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Trial record 41 of 184 for:    "autosomal recessive cerebellar ataxia type 1" OR "Cerebellar Ataxia"

(+) Epicatechin to Treat Friedreich's Ataxia

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ClinicalTrials.gov Identifier: NCT02660112
Recruitment Status : Active, not recruiting
First Posted : January 21, 2016
Last Update Posted : November 1, 2017
Sponsor:
Collaborator:
Cardero Therapeutics, Inc.
Information provided by (Responsible Party):
Ralitza Gavrilova, Mayo Clinic

January 18, 2016
January 21, 2016
November 1, 2017
September 2016
December 2018   (Final data collection date for primary outcome measure)
  • 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 ]
  • 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) 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 ]
Complete list of historical versions of study NCT02660112 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
(+) Epicatechin to Treat Friedreich's Ataxia
A Phase II, Open Label Prospective Single Center Drug Study Evaluating the Safety and Efficacy of (+)-Epicatechin in Subjects With Friedreich's Ataxia
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.
Not Provided
Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Friedreich's Ataxia
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)
Experimental: (+)-Epicatechin
Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks
Intervention: Drug: (+)-Epicatechin
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
10
Same as current
December 2018
December 2018   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
10 Years to 50 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT02660112
15-006845
No
Not Provided
Plan to Share IPD: No
Ralitza Gavrilova, Mayo Clinic
Ralitza Gavrilova
Cardero Therapeutics, Inc.
Principal Investigator: Ralitza H Gavrilova, MD Mayo Clinic
Mayo Clinic
September 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP