Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

Friedreich's Ataxia Clinical Trial

Official title:

Safety and Efficacy Study of EPI-743 on Visual Function in Patients With Friedreich's Ataxia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01728064
• Other study ID #: EPI2010-006
• Status: Completed
• Phase: Phase 2
• Start date: December 31, 2012
• Est. completion date: February 29, 2016

Study information

• Verified date: August 2020
• Source: PTC Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 60
• Est. completion date: February 29, 2016
• Est. primary completion date: October 31, 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 45 Years

Eligibility

Inclusion Criteria:

1. Diagnosis of genetically confirmed Friedreich's ataxia

2. Visual acuity at baseline more than 15 letters on EDTRS at four meters

3. FARS score of 20 to 90

4. Agreement to use contraception if within reproductive years (see specifics in section D1, page 21)

5. Hormone replacement therapy, if used, must remain stable for the duration of the study

6. Willingness and ability to comply with study procedures

7. Willingness and ability to arrive at study site day prior to evaluations

8. 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

9. Abstention from use of other investigative or non-approved drugs within 30 days of enrollment and for the duration of the study

Exclusion Criteria:

1. Allergy to EPI-743 or sesame oil or nuts

2. Clinically significant bleeding condition or abnormal PT/PTT INR (INR > two; PTT > two-times normal)

3. Liver insufficiency with LFTs greater than three-times upper normal limit at screening

4. Renal insufficiency with creatinine > 1.5 at screening

5. Fat malabsorption syndromes

6. Any other respiratory chain diseases of the mitochondria or inborn errors of metabolism

7. Any other ophthalmologic conditions

8. History of alcohol or drug abuse

9. Clinically significant cardiomyopathy with ejection fraction < 40 percent at screening

10. Clinically significant arrhythmia within past two years requiring treatment

11. Anticoagulant therapy within 30 days of enrollment.

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia
• Friedreich's Ataxia

Intervention

Drug:
• Placebo
Placebo capsules
• EPI-743 400 mg

• EPI-743 200 mg

Locations

Country	Name	City	State
United States	University of California Los Angeles	Los Angeles	California
United States	Childrens Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	University of South Florida	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
PTC Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Visual Function	Low contrast visual acuity
Secondary	Color vision	Roth 28 hue test
Secondary	Neurologic function	Friedreich's ataxia rating scale
Secondary	Neuromuscular function	25-foot walk test
Secondary	Neuromuscular function	9-hole peg test
Secondary	Quality of life	SF-36
Secondary	Disease biomarkers	Blood biomarker levels
Secondary	Cardiac function	Echocardiogram
Secondary	Safety	Number of adverse events
Secondary	Disease improvement	Patient Global Improvement Scale
Secondary	Visual Function	Visual field exam	Baseline, Months 3, 6, 9 and 12

External Links

•   Edison Pharmaceuticals Web Site