Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]
Primary Purpose
Leber's Hereditary Optic Neuropathy
Status
No longer available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
EPI-743
Sponsored by
About this trial
This is an expanded access trial for Leber's Hereditary Optic Neuropathy
Eligibility Criteria
Inclusion Criteria:
- Emergency treatment for subjects with LHON who are actively losing site in one eye
Exclusion Criteria:
- 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
Sites / Locations
- Doheny Eye Institute / UCLA
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT02300753
First Posted
November 21, 2014
Last Updated
November 24, 2014
Sponsor
Edison Pharmaceuticals Inc
1. Study Identification
Unique Protocol Identification Number
NCT02300753
Brief Title
Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]
Official Title
Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]
Study Type
Expanded Access
2. Study Status
Record Verification Date
November 2014
Overall Recruitment Status
No longer available
Study Start Date
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Primary Completion Date
undefined (undefined)
Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Edison Pharmaceuticals Inc
4. Oversight
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leber's Hereditary Optic Neuropathy
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
EPI-743
Other Intervention Name(s)
Vincerinone (TM), Vatiquinone
Intervention Description
EPI-743 is a quinone oxidation product of alpha-tocotrienol
10. Eligibility
Sex
All
Minimum Age & Unit of Time
8 Years
Maximum Age & Unit of Time
65 Years
Eligibility Criteria
Inclusion Criteria:
Emergency treatment for subjects with LHON who are actively losing site in one eye
Exclusion Criteria:
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
Facility Information:
Facility Name
Doheny Eye Institute / UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]
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