A Study of the Safety and Effectiveness of (+) Epicatechin for Treating Patients with Friedreich's Ataxia

Overview

About this study

The purpose of this study is to test the safety and effectiveness of synthetically produced (+) Epicatechin for treating patients who have Friedreich's Ataxia, a neurological disorder.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Please contact the study team to discuss whether or not you are eligible to participate in a study.

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
  • Contraindications to regadenoson, i. e. second- or third-degree atrioventricular (AV) block or sinus node dysfunction
  • Has received an investigational drug within 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

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Ralitza Gavrilova, M.D.

Closed for enrollment

Contact information:

Lindsay Mulvihill CCRP

(507)538-5353

Mulvihill.Lindsay@mayo.edu

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available