Condition: Friedreich Ataxia · Sponsor: Biogen
In this study, researchers will learn more about omaveloxolone, also known as BIIB141 or SKYCLARYS®. Omaveloxolone is already approved for people with Friedreich's Ataxia (FA) who are 16 years of age or older. However, it is not yet available for younger teens and children. The main goal of this study is to learn how omaveloxolone affects symptoms of FA and its safety in younger participants between the ages of 2 and 15 years old. The main questions researchers want to answer in this study are: * How does omaveloxolone affect the participants' FA symptoms? * How many participants have adverse events during the study? * Are there any changes in the participants' overall health or heart health? Adverse events are health problems that may or may not be caused by the study drug. Researchers will use the modified Friedreich's Ataxia Rating Scale (mFARS) to test nerve function. The mFARS tests movement ability, balance, coordination, speech, and arm and leg functions. They will also use a number of questionnaires to learn more about participants' quality of life, muscle strength, and ability to perform daily tasks. Researchers will also note any changes as participants go through puberty. Finally, researchers will learn more about how the body processes omaveloxolone in children and teenagers. This study will be done in 2 parts as follows: * Participants will be screened for up to 4 weeks to check if they can join the study. * In Part 1, participants will be randomly assigne…
This description comes directly from the study's public registry record.
Patient Navigator · 1-877-223-3576 · biogenBRAVE_patientnavigator@thermofisher.com
US Biogen Clinical Trial Center · 866-633-4636 · clinicaltrials@biogen.com
Always discuss trial participation with your own doctor first.
| UCLA Neurology Outpatient Clinic at Westwood | Los Angeles, California, United States | Not Yet Recruiting |
| Norman Fixel Institute for Neurological Diseases UF Health | Gainesville, Florida, United States | Recruiting |
| USF Health Morsani College of Medicine Department of Neurology | Tampa, Florida, United States | Recruiting |
| Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN | Philadelphia, Pennsylvania, United States | Recruiting |
| St. Jude Children's Research Hospital - PIN | Memphis, Tennessee, United States | Recruiting |
| CHKD's Health Center - South Campus - PIN | Norfolk, Virginia, United States | Recruiting |
| Seattle Children's Hospital | Seattle, Washington, United States | Recruiting |
| Sydney Children's Hospital | Randwick, New South Wales, Australia | Not Yet Recruiting |
| Murdoch Childrens Research Institute (MCRI) | Parkville, Victoria, Australia | Recruiting |
| Universitätsklinikum Innsbruck | Innsbruck, Austria | Recruiting |
| L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME | Brasília, Federal District, Brazil | Recruiting |
| University of Campinas (UNICAMP) School of Medical Sciences | Campinas, São Paulo, Brazil | Not Yet Recruiting |
+ 22 more locations — full list on the registry record.
Get one email when the public record changes — results posted, or the study's status changes. Nothing else, ever.
We email about this public record only. Unsubscribe anytime with one click. Never medical advice.
This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.
Source record: clinicaltrials.gov/study/NCT06953583