Clinical Trials in France / NCT07681713
Starting soon
Phase 3
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
NCT07681713 · tracked via the Priya Life Science France tracker
Condition(s) studied
Friedreich's Ataxia
Investigational drug(s) / intervention(s)
Vatiquinone: Vatiquinone will be administered per dose and schedule specified in the arm.
Study summary
The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.
Eligibility
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 exclusion criteria may apply.
Primary outcome measure(s)
- Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24 — Baseline, Month 24
Trial sites (9)
| Facility | City | Region | Status |
|---|---|---|---|
| University of California Los Angeles | Los Angeles | California | |
| University of South Florida | Tampa | Florida | |
| University of Iowa | Iowa City | Iowa | |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | |
| Hôpital Érasme (Hôpital Universitaire de Bruxelles) | Brussels | Belgium | |
| University of Campinas | Campinas | São Paulo | |
| Centre Hospitalier de l'Universite de Montreal CHUM | Montreal | Quebec | |
| Institut du Cerveau et de la Moelle Epinière | Paris | France | |
| Hospital Sant Joan de Deu | Barcelona | Spain |
More PTC Therapeutics trials in France
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07681713 on ClinicalTrials.gov ↗ ← All trials in France