RecruitingPhase 3NCT06953583

A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia

A Phase 3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 Years


Sponsor

Biogen

Enrollment

255 participants

Start Date

Jun 9, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

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 assigned to take either omaveloxolone or a placebo by mouth once a day for about 1 year. A placebo looks like the study drug but contains no real medicine. * Part 1 will be double blind. This means that the participants, study doctor, and site staff will not know if the participants are receiving omaveloxolone or a placebo. * Including screening, participants will have up to 9 clinic visits and 1 phone call during Part 1. If a participant does not join Part 2, they will have another safety follow-up phone call a month after their last dose of omaveloxolone. * Participants who complete Part 1 will move onto Part 2 where everyone will receive omaveloxolone for about 2 years. * During Part 2, participants will have up to 8 clinic visits and 1 phone call. Participants will also have a follow-up phone call about a month after they stop taking omaveloxolone. * In total, participants will have up to 17 clinic visits and 3 phone calls. Each participant will be in the study for up to 3 years.


Eligibility

Min Age: 2 YearsMax Age: 15 Years

Inclusion Criteria2

  • Diagnosed with genetically confirmed Friedreich's Ataxia (FA), i.e., homozygous for guanine-adenine-adenine (GAA) repeat expansion in intron-1 of the frataxin gene, or GAA repeat expansion in 1 allele and with point mutations or deletions, or other non-GAA expansion mutations in the other allele.
  • Symptomatic for FA as confirmed by clinician assessment. a. Children 7 to < 16 years must also have an upright stability score (USS) score of 10 to ≤ 34 at baseline

Exclusion Criteria10

  • Glycosylated hemoglobin A1C (HbA1c) > 11%
  • B-type natriuretic peptide (BNP) > 200 picograms per milliliter (pg/mL) at screening
  • Ejection fraction (EF) < 40% \[based on echocardiogram (ECHO) performed at screening visit\]
  • Clinically significant cardiac disease except mild to moderate cardiomyopathy
  • Part 2A: Eligibility criteria:
  • They have completed Part 1 of the study and no discontinuation criteria have been met.
  • Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the investigator.
  • Part 2B: Eligibility criteria:
  • Participants have completed Part 1 of the study and no discontinuation criteria have been met.
  • Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the Investigator.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGOmaveloxolone

Administered as specified in the treatment arm.

DRUGPlacebo

Administered as specified in the treatment arm.


Locations(34)

UCLA Neurology Outpatient Clinic at Westwood

Los Angeles, California, United States

Norman Fixel Institute for Neurological Diseases UF Health

Gainesville, Florida, United States

USF Health Morsani College of Medicine Department of Neurology

Tampa, Florida, United States

Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN

Philadelphia, Pennsylvania, United States

St. Jude Children's Research Hospital - PIN

Memphis, Tennessee, United States

CHKD's Health Center - South Campus - PIN

Norfolk, Virginia, United States

Seattle Children's Hospital

Seattle, Washington, United States

Sydney Children's Hospital

Randwick, New South Wales, Australia

Murdoch Childrens Research Institute (MCRI)

Parkville, Victoria, Australia

Universitätsklinikum Innsbruck

Innsbruck, Austria

L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME

Brasília, Federal District, Brazil

University of Campinas (UNICAMP) School of Medical Sciences

Campinas, São Paulo, Brazil

PSEG Centro de Pesquisa Clinica

São Paulo, São Paulo, Brazil

McGill University

Montreal, Quebec, Canada

CHU de Quebec -Universite Laval

Québec, Quebec, Canada

Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen

Copenhagen, Denmark

CHU de Montpellier- Hôpital Gui De Chauliac

Montpellier, Hérault, France

AP-HP - Hôpital Armand Trousseau

Paris, France

Universitätsklinikum Aachen

Aachen, North Rhine-Westphalia, Germany

UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen

Giessen, Germany

Universitätsklinikum Hamburg Eppendorf

Hamburg, Germany

All India Institute of Medical Sciences (AIIMS) - New Delhi

New Delhi, National Capital Territory of Delhi, India

CHI at Temple Street

Dublin, Ireland

Ospedale Pediatrico Bambino Gesù IRCCS

Rome, Lazio, Italy

IRCCS Eugenio Medea - Polo. Scientifico Veneto

Conegliano, Veneto, Italy

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Italy

Radboud Universitair Medisch Centrum

Nijmegen, Netherlands

King Faisal Specialist Hospital & Research Centre

Riyadh, Ar Riya, Saudi Arabia

Hospital Sant Joan de Deu - PIN

Espluges de Llobregat, Barcelona, Spain

Hospital Universitario La Paz - PPDS

Madrid, Spain

Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

Istanbul, Turkey (Türkiye)

University College Hospital - PPDS

London, Lincolnshire, United Kingdom

John Radcliffe Hospital

Oxford, Oxfordshire, United Kingdom

Sheffield Children's Hospital - PPDS

Sheffield, South Yorkshire, United Kingdom

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT06953583


Related Trials