(+) Epicatechin to Treat Friedreich's Ataxia

Ralitza Gavrilova

Status and phase

Completed

Phase 2

Conditions

Friedreich's Ataxia

Treatments

Drug: (+)-Epicatechin

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT02660112

15-006845

Details and patient eligibility

About

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.

Enrollment

10 patients

Sex

All

Ages

10 to 50 years old

Volunteers

No Healthy Volunteers

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.