exercise Testing at Week 48
Time frame: Baseline, Week 48
Part 2: Change From Baseline in the Activities of Daily Living (ADL) Score at Week 48
Time frame: Baseline, Week 48

Results and safety

Results posted to the registry on 29 Nov 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Part 1: Omaveloxolone 2.5 mg Followed by 5 mg	6	6
Part 1: Omaveloxolone 10 mg	6	6
Part 1: Omaveloxolone 20 mg	6	6
Part 1: Omaveloxolone 40 mg	6	6
Part 1: Omaveloxolone 80 mg	6	6
Part 1: Omaveloxolone 160 mg	12	12
Part 1: Omaveloxolone 300 mg	10	10
Part 1: Placebo	17	16
Part 2: Omaveloxolone 150 mg	0	0
Part 2: Placebo	0	0
OLE: Placebo/ Omaveloxolone	0	0
OLE: Omaveloxolone/ Omaveloxolone	0	0

Primary outcome: Part 1: Change From Baseline in Peak Work During Maximal Exercise Testing at Week 12

Time frame: Baseline, Week 12. Least squares mean (95% Confidence Interval). Unit: Watts per kilogram (W/kg)

Measure	Part 1: Omaveloxolone 2.5 mg Followed by 5 mg n = 4	Part 1: Omaveloxolone 10 mg n = 4	Part 1: Omaveloxolone 20 mg n = 3	Part 1: Omaveloxolone 40 mg n = 4	Part 1: Omaveloxolone 80 mg n = 4	Part 1: Omaveloxolone 160 mg n = 4	Part 1: Omaveloxolone 300 mg n = 7	Part 1: Placebo n = 7
Value	0.14 (0.02 to 0.26)	0.07 (-0.05 to 0.18)	-0.09 (-0.20 to 0.03)	0.06 (-0.06 to 0.18)	0 (-0.11 to 0.12)	0.02 (-0.06 to 0.10)	0.07 (-0.02 to 0.17)	0.04 (-0.03 to 0.11)

Part 1: Omaveloxolone 2.5 mg Followed by 5 mg vs Part 1: Placebo: Least Square (LS) Mean Difference (Net): 0.1 (95% CI -0.04 to 0.23), p =0.1524, Mixed models repeated measures (MMRM)
Part 1: Omaveloxolone 10 mg vs Part 1: Placebo: LS Mean Difference (Net): 0.03 (95% CI -0.11 to 0.16), p =0.7086, MMRM
Part 1: Omaveloxolone 20 mg vs Part 1: Placebo: LS Mean Difference (Net): -0.13 (95% CI -0.26 to 0.01), p =0.0651, MMRM

Population analysed: ITT population included all enrolled participants in Part 1, whether or not they received the study drug. Here, 'overall number of participants analyzed' signifies the number of participants with data available for outcome measure analysis.

Primary outcome: Part 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)

Time frame: From first dose of study drug up to end of Part 1 of the study (up to Week 16). Count of participants. Unit: Participants

Measure	Part 1: Omaveloxolone 2.5 mg Followed by 5 mg n = 6	Part 1: Omaveloxolone 10 mg n = 6	Part 1: Omaveloxolone 20 mg n = 6	Part 1: Omaveloxolone 40 mg n = 6	Part 1: Omaveloxolone 80 mg n = 6	Part 1: Omaveloxolone 160 mg n = 12	Part 1: Omaveloxolone 300 mg n = 10	Part 1: Placebo n = 17
TEAEs	6	5	5	6	6	11	10	16
TESAEs	0	0	0	0	0	0	0	2

Population analysed: The safety population included all participants who received at least 1 dose of study drug.

Primary outcome: Part 2: Change From Baseline in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48

Time frame: Baseline, Week 48. Mean (Standard Deviation). Unit: score on a scale

Measure	Part 2 Omaveloxolone 150 mg n = 34	Part 2: Placebo n = 41
Value	-1.45 (4.209)	0.90 (4.305)

Part 2 Omaveloxolone 150 mg vs Part 2: Placebo: LS Mean difference (Net): -2.40 (95% CI -4.31 to -0.50), p = 0.0141, MMRM

Population analysed: The full analysis set (FAS) included participants enrolled without pes cavus who had at least 1 post-baseline measurement, categorized by their randomized treatment group (whether or not they received study drug). Here, 'overall number of participants analyzed' signifies the number of participants with data available for outcome measure analysis.

