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(+) Epicatechin to Treat Friedreich's Ataxia

Primary Purpose

Friedreich's Ataxia

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
(+)-Epicatechin
Sponsored by
Ralitza Gavrilova
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Friedreich's Ataxia

Eligibility Criteria

10 Years - 50 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Mayo Clinic

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

(+)-Epicatechin

Arm Description

Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks

Outcomes

Primary Outcome Measures

Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score
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 in Ventricular Hypertrophy as Shown on Cardiac MRI
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.

Secondary Outcome Measures

Full Information

First Posted
January 18, 2016
Last Updated
November 18, 2019
Sponsor
Ralitza Gavrilova
Collaborators
Cardero Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT02660112
Brief Title
(+) Epicatechin to Treat Friedreich's Ataxia
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 Type
Interventional

2. Study Status

Record Verification Date
February 2019
Overall Recruitment Status
Completed
Study Start Date
September 2016 (undefined)
Primary Completion Date
December 30, 2018 (Actual)
Study Completion Date
December 30, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Ralitza Gavrilova
Collaborators
Cardero Therapeutics, Inc.

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Friedreich's Ataxia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
(+)-Epicatechin
Arm Type
Experimental
Arm Description
Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks
Intervention Type
Drug
Intervention Name(s)
(+)-Epicatechin
Intervention Description
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)
Primary Outcome Measure Information:
Title
Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score
Description
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.
Time Frame
Baseline, 24 weeks
Title
Change in Ventricular Hypertrophy as Shown on Cardiac MRI
Description
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.
Time Frame
Baseline, 24 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ralitza H Gavrilova, MD
Organizational Affiliation
Mayo Clinic
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
32677106
Citation
Qureshi MY, Patterson MC, Clark V, Johnson JN, Moutvic MA, Driscoll SW, Kemppainen JL, Huston J 3rd, Anderson JR, Badley AD, Tebben PJ, Wackel P, Oglesbee D, Glockner J, Schreiner G, Dugar S, Touchette JC, Gavrilova RH. Safety and efficacy of (+)-epicatechin in subjects with Friedreich's ataxia: A phase II, open-label, prospective study. J Inherit Metab Dis. 2021 Mar;44(2):502-514. doi: 10.1002/jimd.12285. Epub 2020 Aug 31.
Results Reference
derived

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(+) Epicatechin to Treat Friedreich's Ataxia

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