A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants with Friedreich’s Ataxia Aged 2 to 15 Years Old (BRAVE)

Study Overview

In this study, researchers will learn more about the effects and safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedreich’s Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 works in the body and about its safety in children and teens who are 2 to 15 years old.

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

How does BIIB141 affect the participants’ FA symptoms balance and stability?
How many participants have medical problems during the study?
Are there any changes in the participants’ overall health during the study?
Are there any changes in the participants’ heart health?
Are there any changes in how the participants move through puberty? Puberty is the time in someone’s life when their body changes from a child to an adult.
How does the body process BIIB141 in children and teens?

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Study Details

There are 2 parts in this study. During Part 1, participants will take either BIIB141 or a placebo once a day for 52 weeks. After completion of Part 1, participants will be invited to participate in Part 2 during which all participants will take BIIB141 for up to 104 weeks.

Participants will be screened to check to see if they are eligible to join the study. Participants who meet all the eligibility criteria will be invited to begin the study at a designated study research center.

In Part 1, participants will take BIIB141 or the placebo in a study research center on Day 1, and then at in-person visits at Week 4, Week 12, Week 26, and Week 52. On all other days, they will take BIIB141 or the placebo at home. Part 1 lasts up to 52 weeks. Participants will have up to 10 visits to their study research center and a phone call at Week 2.

During Part 2, participants from Part 1 will either continue taking BIIB141 or start it if they were taking the placebo. In Part 2, participants will have visits at Weeks 4, 8,12, 26, and every 26 weeks after that until they leave the study, and a phone call at Week 2. There will be a final phone call to check on the participants’ health 31 days after their last dose. Part 2 will last up to 104 weeks.

Each participant will be in the study up to approximately 3 years.

Key Inclusion Criteria for Part 1:

Diagnosed with genetically confirmed Friedreich’s ataxia (FA). This includes being homozygous for GAA repeat expansions, or having a GAA repeat expansion on one allele and a point mutation, deletion, or other non GAA repeat expansion mutation on the other allele.
Symptomatic for FA as reported by the participant and/or their parent or caregiver.
Children 7 to 15 must have an upright stability score on the modified Friedreich’s Ataxia Rating Scale (mFARS) between 10 and 34 at baseline.

Key Exclusion Criteria for Part 1:

Glycosylated hemoglobin A1C (HbA1c) > 11% (indicator of poorly controlled diabetes)
B-type natriuretic peptide (BNP) > 200 pg/mL at screening (measure of heart health)
Ejection fraction <40% as measured by echocardiogram at screening visit (measure of heart health)
Clinically significant cardiac disease except mild to moderate cardiomyopathy

Open Label Extension (Part 2) Eligibility:

Participants have completed Part 1 and no discontinuation criteria have been met.
Safety and tolerability data from Part 1 are supportive of continuation in the judgement of the investigator.

Other inclusion/exclusion criteria may apply.

clinicaltrials.gov

Additional Information on Biogen's website

Link to the BRAVE study on Biogen's clinical trial website

Study Contact for More Information

To learn more about this study, email Biogen at clinicaltrials@biogen.com or call the Patient Navigator line at 1-877-223-3576 (access code 57078). The Patient Navigator can share information about different sites, whether they are actively enrolling or soon to be active, and help you determine which study site works best for you.

