Clinical trial • Phase III • Neurology|Rare Disease
Omaveloxolone for Friedreich's ataxia
Phase III trial of Omaveloxolone for Friedreich's ataxia.
Overview
- Trial Therapeutic Area
- Neurology|Rare Disease
- Trial Disease
- Friedreich's ataxia
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 07-07-2025
- First CTIS Authorization Date
- 28-10-2025
Trial design
Randomised, open-label, omaveloxolone (biib141) qd vs placebo (hard capsules) qd (doses not specified in provided data)-controlled Phase III trial in Spain, Austria, Denmark and others.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Omaveloxolone (BIIB141) QD vs Placebo (Hard capsules) QD (doses not specified in provided data)
- Target Sample Size
- 199
Eligibility
Recruits 199 paediatric patients.
- Vulnerable Population
- Children aged 2 to < 16 years (vulnerable population). Parental permission/consent and age-appropriate assent are required; multiple country- and age-specific parental ICF and assent documents are provided (examples in documents: Parental-Permission-ICF, Assent forms for ages 2-5, 6-11, 12-14, 15-16, and Optional-Future-Research/Genetic research assent/ICF documents).
Inclusion criteria
- {"criterion_text":"- Part 1 • Aged 2 to < 16 years at the time of informed consent.\n- •Diagnosed with genetically confirmed FA, i.e., homozygous for guanine-adenine-adenine (GAA) repeat expansion inintron-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.\n- •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\n- Part 2 1. They have completed Part 1 RCT of the study and no discontinuation criteria (Section 8) have been met.\n- 2. Safety and tolerability data from Part 1 RCT are supportive of continuation in the judgement of the Investigator."}
Exclusion criteria
- {"criterion_text":"- Key Exclusion Criteria: •Glycosylated hemoglobin A1C (HbA1c) > 11% •B-type natriuretic peptide (BNP) > 200 pg/mL •Ejection fraction (EF) < 40% (based on ECHO performed at Screening visit) •Clinically significant abnormal laboratory test results •Clinically significant cardiac disease except mild to moderate cardiomyopathy"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Part 1 Primary Efficacy Endpoint •Change from baseline in USS (mFARS, Section E)","definition_or_measurement_approach":"Change from baseline in Upright Stability Score (USS) as part of mFARS (Section E); primary efficacy evaluated at Week 52 (Part 1 objective)."}
- {"endpoint_text":"- Secondary Efficacy Endpoints •Change from baseline in FA-HI •Change from baseline in mFARS •Change from baseline in PGI-S and CGI-S •Change from baseline in FA-ADL","definition_or_measurement_approach":"Change from baseline in listed scales/assessments (FA-HI, mFARS, PGI-S, CGI-S, FA-ADL) measured as change versus baseline (timepoints not further specified here)."}
- {"endpoint_text":"- Part 2 •Incidence of AEs and SAEs •Change from baseline in ECHO •Change from baseline in height, weight, and BMI •C-SSRS •Tanner scale","definition_or_measurement_approach":"Safety and tolerability measures including incidence of adverse events and serious adverse events; changes from baseline in ECHO, growth measures (height, weight, BMI); use of Columbia-Suicide Severity Rating Scale (C-SSRS) and Tanner staging as safety/endpoints."}
Secondary endpoints
- {"endpoint_text":"- Part 1 •Incidence of AEs and SAEs during the treatment period •Change from baseline in ECHO •Change from baseline in height, weight, and BMI •C-SSRS •Tanner scale •PK: Plasma concentrations of omaveloxolone by visit and timepoints","definition_or_measurement_approach":"Safety endpoints: incidence of AEs/SAEs during treatment; changes from baseline in ECHO and growth metrics; C-SSRS and Tanner scale assessments; PK measured as plasma concentrations of omaveloxolone by visit and timepoints."}
- {"endpoint_text":"- Part 2 Secondary Efficacy Endpoints: •Change from baseline in mFARS, including USS •Change from baseline in FA-ADL •Change from baseline in FA-HI •Change from baseline in PGI-S and CGI-S","definition_or_measurement_approach":"Efficacy endpoints in the open-label extension measured as change from baseline in mFARS (including USS), FA-ADL, FA-HI, PGI-S and CGI-S."}
Recruitment
- Digital Remote Recruitment
- Yes
- Planned Sample Size
- 199
- Recruitment Window Months
- 49
- Consent Approach
- Parental permission/consent is required for minors; age-appropriate assent forms are provided (multiple age bands: e.g., ages 2-5, 6-11, 12-14, 15-16, and other assent templates). Country- and language-specific informed consent and parental permission forms are available (examples in English, Spanish, French, German, Italian, Dutch, Danish). eConsent/remote consent platforms are used (documents reference IQVIA eConsent materials, 'IQVIA Complete Consent' screenshots for onsite and remote).
