Leber's Hereditary Optic NeuropathyNCT02300753
Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]
A study testing EPI-743 in people with Leber's Hereditary Optic Neuropathy, run by Edison Pharmaceuticals Inc at 1 site in United States.
Trial summary
Expanded access Protocol to treat LHON subjects with EPI743 Read more about this study
- Who is it for?
- Ages 8 to 65 years
- Conditions the study is for
- Leber's Hereditary Optic Neuropathy
- What is being tested?
- EPI-743
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 8 to 65 years
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Emergency treatment for subjects with LHON who are actively losing site in one eye
Who cannot join
- Allergy to EPI-743 or sesame oil
- Clinical history of bleeding or abnormal PT/PTT
- Hepatic insufficiency with LFTs greater than 2-times normal
- Renal insufficiency requiring dialysis
- Fat malabsorption syndromes
- Any other concurrent inborn erros of metabolism
- Severe end-organ hypo-perfusion syndrome secondary to cardiac failure resulting in lactic acidosis
- Anemia with a HCT <25
Trial locations and contacts
1 site in 1 country.
United States1 site
Doheny Eye Institute / UCLA
Los Angeles, California, 90033
About this study
Expanded access Protocol to treat LHON subjects with EPI743
Read the detailed description
Emergency use administration of EPI-743 to subjects with LHON. This Protocol was originally developed to treat a single subject but was expanded to treat several at a single institution.
- Conditions
- Leber's Hereditary Optic Neuropathy
- Being tested
- EPI-743
- Medical subject headings
- Optic Atrophy, Hereditary, Leber
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EPI-743Drug
Also called Vincerinone (TM), Vatiquinone
EPI-743 is a quinone oxidation product of alpha-tocotrienol
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leber's Hereditary Optic NeuropathyStudies 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Edison Pharmaceuticals Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02300753
- Sponsor's protocol ID
- EPI-2010-004
- Status last verified
- Nov 2014
- First submitted
- 21 Nov 2014
- Last updated
- 25 Nov 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Nov 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]
- ClinicalTrials.gov ID
- NCT02300753
- Acronym / sponsor ID
- EPI-2010-004
- Conditions
- Leber's Hereditary Optic Neuropathy
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Edison Pharmaceuticals IncIndustry (pharma, biotech, devices)
- First posted
- 25 Nov 2014 (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.
Expanded access Protocol to treat LHON subjects with EPI743
Detailed description
Emergency use administration of EPI-743 to subjects with LHON. This Protocol was originally developed to treat a single subject but was expanded to treat several at a single institution.
Study design
Who may qualify
- Ages
- 8 to 65 years
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Emergency treatment for subjects with LHON who are actively losing site in one eye
Key exclusion criteria (8 listed)
- Allergy to EPI-743 or sesame oil
- Clinical history of bleeding or abnormal PT/PTT
- Hepatic insufficiency with LFTs greater than 2-times normal
- Renal insufficiency requiring dialysis
- 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 (TM), VatiquinoneEPI-743 is a quinone oxidation product of alpha-tocotrienol | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Nov 2014.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing EPI-743 in people with Leber's Hereditary Optic Neuropathy, run by Edison Pharmaceuticals Inc 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 1 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 25 Nov 2014. 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.
Are results available for this study?
Results are not available in the source record.
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.
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 25 Nov 2014.