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(+) Epicatechin to Treat Friedreich's Ataxia

This study is currently recruiting participants.
See Contacts and Locations
Verified September 2016 by Ralitza Gavrilova, Mayo Clinic
Sponsor:
Collaborator:
Cardero Therapeutics, Inc.
Information provided by (Responsible Party):
Ralitza Gavrilova, Mayo Clinic
ClinicalTrials.gov Identifier:
NCT02660112
First received: January 18, 2016
Last updated: September 26, 2016
Last verified: September 2016
  Purpose
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 Intervention Phase
Friedreich's Ataxia Drug: (+)-Epicatechin Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Ralitza Gavrilova, Mayo Clinic:

Primary Outcome Measures:
  • 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 from baseline in ventricular hypertrophy as shown on cardiac MRI [ Time Frame: Baseline, 24 weeks ]

Estimated Enrollment: 10
Study Start Date: September 2016
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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)

  Eligibility

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02660112

Contacts
Contact: Gillian A Currie, BSc 507-293-0792 Currie.Gillian@mayo.edu
Contact: Kayla M Ehlenfeldt 507-266-2967 Ehlenfeldt.Kayla@mayo.edu

Locations
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Gillian A Currie, BSc    507-293-0792    Currie.Gillian@mayo.edu   
Contact: Jennifer Kemppainen, MS, CGC    507-266-2967    Kemppainen.Jennifer@mayo.edu   
Principal Investigator: Ralitza H. Gavrilova, MD         
Sponsors and Collaborators
Ralitza Gavrilova
Cardero Therapeutics, Inc.
Investigators
Principal Investigator: Ralitza H Gavrilova, MD Mayo Clinic
  More Information

Responsible Party: Ralitza Gavrilova, MD, Sponsor-Investigator, Mayo Clinic
ClinicalTrials.gov Identifier: NCT02660112     History of Changes
Other Study ID Numbers: 15-006845
Study First Received: January 18, 2016
Last Updated: September 26, 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Ataxia
Cerebellar Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
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

ClinicalTrials.gov processed this record on August 18, 2017