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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06953583




Registration number
NCT06953583
Ethics application status
Date submitted
11/04/2025
Date registered
1/05/2025
Date last updated
1/05/2025

Titles & IDs
Public title
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
Scientific title
A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled Study (Part 1) and Open-Label Extension (Part 2) to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 Years
Secondary ID [1] 0 0
2025-520896-13
Secondary ID [2] 0 0
296FA301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Omaveloxolone
Treatment: Drugs - Placebo

Experimental: Part 1 RCT: Omaveloxolone - Participants will receive a single oral dose of omaveloxolone once a day (QD) for up to 52 weeks in Part 1 of the study.

Placebo comparator: Part 1 RCT: Placebo - Participants will receive placebo, orally, QD for up to 52 weeks in Part 1 of the study.

Experimental: Part 2 OLE: Omaveloxolone - Participants who complete Part 1 of the study and are eligible will receive a single oral dose of omaveloxolone, QD for up to 104 weeks in the Part 2 OLE study.


Treatment: Drugs: Omaveloxolone
Administered as specified in the treatment arm.

Treatment: Drugs: Placebo
Administered as specified in the treatment arm.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part 1 RCT: Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreich's Ataxia Rating Scale (mFARS)
Timepoint [1] 0 0
Baseline, Week 52
Primary outcome [2] 0 0
Part 2 OLE: Number of Participants With Treatment-Emergent Adverse Event (TEAE) and Treatment-Emergent Serious Adverse Event (TESAE)
Timepoint [2] 0 0
From the first dose of the study drug in Part 2 up to the end of follow-up period in Part 2 (up to Week 105)
Primary outcome [3] 0 0
Part 2 OLE: Number of Participants With Change From Baseline in Cardiac Function Assessed by Echocardiogram (ECHO)
Timepoint [3] 0 0
Baseline (Week 52) up to Week 104
Primary outcome [4] 0 0
Part 2 OLE: Change From Baseline in Height
Timepoint [4] 0 0
Baseline (Week 52) up to Week 104
Primary outcome [5] 0 0
Part 2 OLE: Change From Baseline in Weight
Timepoint [5] 0 0
Baseline (Week 52) up to Week 104
Primary outcome [6] 0 0
Part 2 OLE: Change From Baseline in Body Mass Index (BMI)
Timepoint [6] 0 0
Baseline (Week 52) up to Week 104
Primary outcome [7] 0 0
Part 2 OLE: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS)
Timepoint [7] 0 0
Baseline (Week 52) up to Week 104
Primary outcome [8] 0 0
Part 2 OLE: Percentage of Participants at Each Tanner Stage
Timepoint [8] 0 0
Baseline (Week 52) up to Week 104
Primary outcome [9] 0 0
Part 2 OLE: Number of Participants at Each Tanner Stage
Timepoint [9] 0 0
Baseline (Week 52) up to Week 104
Secondary outcome [1] 0 0
Part 1 RCT: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI)
Timepoint [1] 0 0
Baseline, Week 52
Secondary outcome [2] 0 0
Part 1 RCT: Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS)
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [3] 0 0
Part 1 RCT: Change From Baseline in Patient Global Impressions-Severity (PGI-S)
Timepoint [3] 0 0
Baseline, Week 52
Secondary outcome [4] 0 0
Part 1 RCT: Change From Baseline in Clinical Global Impressions-Severity (CGI-S)
Timepoint [4] 0 0
Baseline, Week 52
Secondary outcome [5] 0 0
Part 1 RCT: Change From Baseline in Friedreich's Ataxia-Activities of Daily Living (FA-ADL)
Timepoint [5] 0 0
Baseline, Week 52
Secondary outcome [6] 0 0
Part 1 RCT: Number of Participants With Treatment-Emergent Adverse Event (TEAE) and Treatment-Emergent Serious Adverse Event (TESAE)
Timepoint [6] 0 0
From first dose of study drug up to end of follow up period in Part 1 RCT (up to Week 54)
Secondary outcome [7] 0 0
Part 1 RCT: Number of Participants With Change From Baseline in Cardiac Function Assessed by Echocardiogram (ECHO)
Timepoint [7] 0 0
Baseline, Weeks 26 and 52
Secondary outcome [8] 0 0
Part 1 RCT: Change From Baseline in Height
Timepoint [8] 0 0
Baseline up to Week 52
Secondary outcome [9] 0 0
Part 1 RCT: Change From Baseline in Weight
Timepoint [9] 0 0
Baseline up to Week 52
Secondary outcome [10] 0 0
Part 1 RCT: Change From Baseline in Body Mass Index (BMI)
Timepoint [10] 0 0
Baseline up to Week 52
Secondary outcome [11] 0 0
Part 1 RCT: Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS)
Timepoint [11] 0 0
Baseline up to week 52
Secondary outcome [12] 0 0
Part 1 RCT: Percentage of Participants at Each Tanner Stage
Timepoint [12] 0 0
Screening and Week 52
Secondary outcome [13] 0 0
Part 1 RCT: Number of Participants at Each Tanner Stage
Timepoint [13] 0 0
Screening and Week 52
Secondary outcome [14] 0 0
Part 1 RCT: Plasma Concentrations of Omaveloxolone
Timepoint [14] 0 0
Pre-dose and post-dose on Day 1, Weeks 4, 12 and 26
Secondary outcome [15] 0 0
Part 2 OLE: Change From Baseline in Modified Friedreich's Ataxia Rating Scale (mFARS), Including Upright Stability Score (USS)
Timepoint [15] 0 0
Baseline (Week 52) up to Week 104
Secondary outcome [16] 0 0
Part 2 OLE: Change From Baseline in Friedreich's Ataxia-Activities of Daily Living (FA-ADL)
Timepoint [16] 0 0
Baseline (Week 52) up to Week 104
Secondary outcome [17] 0 0
Part 2 OLE: Change From Baseline in Friedreich's Ataxia-Health Index (FA-HI)
Timepoint [17] 0 0
Baseline (Week 52) up to Week 104
Secondary outcome [18] 0 0
Part 2 OLE: Change From Baseline in Patient Global Impressions-Severity (PGI-S)
Timepoint [18] 0 0
Baseline (Week 52) up to Week 104
Secondary outcome [19] 0 0
Part 2 OLE: Change From Baseline in Clinical Global Impressions-Severity (CGI-S)
Timepoint [19] 0 0
Baseline (Week 52) up to Week 104

