(+) Epicatechin to Treat Friedreich's Ataxia

A Phase II, Open Label Prospective Single Center Drug Study Evaluating the Safety and Efficacy of (+)-Epicatechin in Subjects With Friedreich's Ataxia

This 24-week study will test the safety and effectiveness of synthetically produced (+) Epicatechin in treating patients who have Friedreich's Ataxia, a neurological disorder.

Inclusion Criteria:

• Confirmed diagnosis of Friedreich's Ataxia (FA) by Frataxin genetic testing and/or Frataxin enzyme analysis

• Between age 10 and 50 years of age, inclusive

• Body weight of 25 kilograms or higher

• Minimum of one affected organ (cardiac or neurological) system, as evidenced by clinical signs/symptoms

• Disease duration ≤7 years, based on onset date of FA symptoms

• Has no known contraindication to gadolinium contrast such as severe allergy or Glomerular Filtration Rate <30 ml/min/m^2.

• Has no known contraindication to non-contrast Magnetic Resonance Imaging (MRI) evaluation such as pacemaker or magnetically active metal fragments.

• Women of childbearing age must:

  • Have a negative pregnancy human chorionic gonadotropin test prior to receiving study drug.
  • Agree to use contraception for the duration of the study drug dosing, plus 1 month after completion of the study.

Exclusion Criteria:

• Advanced cardiac failure, New York Heart Association (NYHA) Classification Scale-Class IV (advanced stage heart failure)
• Clinically significant comorbidities that may also lead to cardiomyopathy, for example long standing hypertension, familial cardiomyopathy.
• Clinically significant comorbidities that would, in the opinion of the investigators, compromise the interpretation of test results.
• Pregnant, breast-feeding or planning to become pregnant during study timeframe.
• Patients with contraindications to regadenoson, i. e. second- or third-degree atrioventricular (AV) block or sinus node dysfunction. Has received an investigational drug within thirty (30) days of baseline visit.
• Thrombocytopenia (<125 x 10^9/Liter) or prolonged Prothrombin Time/Partial Thromboplastin Time (PT/PTT) at baseline.
• Clinically significant hypotension (systolic blood pressure <90) due to heart failure or other conditions.