Participating Study Locations

Institution Name	Location	Status	Contact
Seattle Children’s Hospital	USA Seattle, WA	Active, recruiting	Principal Investigator: Dr. Henriquez Study Coordinators: Macarena Racciatti, Macarena.Racciatti@seattlechildrens.org Marissa Robertson, Marissa.Robertson@seattlechildrens.org
UCLA Neurology Outpatient Clinic at Westwood	USA Los Angeles, CA	Not yet recruiting
Norman Fixel Institute for Neurological Diseases UF Health	USA Gainesville, FL	Active, recruiting	352-733-3032 s.subramony@neurology.ufl.edu
USF Health Morsani College of Medicine Department of Neurology	USA Tampa, FL	Active, recruiting	Principal Investigator: Theresa Zesiewicz Phone: 813-974-5909 Coordinator: Kayla Zayas Email: kaylazayas@usf.edu Coordinator: Lucretia Campbell Email: lcampbel@usf.edu
Children’s Hospital of Philadelphia Buerger Center	USA Philadelphia, PA	Active, recruiting	Principal Investigator: David Lynch Phone: 215-590-2242 Email: lynchd@pennmedicine.upenn.edu
St Jude Children’s Research Hospital	USA Memphis, TN	Active, recruiting	Principal Investigator: Richard Finkel Phone: 407-650-7250 Email: richard.finkel@stjude.org
Children’s Hospital of the King’s Daughters	USA Norfolk, VA	Active, recruiting	Principal Investigator: Crystal Proud Phone: 757-668-6981 Email: Proud.research@chkd.org
Murdoch Childrens Research Institute (MCRI)	Australia Parkville, Victoria	Active, recruiting	varlli.scott@mcri.edu.au
Universitätsklinikum Innsbruck	Austria Innsbruck	Active, recruiting	Principal Investigator: Sylvia Boesch Phone: +43 5125042 3850 Email: sylvia.boesch@i-med.ac.at
PSEG Centro de Pesquisa Clinica	Brazil São Paulo	Active, recruiting	Principal Investigator: Paulo Sgobbi de Souza Phone: +55 11 99902-7052 Email: pvsgobbi@gmail.com
L2 Ip – Instituto de Pesquisas Clinicas Ltda – ME	Brazil Brasília, Distrito Federal	Active, recruiting	Principal Investigator: Eduardo Vasconellos Phone: +55 61 3445-4300 Email: eduardo.vasconcellos@l2ip.com.br
University of Campinas (UNICAMP) School of Medical Sciences	Brazil Campinas, São Paulo	Active, recruiting	Principal Investigator: Marcondes Franca Phone: +55 19 3521 8922 Email: mcfrancajr@uol.com.br
CHU de Quebec – Universite Laval	Canada Quebec	Active, recruiting	Principal Investigator: Nicolas Chrestian Phone: 418-525-4444 ext 71801 Email: nicolas.chrestian.med@ssss.gouv.qc.ca
McGill University	Canada Montreal, Quebec	Active, recruiting	Principal Investigator: Maryam Oskoui Phone: 514-412-4466 Email: maryam.oskoui@mcgill.ca
Rigshospitalet – Juliane Marie Centret (JMC) Copenhagen	Denmark Copenhagen	Active, recruiting	Principal Investigator: Alfred Peter Born Phone: +45 35-45-50-93 Email: alfred.peter.born@regionh.dk
AP-HP – Hôpital Armand Trousseau	France Paris	Active, recruiting	Principal Investigator: Florence Renaldo Phone: 01 85 34 00 29 Email: Florence.renaldo@aphp.fr
CHU de Montpellier – Hôpital Arnaud de Villeneuve	France Montpellier, Hérault	Active, recruiting	Principal Investigator: Agathe Roubertie Phone: 33 04 67 33 01 82 Email: a-roubertie@chu-montpellier.fr
Universitätsklinikum Aachen	Germany Aachen	Active, recruiting	Principal Investigator: Kathrin Reetz Phone: +49 24180 89600 Email: kreetz@ukaachen.de
UKGM – Universitätsklinikum Giessen und Marburg GmbH – Standort Gießen	Germany Giessen	Active, recruiting	Principal Investigator: Andreas Hahn Phone: +49 641 985 43543 Email: andreas.hahn@paediat.med.uni-giessen.de
UKE Hamburg	Germany Hamburg	Active, recruiting	Principal Investigator: Deike Weiss Phone: +49 40 7410 56126 Email: d.weiss@uke.de
CHI at Temple Street	Ireland Dublin	Active, recruiting	Principal Investigator: Declan O’Rourke Phone: (353) 187-8472 ext 2 Email: declan.orourke@cuh.ie
Fondazione IRCCS Istituto Neurologico Carlo Besta	Italy Milano	Active, recruiting	Principal Investigator: Isabella Moroni Phone: +39 022394 2210 Email: isabella.moroni@istituto-besta.it
Ospedale Pediatrico Bambino Gesù IRCCS	Italy Roma, Lazio	Not yet recruiting
IRCCS Eugenio Medea – Polo. Scientifico Veneto	Italy Conegliano, Veneto	Active, recruiting	Principal Investigator : Gabriella Paparella Phone: +39 3383065324 Email: gabriella.paparella@lanostrafamiglia.it
Radboud Universitair Medisch Centrum	Netherlands Nijmegen	Active, recruiting	Principal Investigator: Nienke van Os Phone: 31 243614415
Hospital Sant Joan de Deu	Spain Espluges De Llobregat	Active, recruiting	Principal Investigator: Alejandra Darling Phone: +34 93 253 21 00 ext 71465 Email: alejandra.darling@sjd.es
Hospital Universitario La Paz – PPDS	Spain Madrid	Active, recruiting	Principal Investigator: Maria del Mar Garcia Romero Phone: +34 91 7277388 Email: yambee@hotmail.com
University College London, Institute of Neurology	United Kingdom London	Active, recruiting	Principal Investigator: Shpresa Pula Phone: +44 773046 1357 Email: shpresa.pula1@nhs.net
John Radcliffe Hospital	United Kingdom Oxford	Active, recruiting	Principal Investigator: Andrea Németh Phone: 44 1865 231556
Sheffield Children’s Hospital	United Kingdom Sheffield, South Yorkshire	Active, recruiting	Principal Investigator: Santosh Mordekar Phone: 0114 226 0675 Email: santosh.mordekar@nhs.net

Explore the FA Drug Development Pipeline

FARA believes that there are many different approaches to treating Friedreich’s ataxia, and that it will require a cocktail approach of two or more treatments to slow, stop, reverse, and cure FA. Learn more about the approach behind this potential treatment and explore the other approaches that are in the FA Drug Development pipeline.

Omaveloxolone Pipeline Overview