Eligibility
Key inclusion criteria
Part 1 RCT: Key inclusion criteria:

* 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 reported by the participant and/or the parent/caregiver a. Children 7 to < 16 years must also have an upright stability score (USS) score of 10 to = 34 at baseline

Part 1 RCT: Key exclusion criteria:

* 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 2 OLE: Eligibility criteria:

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

1. If alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or total bilirubin (TBL) are > 2× upper limit of normal (ULN) at the previous visit assessment, Part 2 Day 1 should be delayed until ALT and AST are < 1.5× ULN and TBL is < 2× ULN
2. If BNP is > 200 pg/mL at the previous visit assessment, Part 2 Day 1 should be delayed until BNP is < 200 pg/mL
3. If any other clinically significant laboratory abnormalities are present based on the previous visit assessments, Part 2 Day 1 should be delayed until the abnormalities are resolved
4. In the event of intercurrent illness or other change in health status of the participant, additional Part 1 screening assessments may be repeated prior to initiation of Part 2, based on the judgement of the investigator in consultation with the medical monitor

Note: Other protocol-defined Inclusion/
Minimum age
2 Years
Maximum age
15 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria may apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
3/07/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
22/11/2029
Actual
Sample size
Target
255
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Childrens Research Institute (MCRI) - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Pennsylvania
Country [4] 0 0
United States of America
State/province [4] 0 0
Tennessee
Country [5] 0 0
United States of America
State/province [5] 0 0
Virginia
Country [6] 0 0
Austria
State/province [6] 0 0
Innsbruck
Country [7] 0 0
Brazil
State/province [7] 0 0
Distrito Federal
Country [8] 0 0
Brazil
State/province [8] 0 0
Sao Paulo
Country [9] 0 0
Canada
State/province [9] 0 0
Quebec
Country [10] 0 0
Denmark
State/province [10] 0 0
Copenhagen
Country [11] 0 0
France
State/province [11] 0 0
Hérault
Country [12] 0 0
France
State/province [12] 0 0
Paris
Country [13] 0 0
Germany
State/province [13] 0 0
Aachen
Country [14] 0 0
Germany
State/province [14] 0 0
Giessen
Country [15] 0 0
Germany
State/province [15] 0 0
Hamburg
Country [16] 0 0
Ireland
State/province [16] 0 0
Dublin
Country [17] 0 0
Italy
State/province [17] 0 0
Lazio
Country [18] 0 0
Italy
State/province [18] 0 0
Veneto
Country [19] 0 0
Italy
State/province [19] 0 0
Milano
Country [20] 0 0
Netherlands
State/province [20] 0 0
Nijmegen
Country [21] 0 0
Spain
State/province [21] 0 0
Espluges De Llobregat
Country [22] 0 0
Spain
State/province [22] 0 0
Madrid
Country [23] 0 0
United Kingdom
State/province [23] 0 0
South Yorkshire
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Biogen
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Biogen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
US Biogen Clinical Trial Center
Address 0 0
Country 0 0
Phone 0 0
866-633-4636
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT06953583