Phase 2
N=10
(+) Epicatechin to Treat Friedreich's Ataxia
Friedreich's Ataxia
Bottom Line
View on ClinicalTrials.gov: NCT02660112 ↗Enrolled (actual)
10
Serious AEs
0.0%
Results posted
Dec 2019
Primary outcome: Primary: Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score — 55.0 score on a scale — p=0.336
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- (+)-Epicatechin (Drug)
- Age
- Pediatric, Adult · 10+ yrs
- Sex
- All
- Sponsor
- Ralitza Gavrilova
- Primary completion
- Dec 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score |
55.0 | 0.336 |
| PRIMARY Change in Ventricular Hypertrophy as Shown on Cardiac MRI |
55.9 | — |
Summary
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.
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT02660112). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.