Friedreich Ataxia
Conditions
Brief summary
The primary objective of the study is to assess the pharmacokinetics (PK) and safety of vatiquinone administered in participants with Friedreich ataxia (FA) younger than 7 years.
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
No minimum to 6 Years
Healthy volunteers
No
Inclusion criteria
* Friedreich ataxia diagnosis (homozygous for GAA repeat expansion in intron-1 of FXN gene), confirmed by clinical genetic testing. * Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30 days before the Baseline Visit and for the duration of the study; any possible discontinuation of anticoagulants should be monitored and indicated by a specialist (for example, cardiologist, neurologist, or hematologist), and discontinuation will be noted by the prescribing physician. * Must be able to abstain from strong cytochrome P450 (CYP) 3A4 inducers/inhibitors (for example, ketoconazole, rifampin, St. John's wort, grapefruit juice, or any grapefruit product) for at least 30 days prior to enrollment and for the duration of the study. * Must be able to take vatiquinone oral solution with food.
Exclusion criteria
* Previous treatment with vatiquinone. * Allergy to vatiquinone or sesame oil. * Ejection fraction \<50%. * Participation in any other interventional clinical trial or receipt of any study drug in any other clinical trial within 60 days prior to the Baseline Visit. Participants may be rescreened after the exclusionary period of 60 days has passed. * Concomitant use of interventional CoQ10, vitamin E, or any approved or non-approved medication for FA within 30 days prior to the Baseline Visit. These prohibited medications can be discontinued at the Screening Visit. Note: Other inclusion and
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Plasma Concentration of Vatiquinone | Pre-morning dose (0 hour) at Week 4; 1 to 3 hours and 3 to 6 hours post-morning dose at Weeks 4, 12, and 24 |
| Area Under the Curve (AUC) of Vatiquinone | Pre-morning dose (0 hour) at Week 4; 1 to 3 hours and 3 to 6 hours post-morning dose at Weeks 4, 12, and 24 |
| Number of Participants With Adverse Events | Baseline up to Week 76 |
Countries
United States
Outcome results
None listed