Last updated 2 months ago
A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
255 patients around the world
Available in Brazil, United States
The primary objective of Part 1 is to evaluate the efficacy of omaveloxolone as measured by upright stability score (USS) and the secondary objectives are to evaluate the efficacy of omaveloxolone as measured by additional secondary efficacy outcomes, safety of omaveloxolone and the plasma concentration of omaveloxolone after single and multiple dose administration.
The primary objective of Part 2A is to evaluate the efficacy of omaveloxolone and the secondary objectives are to characterize the efficacy of omaveloxolone as measured by additional secondary outcomes, evaluate the safety and tolerability of omaveloxolone and plasma concentration of omaveloxolone after single and multiple dose administration.
The primary objective for Part 2B is to evaluate the safety and tolerability of long-term omaveloxolone use and the secondary objective is to evaluate the efficacy of omaveloxolone following long-term use.
Biogen
2Research sites
255Patients around the world
This study is for people with
Ataxia
Friedreich's ataxia
Requirements for the patient
To 15 Years
All Gender
Medical requirements
- Part 1: RCTDiagnosed 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.Children 7 to < 16 years must also have an upright stability score (USS) score of 10 to ≤ 34 at baseline.Part 2: OLEParticipants 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 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.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.
- Part 1: RCTGlycosylated 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.
Sites
L2IP Instituto de Pesquisas Clínicas - Brasilia
Hospital de Clínicas - Universidade Estadual de Campinas - UNICAMP
StudyBRAVE
SponsorBiogen
Study typeInterventional
Conditions
Friedreich's ataxia
Requirements
To 15 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT06953583
