Friedreich's AtaxiaPhase 2NCT01728064
Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
A Phase 2 study testing EPI-743 400 mg and EPI-743 200 mg in people with Friedreich's Ataxia, run by PTC Therapeutics at 3 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Friedreich's Ataxia
- What is being tested?
- EPI-743 400 mg and EPI-743 200 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 3 sitesUnited States
- Study dates
- 31 Dec 2012to 29 Feb 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 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
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- 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
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
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- 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.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of California Los Angeles
Los Angeles, California, 90095
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of South Florida
Tampa, Florida, 33612
About this study
Official title: Safety and Efficacy Study of EPI-743 on Visual Function in Patients With Friedreich's Ataxia
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.
- Conditions
- Friedreich's Ataxia
- Being tested
- Placebo, EPI-743 400 mg, EPI-743 200 mg
- Keywords
- Friedreich's, Ataxia, FRDA, FA, EPI-743, Edison
- Medical subject headings
- Friedreich Ataxia, Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Placebo capsules
EPI-743 400 mgDrug
EPI-743 200 mgDrug
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo capsules three times daily Receives: Drug: Placebo |
| EPI-743 400 mg | Active comparator | EPI-743 at a dose of 400 mg three times daily Receives: Drug: EPI-743 400 mg |
| EPI-743 200 mg | Active comparator | EPI-743 at a dose of 200 mg three times daily Receives: Drug: EPI-743 200 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Visual Function
Low contrast visual acuity
Secondary outcomes (10)
Color vision
Roth 28 hue test
Neurologic function
Friedreich's ataxia rating scale
Neuromuscular function
25-foot walk test
Neuromuscular function
9-hole peg test
Quality of life
SF-36
Disease biomarkers
Blood biomarker levels
Cardiac function
Echocardiogram
Safety
Number of adverse events
Disease improvement
Patient Global Improvement Scale
Visual Function
Visual field exam
Measured: Baseline, Months 3, 6, 9 and 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Nov 2012 (estimated)
- Study start31 Dec 2012
- Primary completion31 Oct 2015
- Study completion29 Feb 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.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- PTC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01728064
- Sponsor's protocol ID
- EPI2010-006
- Status last verified
- Aug 2020
- First submitted
- 13 Nov 2012
- Last updated
- 31 Aug 2020
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy Study of EPI-743 on Visual Function in Patients With Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT01728064
- Acronym / sponsor ID
- EPI2010-006
- Conditions
- Friedreich's Ataxia
- Keywords
- Friedreich's, Ataxia, FRDA, FA, EPI-743, Edison
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 31 Dec 2012 (actual)
- Primary completion
- 31 Oct 2015 (actual)
- Study completion
- 29 Feb 2016 (actual)
- First posted
- 16 Nov 2012 (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 examine the effects of EPI-743 on visual function and neurologic function in patients with Friedreich's ataxia.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPlacebo capsules three times daily |
| EPI-743 400 mg | Active comparator | Drug: EPI-743 400 mgEPI-743 at a dose of 400 mg three times daily |
| EPI-743 200 mg | Active comparator | Drug: EPI-743 200 mgEPI-743 at a dose of 200 mg three times daily |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (11 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 |
|---|---|---|
| PlaceboPlacebo capsules | Drug | Placebo |
| EPI-743 400 mg | Drug | EPI-743 400 mg |
| EPI-743 200 mg | Drug | EPI-743 200 mg |
Primary outcome
Visual Function
Low contrast visual acuity
10 secondary outcomes
Color vision
Neurologic function
Neuromuscular function
Neuromuscular function
Quality of life
Disease biomarkers
Cardiac function
Safety
Disease improvement
Visual Function
Time frame: Baseline, Months 3, 6, 9 and 12
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 31 Aug 2020.
- Edison Pharmaceuticals Web SiteLink provided by the sponsor.
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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.
The record lists no contact. Use the official record to reach the sponsor.
3 sites 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-743 400 mg and EPI-743 200 mg in people with Friedreich's Ataxia, run by PTC Therapeutics at 3 sites 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 9 inclusion and 11 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 31 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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. Some participants in this study may receive a placebo. 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. 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 31 Aug 2020.