Methods
- Country-specific recruitment brochures and study planners (examples present for ES, AT, DK, IE, NL, DE, IT, FR) targeted to potential participants/patients and caregivers.
- Recruitment informed-consent procedure documents (K1) for multiple countries describing site-level recruitment arrangements.
- Email to potential participants (document: Email-to-potential-participant_DNK_Danish_Public) — Denmark.
- GP-letter (document: K2_296FA301_GP-letter_IE_English_Public) — Ireland.
- Patient advocacy / site patient advocacy contact lists (document: Site_Patient advocacy_Contact List for ICF_AT_Public).
- Thank-you cards, study-planner materials and other participant-facing printed materials (country-specific K2/K3 documents).
Geography
- Total Number Of Sites
- 14
- Total Number Of Participants
- 127
Spain
- Earliest CTIS Part Ii Submission Date
- 06-10-2025
- Latest Decision Or Authorization Date
- 29-10-2025
- Processing Time Days
- 23
- Number Of Sites
- 2
- Number Of Participants
- 16
Sites
- Site Name
- Hospital Sant Joan De Deu Barcelona
- Department Name
- Neurpediatric Service
- Principal Investigator Name
- Alejandra Darling
- Principal Investigator Email
- alejandra.darling@sjd.es
- Contact Person Name
- Alejandra Darling
- Contact Person Email
- alejandra.darling@sjd.es
- Site Name
- Hospital Universitario La Paz
- Department Name
- Neurpediatric Service
- Principal Investigator Name
- Maria del Mar Garcia Romero
- Principal Investigator Email
- yambee@hotmail.com
- Contact Person Name
- Maria del Mar Garcia Romero
- Contact Person Email
- yambee@hotmail.com
Austria
- Earliest CTIS Part Ii Submission Date
- 02-10-2025
- Latest Decision Or Authorization Date
- 03-11-2025
- Processing Time Days
- 32
- Number Of Sites
- 1
- Number Of Participants
- 8
Sites
- Site Name
- Medizinische Universitaet Innsbruck
- Department Name
- Department of Neurology Frauen-Kopf-Klinik
- Principal Investigator Name
- Sylvia Boesch
- Principal Investigator Email
- sylvia.boesch@i-med.ac.at
- Contact Person Name
- Sylvia Boesch
- Contact Person Email
- sylvia.boesch@i-med.ac.at
Denmark
- Earliest CTIS Part Ii Submission Date
- 17-10-2025
- Latest Decision Or Authorization Date
- 28-10-2025
- Processing Time Days
- 11
- Number Of Sites
- 1
- Number Of Participants
- 14
Sites
- Site Name
- Rigshospitalet
- Department Name
- Department of Pediatric Neurology, Juliane Marie Centret
- Principal Investigator Name
- Peter Born
- Principal Investigator Email
- alfred.peter.born@regionh.dk
- Contact Person Name
- Peter Born
- Contact Person Email
- alfred.peter.born@regionh.dk
Ireland
- Earliest CTIS Part Ii Submission Date
- 14-10-2025
- Latest Decision Or Authorization Date
- 31-10-2025
- Processing Time Days
- 17
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Children's Health Ireland
- Department Name
- Neurology
- Principal Investigator Name
- Declan O’Rourke
- Principal Investigator Email
- declan.orourke@cuh.ie
- Contact Person Name
- Declan O’Rourke
- Contact Person Email
- declan.orourke@cuh.ie
Netherlands
- Earliest CTIS Part Ii Submission Date
- 03-10-2025
- Latest Decision Or Authorization Date
- 06-11-2025
- Processing Time Days
- 34
- Number Of Sites
- 1
- Number Of Participants
- 5
Sites
- Site Name
- Radboud universitair medisch centrum Stichting
- Department Name
- Department of Pediatrics
- Principal Investigator Name
- Nienke Van Os
- Principal Investigator Email
- nienke.vanos@radboudumc.nl
- Contact Person Name
- Nienke Van Os
- Contact Person Email
- nienke.vanos@radboudumc.nl
Germany
- Earliest CTIS Part Ii Submission Date
- 15-10-2025
- Latest Decision Or Authorization Date
- 29-10-2025
- Processing Time Days
- 14
- Number Of Sites
- 3
- Number Of Participants
- 23
Sites
- Site Name
- University Medical Center Hamburg-Eppendorf
- Department Name
- Klinik und Poliklinik für Kinder- und Jugendmedizin
- Principal Investigator Name
- Deike Weiss
- Principal Investigator Email
- d.weiss@uke.de
- Contact Person Name
- Deike Weiss
- Contact Person Email
- d.weiss@uke.de
- Site Name
- Universitaetsklinikum Aachen AöR
- Department Name
- Klinik für Neurologie Sektion Translationale Neurodegeneration
- Principal Investigator Name
- Kathrin Reetz
- Principal Investigator Email
- kreetz@ukaachen.de
- Contact Person Name
- Kathrin Reetz
- Contact Person Email
- kreetz@ukaachen.de
- Site Name
- Justus-Liebig-Universitaet Giessen
