Clinical Research Alert: Phase 3 Study of Vatiquinone in Individuals with Friedreich’s Ataxia

Researchers at PTC Therapeutics are conducting the phase 3 Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich’s Ataxia (PROVE-FA) study to learn more about Friedreich’s ataxia (FA) and to evaluate vatiquinone, an investigational therapy being studied for the treatment of FA.

About the PROVE-FA study

The PROVE-FA study is evaluating the efficacy and safety of vatiquinone in individuals 7-21 years old with FA.

In FA, reduced levels of the frataxin protein disrupt the energy-producing structures in cells (mitochondria), leading to iron buildup, oxidative stress and cellular damage, particularly in the nervous system and heart. Vatiquinone is a small molecule therapy designed to protect against oxidative stress and the cell and tissue damage that cause the symptoms of FA. Previous studies have shown that vatiquinone treatment slows disease progression in FA patients.

The PROVE-FA study is open-label, meaning that all participants will receive the investigational therapy. Vatiquinone will be administered orally (by mouth). Participation will last approximately 24 months and require six in-clinic visits, including the screening and baseline visits.

The effects of vatiquinone will be evaluated using a number of assessments including but not limited to: modified Friedreich’s Ataxia Rating Scale (mFARS), FA-activity of daily living, and timed 25-foot walk.

Study criteria

Individuals may be eligible for the PROVE-FA study if they meet the following key criteria:

• Confirmed diagnosis of GAA repeat expansion FA
• Ambulatory
• Age 7-21

Individuals may not be eligible for the PROVE-FA study if they have the following excluding factors:

• Clinical diagnosis of FA due to point mutations, deletions, or other non-GAA expansion mutations
• Participation in an interventional clinical study or treatment with an investigational drug
• Current use of omaveloxolone

Additional eligibility criteria may also apply. Please visit this link for the full list of inclusion and exclusion criteria.

Interested in participating?

Travel support will be available for eligible participants and their families.

Individuals interested in learning more about the PROVE-FA study should visit the sponsor’s trial website or contact the study coordinator by email: medinfo@ptcbio.com or phone: (+1) 866-562-4620.

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