NCT01728064

Brief Summary

The purpose of this study is to examine the effects of EPI-743 on visual function and neurologic function in patients with Friedreich's ataxia.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
60

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Dec 2012

Typical duration for phase_2

Geographic Reach
1 country

3 active sites

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

November 13, 2012

Completed
3 days until next milestone

First Posted

Study publicly available on registry

November 16, 2012

Completed
2 months until next milestone

Study Start

First participant enrolled

December 31, 2012

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2015

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

February 29, 2016

Completed
Last Updated

August 31, 2020

Status Verified

August 1, 2020

Enrollment Period

2.8 years

First QC Date

November 13, 2012

Last Update Submit

August 24, 2020

Conditions

Friedreich's Ataxia

Keywords

Friedreich'sAtaxiaFRDAFAEPI-743Edison

Outcome Measures

Primary Outcomes (1)

  • Visual Function

    Low contrast visual acuity

Secondary Outcomes (10)

  • Color vision

  • Neurologic function

  • Neuromuscular function

  • Neuromuscular function

  • Quality of life

  • +5 more secondary outcomes

Study Arms (3)

Placebo

PLACEBO COMPARATOR

Placebo capsules three times daily

Drug: Placebo

EPI-743 400 mg

ACTIVE COMPARATOR

EPI-743 at a dose of 400 mg three times daily

Drug: EPI-743 400 mg

EPI-743 200 mg

ACTIVE COMPARATOR

EPI-743 at a dose of 200 mg three times daily

Drug: EPI-743 200 mg

Interventions

PlaceboDRUG

Placebo capsules

Placebo
EPI-743 400 mgDRUG
EPI-743 400 mg
EPI-743 200 mgDRUG
EPI-743 200 mg

Eligibility Criteria

Age18 Years - 45 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • Diagnosis of genetically confirmed Friedreich's ataxia
  • Visual acuity at baseline more than 15 letters on EDTRS at four meters
  • FARS score of 20 to 90
  • Agreement to use contraception if within reproductive years (see specifics in section D1, page 21)
  • Hormone replacement therapy, if used, must remain stable for the duration of the study
  • Willingness and ability to comply with study procedures
  • Willingness and ability to arrive at study site day prior to evaluations
  • Abstention from use of dietary supplements and non-prescribed medications at least 30 days prior to initiation of treatment and for the duration of the study. This would specifically include idebenone, Coenzyme Q10 and vitamin E
  • Abstention from use of other investigative or non-approved drugs within 30 days of enrollment and for the duration of the study

You may not qualify if:

  • Allergy to EPI-743 or sesame oil or nuts
  • Clinically significant bleeding condition or abnormal PT/PTT INR (INR \> two; PTT \> two-times normal)
  • Liver insufficiency with LFTs greater than three-times upper normal limit at screening
  • Renal insufficiency with creatinine \> 1.5 at screening
  • Fat malabsorption syndromes
  • Any other respiratory chain diseases of the mitochondria or inborn errors of metabolism
  • Any other ophthalmologic conditions
  • History of alcohol or drug abuse
  • Clinically significant cardiomyopathy with ejection fraction \< 40 percent at screening
  • Clinically significant arrhythmia within past two years requiring treatment
  • Anticoagulant therapy within 30 days of enrollment.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

University of California Los Angeles

Los Angeles, California, 90095, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Links

MeSH Terms

Conditions

Friedreich AtaxiaAtaxia

Interventions

alpha-tocotrienol quinone

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 DiseasesDyskinesiasNeurologic ManifestationsSigns and SymptomsPathological Conditions, Signs and Symptoms

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 13, 2012

First Posted

November 16, 2012

Study Start

December 31, 2012

Primary Completion

October 31, 2015

Study Completion

February 29, 2016

Last Updated

August 31, 2020

Record last verified: 2020-08

Locations

US(3)