Primary outcome: Part 2: Number of Participants With TEAEs and TESAEs

Time frame: From first dose of study drug up to end of Part 2 of the study (up to Week 52). Count of participants. Unit: Participants

Measure	Part 2: Omaveloxolone 150 mg n = 51	Part 2: Placebo n = 52
TEAEs	51	52
TESAEs	5	3

Population analysed: The safety population included all participants who received at least 1 dose of the randomized study drug.

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: From first dose of study drug up to end of OLE period (up to approximately 130 months). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Part 1: Omaveloxolone 2.5 mg Followed by 5 mg	0 of 6 (0%)	6 of 6 (100%)	0 of 6
Part 1: Omaveloxolone 10 mg	0 of 6 (0%)	5 of 6 (83.3%)	0 of 6
Part 1: Omaveloxolone 20 mg	0 of 6 (0%)	5 of 6 (83.3%)	0 of 6
Part 1: Omaveloxolone 40 mg	0 of 6 (0%)	6 of 6 (100%)	0 of 6
Part 1: Omaveloxolone 80 mg	0 of 6 (0%)	6 of 6 (100%)	0 of 6
Part 1: Omaveloxolone 160 mg	0 of 12 (0%)	11 of 12 (91.7%)	0 of 12
Part 1: Omaveloxolone 300 mg	0 of 10 (0%)	10 of 10 (100%)	0 of 10
Part 1: Placebo	2 of 17 (11.8%)	16 of 17 (94.1%)	0 of 17
Part 2: Omaveloxolone 150 mg	5 of 51 (9.8%)	51 of 51 (100%)	0 of 51
Part 2: Placebo	3 of 52 (5.8%)	50 of 52 (96.2%)	0 of 52
OLE: Placebo/ Omaveloxolone	12 of 106 (11.3%)	103 of 106 (97.2%)	0 of 106
OLE: Omaveloxolone/ Omaveloxolone	13 of 43 (30.2%)	42 of 43 (97.7%)	0 of 43

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 40614535Perlman S, Zhang S, Setyawan J, Yang H, Jiang A, Hua Q, Boudreau J, Khan S, Bajaj A, Lynch DR, Lawson R. The clinical burden of Friedreich ataxia in the United States: A retrospective claims database analysis. J Neurol Sci. 2025 Aug 15;475:123594. doi: 10.1016/j.jns.2025.123594. Epub 2025 Jun 25.
Derived from this study: PubMed 33068037Lynch DR, Chin MP, Delatycki MB, Subramony SH, Corti M, Hoyle JC, Boesch S, Nachbauer W, Mariotti C, Mathews KD, Giunti P, Wilmot G, Zesiewicz T, Perlman S, Goldsberry A, O'Grady M, Meyer CJ. Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study). Ann Neurol. 2021 Feb;89(2):212-225. doi: 10.1002/ana.25934. Epub 2020 Nov 5.
Derived from this study: PubMed 30656180Lynch DR, Farmer J, Hauser L, Blair IA, Wang QQ, Mesaros C, Snyder N, Boesch S, Chin M, Delatycki MB, Giunti P, Goldsberry A, Hoyle C, McBride MG, Nachbauer W, O'Grady M, Perlman S, Subramony SH, Wilmot GR, Zesiewicz T, Meyer C. Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia. Ann Clin Transl Neurol. 2018 Nov 10;6(1):15-26. doi: 10.1002/acn3.660. eCollection 2019 Jan.
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. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

11 sites in 5 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg and Omaveloxolone Capsules, 10 mg in people with Friedreich Ataxia, run by Biogen at 11 sites in 5 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 6 inclusion and 14 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: finished, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in United States, Australia, Austria, Italy, United Kingdom. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 29 Nov 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part 1: Change From Baseline in Peak Work During Maximal Exercise Testing at Week 12 (time frame: Baseline, Week 12). 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?

On the ClinicalTrials.gov record for NCT02255435, which also lists 3 linked publications.

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 3 Sep 2026.