EPI-743 in Friedreich's Ataxia Point Mutations

NCT01962363 | Completed Phase 2

• 1 other identifier: EPI-743 PM
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the effects of EPI-743 in patients with Friedreich's Ataxia point mutations

Conditions

Friedreich's Ataxia

Keywords

Friedreich's ataxia point mutation

Outcome Measures

Primary Outcomes (2)

• Visual function

  Low contrast acuity

  3 months

• Safety parameters

  Clinical and laboratory safety parameters

  3 months

Secondary Outcomes (8)

• Visual function

  3 months

• Neurologic function

  3 months

• Physical function

  3 months

• Health related quality of life

  3 months

• Activities of Daily Living

  3 months

Study Arms (1)

EPI-743

EXPERIMENTAL

EPI-743, oral, 400mg three times daily for 3 months

Drug: EPI-743

Interventions

EPI-743 DRUG

EPI-743 (alpha-tocotrienol quinone) is a small molecule therapeutic that was rationally designed to replete reduced glutathione through NQO1-catalyzed electron transfer from NADPH.

Also known as: Vincerinone, alpha-tocotrienol quinone

EPI-743

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of genetically confirmed Friedreich's ataxia point mutation.
• Visual acuity at baseline more than 15 letters on high contrast EDTRS at four meters.
• FARS score of 20 to 90.
• Male or female between 18 and 65 years of age.
• Agreement to use contraception if within reproductive years
• 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 metropolitan area 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 foods or beverages or bars fortified with Coenzyme Q10, vitamin E, super fortified functional foods or beverages at least 30 days prior to initiation of treatment and for the duration of the study.
• Abstention from use of other investigative or non-approved drugs within 30 days of enrollment and for the duration of the study.
• Subject can swallow multiple size 0 capsules.
• Subject has voluntarily signed an IRB approved informed consent form to participate in the study after all relevant aspects of the study have been explained and discussed with the subject.

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.
• Clinically significant cardiomyopathy with ejection fraction \< 40 percent at screening.
• Clinically significant arrhythmia within past two years requiring treatment.
• Surgery planned through the duration of the study, including follow-up.
• Pregnancy or breastfeeding.
• Anticoagulant therapy within 30 days of enrollment.

Sponsors & Collaborators

• University of South Florida: lead
• Edison Pharmaceuticals Inc: collaborator
• Friedreich's Ataxia Research Alliance: collaborator

Study Sites (1)

University of South Florida

Tampa, Florida, 33612, United States

Location

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

alpha-tocotrienol quinone

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Theresa A Zesiewicz, MD

  University of South Florida

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 10, 2013
• First Posted: October 14, 2013
• Study Start: October 1, 2013
• Primary Completion: June 1, 2016
• Study Completion: June 1, 2016
• Last Updated: June 29, 2016

Record last verified: 2016-06

Locations

US (1)