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

Completed · Phase 2 · Has a placebo group

Conditions studied: Friedreich Ataxia

In brief

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. 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.

Key facts

Study ID

NCT02255435

Run by

Biogen

People needed

172

Starts

2015-01-31

Expected to finish

2025-12-19

Last updated by the study team

2026-01-23

Who can join

Age: 16 and older, up to 40. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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
• 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)

Where it is running

• UCLA — Los Angeles, California, United States
• University of Florida - Neurology — Gainesville, Florida, United States
• USF Ataxia Research Center — Tampa, Florida, United States
• Emory University Hospital - Neurology — Atlanta, Georgia, United States
• University of Iowa Stead Family Children's Hospital — Iowa City, Iowa, United States
• Ohio State University - Neurology — Columbus, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Murdoch Childrens Research Institute — Parkville, Victoria, Australia
• Medical University Innsbruck — Innsbruck, Austria
• Neurological Institute Carlo Besta — Milan, Italy
• University College of London — London, United Kingdom

Full record on ClinicalTrials.gov

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