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EPI-743 in FA Point Mutations

EPI-743 in Friedreich's Ataxia Point Mutations

Ages: 18-65

Location: University of South Florida

Details: The University of South Florida (USF) in Tampa, FL is looking for adults ages 18-65 to complete a trial for EPI-743 in patients with point mutations. This is a Phase 2A Clinical Trial for Edison Pharmaceutical's EPI-743, also known as Vincerinone™, which aims to improve mitochondrial function by reducing oxidative stress. All participants will receive 400mg of EPI-743 three times daily for 3 months. The sponsor of the trial is Edison Pharmaceuticals, Inc. and the study's lead investigator is Theresa Zesiewicz, MD.

To be eligible for participation, genetic confirmation of Friedreich’s Ataxia is required. Additional eligibility details are available at:

If you are interested in participating, please contact the study coordinator below:

Read more: EPI-743 in FA Point Mutations

Newcastle Imaging Study

Developing a new tool to measure how nerves are affected in FA

Ages: 10-50

Location: Newcastle University, UK

Details: The main aim of this research is to allow us to develop a more accurate method of monitoring the progression of your condition. This will then allow us to work out whether a particular treatment is slowing the progression of the disease in future clinical trials. This study involves a combination of clinical assessments and non-invasive neurophysiological tests, a blood test and a very small skin sample.

Please see the study flyer below and contact Dr. Vandy Dhawan at 0191 2086897 or with any questions.

Read more: Newcastle Imaging Study

Shire's SHP622 Phase 1 Study (formerly VP-20629)

Safety and Pharmacology Study of VP 20629 (now called SHP622) in Adults With FA

Ages: 18-45

Locations: Children's Hospital of Philadephia (CHOP), Emory, University of South Florida, University of Iowa, UCLA

Details: This is a Phase 1 study sponsored by Shire (formerly initiated by ViroPharma) designed to evaluate dosage and safety of VP20629, a naturally occurring small molecular weight drug compound that can prevent oxidative stress to improve mitochondrial function. Participation in this study requires both outpatient and inpatient visits. Subjects in the second arm need to stay in the hospital for 10 days. In the second arm, subjects will receive multiple doses of VP 20629 (300 mg, 600 mg, or 900 mg total daily dose) or placebo. VP20629 or placebo will be administered every 8 hours for 7 days with a single morning dose on Day 8.

Read more: Shire's SHP622 Phase 1 Study (formerly VP-20629)

Cardiovascular Effects of Acetyl-L-Carnitine

Description: An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia.
The purpose of this study is to learn how treatment with acetyl-L-carnitine (ALCAR) will affect the hearts of patients with Friedreich's Ataxia as well as how it may affect other symptoms of Friedreich's Ataxia such as difficulties with balance, walking, or upper arm function. There are 11 visits to the research clinic at the University of South Florida Morsani Center for Advanced Health Care. Study related procedures and medication will be provided by the sponsor.

Key Criteria: Ages 18-80 years

Location: University of South Florida -

Please review the study flyer below, and contact the Study Coordinator with any questions.

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