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(+) Epicatechin to Treat Friedreich's Ataxia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02660112
Recruitment Status : Completed
First Posted : January 21, 2016
Results First Posted : December 5, 2019
Last Update Posted : December 5, 2019
Sponsor:
Collaborator:
Cardero Therapeutics, Inc.
Information provided by (Responsible Party):
Ralitza Gavrilova, Mayo Clinic

Study Description
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Brief 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.

Condition or disease Intervention/treatment Phase
Friedreich's Ataxia Drug: (+)-Epicatechin Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

Drug Information available for: Cianidanol

Arms and Interventions
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Arm Intervention/treatment
Experimental: (+)-Epicatechin
Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks
 Drug: (+)-Epicatechin
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)


Outcome Measures
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Primary Outcome Measures :
  1. 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.


  2. 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.


Eligibility Criteria
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Ages Eligible for Study:   10 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Ralitza Gavrilova
Cardero Therapeutics, Inc.
Investigators
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Principal Investigator: Ralitza H Gavrilova, MD Mayo Clinic
  Study Documents (Full-Text)

Documents provided by Ralitza Gavrilova, Mayo Clinic:
Study Protocol and Statistical Analysis Plan  [PDF] January 27, 2017

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Ralitza Gavrilova, MD, Sponsor-Investigator, Mayo Clinic
ClinicalTrials.gov Identifier: NCT02660112    
Other Study ID Numbers: 15-006845
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
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
 Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases