(+) Epicatechin to Treat Friedreich's Ataxia
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|ClinicalTrials.gov Identifier: NCT02660112|
Recruitment Status : Completed
First Posted : January 21, 2016
Results First Posted : December 5, 2019
Last Update Posted : December 5, 2019
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|Condition or disease||Intervention/treatment||Phase|
|Friedreich's Ataxia||Drug: (+)-Epicatechin||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II, Open Label Prospective Single Center Drug Study Evaluating the Safety and Efficacy of (+)-Epicatechin in Subjects With Friedreich's Ataxia|
|Study Start Date :||September 2016|
|Actual Primary Completion Date :||December 30, 2018|
|Actual Study Completion Date :||December 30, 2018|
Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks
25mg (+)-Epicatechin capsules. Starting dose 75mg total daily dose (one 25mg cap taken three times per day). Dose escalation at 12 weeks for non-responders to 150mg total daily dose (two 25mg caps taken three times per day)
- Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score [ Time Frame: Baseline, 24 weeks ]
The Friedreich Ataxia Rating Scale (FARS) is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate (assesses speech speed by repetitions of pronouncing "PaTa" ) and low-contrast letter acuity.
FARS is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate and low-contrast letter acuity. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability.
- Change in Ventricular Hypertrophy as Shown on Cardiac MRI [ Time Frame: Baseline, 24 weeks ]Left ventricular mass and left ventricular (LV) mass indexed to body surface area estimated by Left Ventricle (LV)cavity dimension and wall thickness at end-diastole. Normal values of LV mass indexed to body surface area are found to be 49-115 gL/m2 in men and 43-95 g/m2 in women.
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|Ages Eligible for Study:||10 Years to 50 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02660112
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|Principal Investigator:||Ralitza H Gavrilova, MD||Mayo Clinic|
Documents provided by Ralitza Gavrilova, Mayo Clinic:
|Responsible Party:||Ralitza Gavrilova, MD, Sponsor-Investigator, Mayo Clinic|
|Other Study ID Numbers:||
|First Posted:||January 21, 2016 Key Record Dates|
|Results First Posted:||December 5, 2019|
|Last Update Posted:||December 5, 2019|
|Last Verified:||February 2019|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
Nervous System Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn