Friedreich's Ataxia Program Clinical Studies

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RTA-408 in Friedreich Ataxia

The Children’s Hospital of Philadelphia is recruiting patients with Friedreich’s ataxia (FA) in the United States for a Phase 2 clinical trial studying the safety and effects of RTA 408 in FA. This drug has potential antioxidant properties and we are looking to see how it affects the symptoms of FA. In order to participate, you must have genetic confirmation of FA, maintain stable exercise and medications throughout the study, and discontinue all antioxidant supplements. This is a double-blind placebo-control trial, so half of all subjects will receive study drug and half will receive placebo. Each subject's study participation would last 12 months and include 10 study visits to CHOP. IRB 14-011262

Friedreich Ataxia Clinical Outcomes Measures

This is a multicenter natural history and clinical measure study. Our objectives are to advance clinical care, research and therapeutic approaches in FA through the development and validation of clinical outcome measures in FA, collection of quantitative serial clinical data on patients and expansion of our established research network. A secondary objective is to build a parallel DNA and RNA repository for use in large scale translational research including modifier gene studies. We wish to also enroll control subjects for a hearing test and sample collection to compare to data from FA patients.

IRB 01-002609