Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New drug shows promise for rare movement disorder

NCT ID NCT02255435

Disease control Sponsor: Biogen Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 04, 2026 · Updated 2 times

Summary

This study tested a drug called RTA 408 (omaveloxolone) in 172 people with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to see if the drug is safe and can improve exercise ability and daily function. Participants took the drug or a placebo for up to 48 weeks, and researchers measured changes in cycling tests and a rating scale for movement.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

172 people

The number who actually took part.

Started

Jan 2015

Finished

Dec 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

16 to 40 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Have genetically confirmed Friedreich's ataxia 2. Have a modified FARS score ≥20 and ≤80 3. Be male or female and ≥16 years of age and ≤40 years of age 4. 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 5. Have the ability to complete maximal exercise testing 6. Be able to swallow capsules Exclusion Criteria: 1. Have uncontrolled diabetes (HbA1c \>11.0%) 2. Have B-type natriuretic peptide value \>200 pg/mL 3. Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease 4. Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C) 5. Have known or suspected active drug or alcohol abuse 6. 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 7. 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 8. 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: 1. Sensitive substrates for cytochrome P450 2C8 or 3A4 (e.g., repaglinide, midazolam, sildenafil) 2. Moderate or strong inhibitors or inducers of cytochrome P450 3A4 (e.g., carbamazepine, phenytoin, ciprofloxacin, grapefruit juice) 3. Substrates for p-glycoprotein transporter (e.g., ambrisentan, digoxin) 9. Have participated in any other interventional clinical study within 30 days prior to Study Day 1 10. Have a cognitive impairment that may preclude ability to comply with study procedures 11. Prior participation in a trial with omaveloxolone (RTA 408)

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Friedreich ataxia are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

Friedreich ataxia inborn mitochondrial metabolism disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Friedreich ataxia

Contacts and locations

Locations

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Emory University Hospital - Neurology

    Atlanta, Georgia, 30329, United States

  • Medical University Innsbruck

    Innsbruck, 6020, Austria

  • Murdoch Childrens Research Institute

    Parkville, Victoria, 3052, Australia

  • Neurological Institute Carlo Besta

    Milan, 20133, Italy

  • Ohio State University - Neurology

    Columbus, Ohio, 43221, United States

  • UCLA

    Los Angeles, California, 90095, United States

  • USF Ataxia Research Center

    Tampa, Florida, 33612, United States

  • University College of London

    London, WC1E 6BT, United Kingdom

  • University of Florida - Neurology

    Gainesville, Florida, 32610, United States

  • University of Iowa Stead Family Children's Hospital

    Iowa City, Iowa, 52242, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.