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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
Edison Pharmaceuticals Inc
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Leber's Hereditary Optic Neuropathy

Eligibility Criteria

8 Years - 65 Years (Child, Adult, Older Adult)All Sexes

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

First Posted
November 21, 2014
Last Updated
November 24, 2014
Sponsor
Edison Pharmaceuticals Inc
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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
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

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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