EPI-743 in Friedreich's Ataxia Point Mutations

Friedreich's Ataxia Clinical Trial

Official title:

A Phase 2A Clinical Trial of EPI-743 (Vincerinone™) on Visual Function in Friedreich's Ataxia Patients With Point Mutations

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01962363
• Other study ID #: EPI-743 PM
• Status: Completed
• Phase: Phase 2
• First received: October 10, 2013
• Last updated: June 28, 2016
• Start date: October 2013
• Est. completion date: June 2016

Study information

• Verified date: June 2016
• Source: University of South Florida
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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

Description:

The primary objective of this study is to investigate whether treatment with EPI-743 has a discernible impact on visual function—including visual acuity, visual fields and color vision as well as on any of a number of functional and subject/clinician-rated scales relevant in the treatment of Friedreich's ataxia, and to determine the safety of treatment with EPI-743.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: June 2016
• Est. primary completion date: June 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

Inclusion Criteria:

1. Diagnosis of genetically confirmed Friedreich's ataxia point mutation.

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

3. FARS score of 20 to 90.

4. Male or female between 18 and 65 years of age.

5. Agreement to use contraception if within reproductive years

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

7. Willingness and ability to comply with study procedures.

8. Willingness and ability to arrive at study site metropolitan area day prior to evaluations.

9. 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.

10. 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.

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

12. Subject can swallow multiple size 0 capsules.

13. 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.

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

9. Clinically significant arrhythmia within past two years requiring treatment.

10. Surgery planned through the duration of the study, including follow-up.

11. Pregnancy or breastfeeding.

12. Anticoagulant therapy within 30 days of enrollment.

Study Design

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

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

Intervention

Drug:
• EPI-743
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.

Locations

Country	Name	City	State
United States	University of South Florida	Tampa	Florida

Sponsors (3)

Lead Sponsor	Collaborator
University of South Florida	Edison Pharmaceuticals Inc, Friedreich's Ataxia Research Alliance

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Visual function	Low contrast acuity	3 months	No
Primary	Safety parameters	Clinical and laboratory safety parameters	3 months	Yes
Secondary	Visual function	Visual fields as assessed by Humphrey 30-2 exam; High contrast visual acuity	3 months	No
Secondary	Neurologic function	Friedreich's Ataxia Rating Scale	3 months	No
Secondary	Physical function	25-foot walk (for subjects capable of completing the test on enrollment);	3 months	No
Secondary	Health related quality of life	Patient report via rating scale	3 months	No
Secondary	Activities of Daily Living	Patient report via rating scale	3 months	No
Secondary	Cardiac indices	Echocardiogram	3 months	Yes
Secondary	Upper extremity function	9 hole peg test	3 months	No
Secondary	Disease biomarkers	Glutathione cycle components	3 months	No