NCT02660112

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Sep 2016

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 18, 2016

Completed
3 days until next milestone

First Posted

Study publicly available on registry

January 21, 2016

Completed
7 months until next milestone

Study Start

First participant enrolled

September 1, 2016

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 30, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 30, 2018

Completed
11 months until next milestone

Results Posted

Study results publicly available

December 5, 2019

Completed
Last Updated

December 5, 2019

Status Verified

February 1, 2019

Enrollment Period

2.3 years

First QC Date

January 18, 2016

Results QC Date

November 18, 2019

Last Update Submit

November 18, 2019

Conditions

Friedreich's Ataxia

Outcome Measures

Primary Outcomes (2)

  • 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.

    Baseline, 24 weeks

  • 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.

    Baseline, 24 weeks

Study Arms (1)

(+)-Epicatechin

EXPERIMENTAL

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

Drug: (+)-Epicatechin

Interventions

(+)-EpicatechinDRUG

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)

(+)-Epicatechin

Eligibility Criteria

Age10 Years - 50 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Related Publications (1)

  • 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.

    PMID: 32677106DERIVED

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

Catechin

Condition Hierarchy (Ancestors)

Spinocerebellar DegenerationsCerebellar DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesSpinal Cord DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMitochondrial DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

ChromansBenzopyransPyransHeterocyclic Compounds, 1-RingHeterocyclic CompoundsFlavonoidsChromonesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-Ring

Results Point of Contact

Title
Ralitza H. Gavrilova, M.D.
Organization
Mayo Clinic
Gavrilova.Ralitza@mayo.edu
507-293-1283

Study Officials

  • Ralitza H Gavrilova, MD

    Mayo Clinic

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
MD, Sponsor-Investigator

Study Record Dates

First Submitted

January 18, 2016

First Posted

January 21, 2016

Study Start

September 1, 2016

Primary Completion

December 30, 2018

Study Completion

December 30, 2018

Last Updated

December 5, 2019

Results First Posted

December 5, 2019

Record last verified: 2019-02

Data Sharing

IPD Sharing
Will not share

Locations

US(1)