Status:
RECRUITING
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)
Lead Sponsor:
Biogen
Conditions:
Friedreich Ataxia
Eligibility:
All Genders
2-15 years
Phase:
PHASE3
Brief Summary
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, ...
Detailed Description
The primary objective of Part 1 randomized controlled trial (RCT) is to evaluate the efficacy of omaveloxolone at Week 52 and the secondary objectives are to evaluate safety of omaveloxolone through W...
Eligibility Criteria
Inclusion
- 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 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
- 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
- If BNP is \> 200 pg/mL at the previous visit assessment, Part 2 Day 1 should be delayed until BNP is \< 200 pg/mL.
- 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.
- 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.
- If dosing has been interrupted at the end of Part 1, Part 2 Day 1 should be delayed until resumption of study drug treatment is appropriate per Section 8.2.
- Note: Other protocol-defined Inclusion/Exclusion criteria may apply.
Exclusion
Key Trial Info
Start Date :
June 9 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 22 2029
Estimated Enrollment :
255 Patients enrolled
Trial Details
Trial ID
NCT06953583
Start Date
June 9 2025
End Date
November 22 2029
Last Update
December 18 2025
Active Locations (34)
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1
UCLA Neurology Outpatient Clinic at Westwood
Los Angeles, California, United States, 90095
2
Norman Fixel Institute for Neurological Diseases UF Health
Gainesville, Florida, United States, 32608-3928
3
USF Health Morsani College of Medicine Department of Neurology
Tampa, Florida, United States, 33612
4
Children's Hospital of Philadelphia - Buerger Center for Advanced Pediatric Care - PIN
Philadelphia, Pennsylvania, United States, 19104