Friedreich's AtaxiaPhase 2NCT01962363
EPI-743 in Friedreich's Ataxia Point Mutations
A Phase 2 study testing EPI-743 in people with Friedreich's Ataxia, run by University of South Florida at 1 site in United States.
Trial summary
The purpose of this study is to evaluate the effects of EPI-743 in patients with Friedreich's Ataxia point mutations Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Friedreich's Ataxia
- What is being tested?
- EPI-743
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2013to Jun 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Show all 13Show fewer
- 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.
Who cannot join
- 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.
Show all 12Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2A Clinical Trial of EPI-743 (Vincerinone™) on Visual Function in Friedreich's Ataxia Patients With Point Mutations
The purpose of this study is to evaluate the effects of EPI-743 in patients with Friedreich's Ataxia point mutations
Read the detailed 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.
- Conditions
- Friedreich's Ataxia
- Being tested
- EPI-743
- Keywords
- Friedreich's ataxia point mutation
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EPI-743Drug
Also called Vincerinone, alpha-tocotrienol quinone
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.
| Study group | Role | What they receive |
|---|---|---|
| EPI-743 | Experimental | EPI-743, oral, 400mg three times daily for 3 months Receives: Drug: EPI-743 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Visual function
Low contrast acuity
Measured: 3 months
Safety parameters
Clinical and laboratory safety parameters
Measured: 3 months
Secondary outcomes (8)
Visual function
Visual fields as assessed by Humphrey 30-2 exam; High contrast visual acuity
Measured: 3 months
Neurologic function
Friedreich's Ataxia Rating Scale
Measured: 3 months
Physical function
25-foot walk (for subjects capable of completing the test on enrollment);
Measured: 3 months
Health related quality of life
Patient report via rating scale
Measured: 3 months
Activities of Daily Living
Patient report via rating scale
Measured: 3 months
Cardiac indices
Echocardiogram
Measured: 3 months
Upper extremity function
9 hole peg test
Measured: 3 months
Disease biomarkers
Glutathione cycle components
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2013 (estimated)
- Study startOct 2013
- Primary completionJun 2016
- Study completionJun 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
- More trials run by University of South FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Edison Pharmaceuticals Inc, Friedreich's Ataxia Research Alliance
- Responsible party
- Sponsor
- Investigators
- Theresa A Zesiewicz, MD, Principal investigator, University of South Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01962363
- Sponsor's protocol ID
- EPI-743 PM
- Status last verified
- Jun 2016
- First submitted
- 10 Oct 2013
- Last updated
- 29 Jun 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2A Clinical Trial of EPI-743 (Vincerinone™) on Visual Function in Friedreich's Ataxia Patients With Point Mutations
- ClinicalTrials.gov ID
- NCT01962363
- Acronym / sponsor ID
- EPI-743 PM
- Conditions
- Friedreich's Ataxia
- Keywords
- Friedreich's ataxia point mutation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- Collaborators
- Edison Pharmaceuticals Inc, Friedreich's Ataxia Research Alliance
- Study start
- Oct 2013
- Primary completion
- Jun 2016 (actual)
- Study completion
- Jun 2016 (actual)
- First posted
- 14 Oct 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the effects of EPI-743 in patients with Friedreich's Ataxia point mutations
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| EPI-743 | Experimental | Drug: EPI-743EPI-743, oral, 400mg three times daily for 3 months |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- 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
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EPI-743Also: Vincerinone, alpha-tocotrienol quinoneEPI-743 (alpha-tocotrienol quinone) is a small molecule therapeutic that was rationally designed to replete reduced glutathione through NQO1-catalyzed electron transfer from NADPH. | Drug | EPI-743 |
Primary outcome
Visual function
Time frame: 3 months
Low contrast acuity
Safety parameters
Time frame: 3 months
Clinical and laboratory safety parameters
8 secondary outcomes
Visual function
Time frame: 3 months
Neurologic function
Time frame: 3 months
Physical function
Time frame: 3 months
Health related quality of life
Time frame: 3 months
Activities of Daily Living
Time frame: 3 months
Cardiac indices
Time frame: 3 months
Upper extremity function
Time frame: 3 months
Disease biomarkers
Time frame: 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2016.
Related trials for Friedreich's Ataxia
RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Friedreich's Ataxia (FA)
Active, not recruiting
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
Friedreich Ataxia
Recruiting
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Friedreich's Ataxia, Motor Disorders
Active, not recruitingObservational study
Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia
Friedreich Ataxia
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Theresa A Zesiewicz, MD principal investigator
University of South Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing EPI-743 in people with Friedreich's Ataxia, run by University of South Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Jun 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Visual function (time frame: 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jun 2016.