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Phase 2 N=10 Treatment

(+) Epicatechin to Treat Friedreich's Ataxia

Friedreich's Ataxia

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

OutcomeResultp-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.
View full record on ClinicalTrials.gov →

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.

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