Study Overview:

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.

Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.

Study Details:

This clinical research study is being conducted at the Children’s Hospital of Philadelphia (CHOP). It will test the safety and effectiveness of a study drug called Vatiquinone in young children with FA. Vatiquinone has been tested previously in adults with FA and was found to be safe and well tolerated. Everyone who participates in this study will receive the study drug (no placebo). The study drug might help slow down the progression of FA symptoms and the information learned may also help others with FA in the future.

Participation in this study will involve approximately 9 in-person study visits at CHOP, as well as study phone calls in between visits. Participating children will need to take the study drug three times a day for 72 weeks. After those 72 weeks, there will be a follow-up with one final phone call approximately 30 days after the last dose.

The study drug and all study-related assessments will be provided at no cost. Travel expenses, along with study-related expenses (e.g., meals) for the child and a parent/ caregiver will be reimbursed.

Key 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 enrolment and for the duration of the study.
  • Must be able to take Vatiquinone oral solution with food.

Key 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.

Additional inclusion and exclusion criteria may apply and will be evaluated by a study doctor.

Length of Study Commitment:

  • 82 weeks

Participating Study Locations

Institution Name and LocationInvestigatorStudy Coordinator Contact Information

Children’s Hospital of Philadelphia USA

David Lynch, MD, PhD

Courtney Cheek Park

Explore the FA Drug Development Pipeline

FARA believes that there are many different approaches to treating Friedreich’s ataxia, and that it will require a cocktail approach of two or more treatments to slow, stop, reverse, and cure FA. Learn more about the approach behind this potential treatment and explore the other approaches that are in the FA Drug Development pipeline.