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

Launched by BIOGEN · Apr 29, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a medication called BIIB141, also known as omaveloxolone or SKYCLARYS®, to see how it affects children and teens aged 2 to 15 years with Friedreich's Ataxia (FA). While this drug is already available for adults with FA, researchers want to learn more about its safety and effectiveness for younger patients. The study aims to understand how the medication impacts symptoms such as balance and stability, any health problems that may arise during the trial, and how it affects overall health and heart health as participants go through puberty.

To participate, children must have a confirmed diagnosis of Friedreich's Ataxia and show signs of symptoms, such as difficulty with balance. The study includes two parts: the first part lasts up to 52 weeks where participants will take either the medication or a placebo (a non-active pill) daily, with several visits to a research center for monitoring. The second part lasts up to 104 weeks, where participants can continue taking the medication based on their experience in the first part. Throughout the study, regular check-ins will help researchers monitor participants' health and any side effects. This trial is an important step in finding effective treatments for younger patients with Friedreich's Ataxia.

Gender

ALL

Eligibility 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/Exclusion criteria may apply.