Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

Официальное название

Evaluation of Long-Term Efficacy of Vatiquinone for the Treatment of Friedreich's Ataxia

Обзор

The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.

Вмешательства

• Препарат Vatiquinone
  Vatiquinone will be administered per dose and schedule specified in the arm.

Первичные конечные точки

• Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24 [Срок оценки: Baseline, Month 24]

Вторичные конечные точки (4)

• Change From Baseline in mFARS Subscale Scores (Upright Stability Subscale [USS], Upper Limb [UL], Lower Limb [LL], Bulbar [BUL]) at Month 24 [Срок оценки: Baseline, Month 24]
• Change From Baseline in Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Score at Month 24 [Срок оценки: Baseline, Month 24]
• Change From Baseline in 25-Foot Walk Test (T25FW) at Month 24 [Срок оценки: Baseline, Month 24]
• Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [Срок оценки: Baseline up to Month 25]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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:

• Use was less than 3 cumulative months and participants have been off treatment for >30 days.
• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07681713 · PTC743-FA-304 · 2026-526947-37-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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