- Department Name
- Zentrum für Kinderheilkunde und Jugendmedizin Abteilung für Pädiatrische Neurologie
- Principal Investigator Name
- Andreas Hahn
- Principal Investigator Email
- Andreas.Hahn@paediat.med.uni-giessen.de
- Contact Person Name
- Andreas Hahn
- Contact Person Email
- Andreas.Hahn@paediat.med.uni-giessen.de
Italy
- Earliest CTIS Part Ii Submission Date
- 03-10-2025
- Latest Decision Or Authorization Date
- 30-10-2025
- Processing Time Days
- 27
- Number Of Sites
- 3
- Number Of Participants
- 26
Sites
- Site Name
- IRCCS Foundation Istituto Neurologico Carlo Besta
- Department Name
- Dipartimento Neuroscienze Pediatriche SC Neuropsichiatria Infantile – Disordini del Movimento (NPI)
- Principal Investigator Name
- Isabella Moroni
- Principal Investigator Email
- isabella.moroni@istituto-besta.it
- Contact Person Name
- Isabella Moroni
- Contact Person Email
- isabella.moroni@istituto-besta.it
- Site Name
- Ospedale Pediatrico Bambino Gesu
- Department Name
- Neurorehabilitation Unit
- Principal Investigator Name
- Gessica Vasco
- Principal Investigator Email
- gessica.vasco@opbg.net
- Contact Person Name
- Gessica Vasco
- Contact Person Email
- gessica.vasco@opbg.net
- Site Name
- Associazione La Nostra Famiglia
- Department Name
- UOC Riabilitazione Neuromotoria
- Principal Investigator Name
- Gabriella Paparella
- Principal Investigator Email
- gabriella.paparella@lanostrafamiglia.it
- Contact Person Name
- Gabriella Paparella
- Contact Person Email
- gabriella.paparella@lanostrafamiglia.it
France
- Earliest CTIS Part Ii Submission Date
- 17-10-2025
- Latest Decision Or Authorization Date
- 03-11-2025
- Processing Time Days
- 17
- Number Of Sites
- 2
- Number Of Participants
- 30
Sites
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Service de Neuropédiatrie
- Principal Investigator Name
- Florence RENALDO
- Principal Investigator Email
- florence.renaldo@aphp.fr
- Contact Person Name
- Florence RENALDO
- Contact Person Email
- florence.renaldo@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Montpellier
- Department Name
- Service de Neurologie Pédiatrique
- Principal Investigator Name
- Agathe ROUBERTIE
- Principal Investigator Email
- a-roubertie@chu-montpellier.fr
- Contact Person Name
- Agathe ROUBERTIE
- Contact Person Email
- a-roubertie@chu-montpellier.fr
Sponsor
Primary sponsor
- Full Name
- Biogen Idec Research Limited
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United Kingdom
Contract research organisations
- Name
- IQVIA Limited
- Responsibilities
- eConsent, site payments and Drug Dev.
- Name
- PPD Global Central Labs
- Responsibilities
- Central Labs
- Name
- PPD Development LP
- Responsibilities
- Patient Cards, Patient services
- Name
- Suvoda LLC
- Name
- Medidata Solutions Inc.
Third parties
- {"country":"United States","full_name":"Small Planet","duties_or_roles":"Investigator Meeting Planner","organisation_type":"Health care"}
- {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"eConsent, site payments and Drug Dev.","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Praxis Communications LLC","duties_or_roles":"Development of Patient Recruitment Materials","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"Central Labs","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Q2 Solutions LLC","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Patient Cards, Patient services","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Cyberchrome Inc.","duties_or_roles":"Patient Retention- Printing and Shipping of Patient Recruitment Materials","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Ireland","full_name":"Accellacare Limited","duties_or_roles":"Home Health","organisation_type":"Pharmaceutical company"}
- {"country":"Ireland","full_name":"Teckro Limited","duties_or_roles":"Site Technical Portal - Site Communication and Training","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"eCOA/ePRO","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient Travel","organisation_type":"Non-Pharmaceutical company"}
Investigational products
- Investigational Product Name
- BIIB141
- Active Substance
- Omaveloxolone
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- ORAL
- Orphan Designation
- Yes
- Frequency
- QD
- Investigational Product Name
- Hard capsules (Placebo)
- Modality
- Other
- Frequency
- QD
Related trials
Other published trials that may interest you.