Health Condition 1: G111- Early-onset cerebellar ataxia
Conditions
Interventions
Intervention1: omaveloxolone: omaveloxolone dose of
150 mg QD total duration of 104 weeks (Part-1 52 weeks and Part-2 upto 104 weeks)
Control Intervention1: Placebo: Matching Placebo 150mg QD (upto 52
Sponsors
Biogen Idec Research Limited
PPD Pharmaceutical Development India Pvt Ltd
Eligibility
Inclusion criteria
Inclusion criteria: Part 1 RCT: Key inclusion criteria: Diagnosed with genetically confirmed Friedreichs 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 less than 16 years must also have an upright stability score (USS) score of 10 to less than equals to 34 at baseline. 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 oIf BNP is greater than 200 pg per mL at the previous visit assessment, Part 2 Day 1 should be delayed until BNP is less than 200 pg per mL. oIf 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. oIn 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. oIf 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. Note: Other protocol-defined Inclusion/Exclusion criteria may apply.
Exclusion criteria
Exclusion criteria: Part 1 RCT: Key exclusion criteria: Glycosylated hemoglobin A1C (HbA1c) greater than 11percentage B-type natriuretic peptide (BNP) greater than 200 picograms per milliliter (pg per mL) at screening Ejection fraction (EF) less than 40percentage [based on echocardiogram (ECHO) performed at screening visit Clinically significant cardiac disease except mild to moderate cardiomyopathy Note: Other protocol-defined Inclusion/Exclusion criteria may apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part 1 Primary Efficacy Endpoint Change From Baseline in Upright Stability Score (USS) Subscale E of Modified Friedreichs Ataxia Rating Scale (mFARS) Part-2 Number of Participants With Treatment-Emergent Adverse Event (TEAE) and Treatment-Emergent Serious Adverse Event (TESAE) Number of Participants With Change From Baseline in Cardiac Function Assessed by Echocardiogram (ECHO) Change From Baseline in Height Change From Baseline in Weight Change From Baseline in Body Mass Index (BMI) Change From Baseline in Columbia Suicide Severity Rating Scale (C-SSRS) Percentage of Participants at Each Tanner Stage Number of Participants at Each Tanner StageTimepoint: Part-1 Baseline to week 54 Part-2 Baseline (Week 52) up to Week 104 | — |
Secondary
| Measure | Time frame |
|---|---|
| Part1 Change From Baseline in Friedreichs Ataxia Health Index (FA-HI) Change From Baseline in Modified Friedreichs Ataxia Rating Scale (mFARS) Change From Baseline in Patient Global Impressions Severity (PGI-S) Change From Baseline in Clinical Global Impressions Severity (CGI-S) Change From Baseline in Friedreichs Ataxia Activities of Daily Living (FA-ADL) Part2 Change From Baseline in Modified Friedreichs Ataxia Rating Scale (mFARS) Including Upright Stability Score (USS) Change From Baseline in Friedreichs Ataxia Activities of Daily Living (FA-ADL) Change From Baseline in Friedreichs Ataxia Health Index (FA-HI) Change From Baseline in Patient Global Impressions Severity (PGI-S) Change From Baseline in Clinical Global Impressions Severity (CGI-s) Timepoint: Part-1 Baseline to week 54 Part-2 Baseline (Week 52) up to Week 104 | — |
Countries
Australia, Austria, Brazil, Canada, Denmark, France, Germany, India, Ireland, Italy, Netherlands, Saudi Arabia, Spain, Turkey, United Kingdom, United States of America
Contacts
Public ContactRashmi Chitgupi
PPD Pharmaceutical Development India Private Limited, part of Thermo Fisher Scientific
Outcome results
None listed