(+) Epicatechin to Treat Friedreich's Ataxia
Status: | Completed |
---|---|
Conditions: | Neurology |
Therapuetic Areas: | Neurology |
Healthy: | No |
Age Range: | 10 - 50 |
Updated: | 2/8/2019 |
Start Date: | September 2016 |
End Date: | December 30, 2018 |
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.
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.
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