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An Open-Label Study to Evaluate Vatiquinone (PTC743) in Children With Friedreich Ataxia

About this research study:

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 last for about 82 weeks. It will involve approximately 9 in-person study visits at CHOP, as well as study phone calls in between visits. Your child will need to take the study drug three times a day for 72 weeks. After those 72 weeks, we will 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.

Who can participate:

Children under 7 years of age who:

  • Have been diagnosed with FA (confirmed by genetic testing)
  • Have not participated in any other interventional clinical trial in the past 2 months

How to Participate:

If you are interested in participating in this study and would like more information, please contact:

Medina Keita
(267) 426-7584

Courtney Cheek Park
(267) 426-9567

McKenzie Wells
(267) 426-9608

Kellie McIntyre
(215) 590-2975

Victoria Profeta
(215) 590-3129

Additional information can be found:

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

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