Phase
Condition
Spinocerebellar Disorders
Dyskinesias
Friedreich's Ataxia
Treatment
Placebo
Omaveloxolone
Clinical Study ID
Ages 2-15 All Genders
Study Summary
Eligibility Criteria
Inclusion
Part 1 RCT: Key inclusion criteria:
Diagnosed with genetically confirmed Friedreich's Ataxia (FA), i.e., homozygous forguanine-adenine-adenine (GAA) repeat expansion in intron-1 of the frataxin gene, orGAA repeat expansion in 1 allele and with point mutations or deletions, or othernon-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 10to ≤ 34 at baseline
Exclusion
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 screeningvisit]
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 criteriahave been met
Safety and tolerability data from Part 1 RCT are supportive of continuation in thejudgement of the investigator
If alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/ortotal bilirubin (TBL) are > 2× upper limit of normal (ULN) at the previousvisit 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 bedelayed until BNP is < 200 pg/mL
If any other clinically significant laboratory abnormalities are present basedon the previous visit assessments, Part 2 Day 1 should be delayed until theabnormalities are resolved
In the event of intercurrent illness or other change in health status of theparticipant, additional Part 1 screening assessments may be repeated prior toinitiation of Part 2, based on the judgement of the investigator inconsultation with the medical monitor
Note: Other protocol-defined Inclusion/Exclusion criteria may apply.
Study Design
Study Description
Connect with a study center
Murdoch Childrens Research Institute (MCRI)
Parkville, Victoria 3052
AustraliaSite Not Available
Universitätsklinikum Innsbruck
Innsbruck, 6020
AustriaSite Not Available
L2 Ip - Instituto de Pesquisas Clinicas Ltda - ME
Brasília, Distrito Federal 70200-730
BrazilSite Not Available
University of Campinas (UNICAMP) School of Medical Sciences
Campinas, Sao Paulo 13083-970
BrazilSite Not Available
PSEG Centro de Pesquisa Clinica
Sao Paulo, 04038-002
BrazilSite Not Available
McGill University
Montreal, Quebec H3H 2R9
CanadaSite Not Available
Rigshospitalet - Juliane Marie Centret (JMC) Copenhagen
Copenhagen, 2100
DenmarkSite Not Available
CHU de Montpellier - Hôpital Arnaud de Villeneuve
Montpellier, Hérault 34090
FranceSite Not Available
AP-HP - Hôpital Armand Trousseau
Paris, 75012
FranceSite Not Available
Universitätsklinikum Aachen
Aachen, 52074
GermanySite Not Available
UKGM - Universitätsklinikum Giessen und Marburg GmbH - Standort Gießen
Giessen, 35392
GermanySite Not Available
UKE Hamburg
Hamburg, 20251
GermanySite Not Available
CHI at Temple Street
Dublin, D01 XD99
IrelandSite Not Available
Ospedale Pediatrico Bambino Gesù IRCCS
Roma, Lazio 165
ItalySite Not Available
IRCCS Eugenio Medea - Polo. Scientifico Veneto
Conegliano, Veneto 31015
ItalySite Not Available
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milano, 20133
ItalySite Not Available
Radboud Universitair Medisch Centrum
Nijmegen, 6525 GA
NetherlandsSite Not Available
Hospital Sant Joan de Deu
Espluges De Llobregat, 8950
SpainSite Not Available
Hospital Universitario La Paz - PPDS
Madrid, 28046
SpainSite Not Available
Sheffield Children's Hospital
Sheffield, South Yorkshire S10 2TH
United KingdomSite Not Available
University College London, Institue of Neurology
London, WC1N 3BG
United KingdomSite Not Available
UCLA Neurology Outpatient Clinic at Westwood
Los Angeles, California 90095
United StatesSite Not Available
Norman Fixel Institute for Neurological Diseases UF Health
Gainesville, Florida 32608-3928
United StatesSite Not Available
USF Health Morsani College of Medicine Department of Neurology
Tampa, Florida 33612
United StatesSite Not Available
Children's Hospital of Philadelphia Buerger Center
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
St Jude Children's Research Hospital
Memphis, Tennessee 38105-3678
United StatesSite Not Available
Children's Hospital of the King's Daughters
Norfolk, Virginia 23507-1910
United StatesActive - Recruiting
Not the study for you?
Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.