Friedreich's Ataxia
Conditions
Brief summary
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
Interventions
DRUGVatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm.
Sponsors
PTC Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
7 Years to 21 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * mFARS ≥20 to ≤70 at Screening (4 weeks prior to Day 1) and Baseline (Day 1). * Must be ambulatory as defined by a E7 score of 4 or less on the USS at Screening and Baseline. * Documentation that participants reached maximum score on items E4, E5, and E3b on the USS of mFARS at Screening and Baseline. * FA diagnosis (homozygous for guanine-adenine-adenine \[GAA\] repeat expansion in intron-1 of the frataxin gene), confirmed and documented with GAA repeat length for both alleles by clinical genetic testing. * Difference in the mFARS score between Screening and Baseline of no more than 4 points. * Ability to abstain from strong cytochrome P450 (CYP) 3A4 inducers/inhibitors (for example, ketoconazole, rifampin, St. John's wort, grapefruit juice) for at least 4 weeks prior to Baseline and for the duration of the study. Key
Exclusion criteria
* Individuals with clinical diagnosis of FA who have point mutations, deletions, or other non-GAA expansion mutations. * Allergy to vatiquinone, sesame oil, gelatin (bovine and/or porcine), titanium dioxide, or red iron oxide. * Pregnant or lactating participants or those sexually active participants who are unwilling to comply with proper birth control methods; females of childbearing potential must have a negative pregnancy test at Screening and during the Baseline Visit. * Comorbidities that may confound study results (for example, fat malabsorption syndrome, other mitochondrial disorder) in the opinion of the investigator. * Participation in an interventional clinical study or received investigational drug (other than SKYCLARYS® \[omaveloxolone\]) within 60 days prior to Screening. Participants may be screened after the exclusionary period of 60 days has passed. * Current use of omaveloxolone. Previous use of omaveloxolone will be allowed if: 1. Use was less than 3 cumulative months and participants have been off treatment for \>30 days. 2. Use was less than 6 cumulative months and participants have been off treatment for \>90 days * Previous or concurrent use of other investigational treatment for FA. * Participation in any cell or gene therapy-based treatment for FA. * Participation in an ongoing study for vatiquinone or current or previous use of vatiquinone. * Illicit drug use 30 days prior to Screening and during the study. Note: Other protocol-defined inclusion and
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24 | Baseline, Month 24 |
Secondary
| Measure | Time frame |
|---|---|
| Change From Baseline in mFARS Subscale Scores (Upright Stability Subscale [USS], Upper Limb [UL], Lower Limb [LL], Bulbar [BUL]) at Month 24 | Baseline, Month 24 |
| Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Score at Month 24 | Baseline, Month 24 |
| Change From Baseline in 25-Foot Walk Test (T25FW) at Month 24 | Baseline, Month 24 |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Baseline up to Month 25 |
Countries
Belgium, Brazil, Canada, France, Spain, United States
Contacts
CONTACTPatient Advocacy
Outcome results
None listed