NCT02300753

Brief Summary

Expanded access Protocol to treat LHON subjects with EPI743

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 21, 2014

Completed
4 days until next milestone

First Posted

Study publicly available on registry

November 25, 2014

Completed
Last Updated

November 25, 2014

Status Verified

November 1, 2014

First QC Date

November 21, 2014

Last Update Submit

November 24, 2014

Conditions

Interventions

EPI-743 is a quinone oxidation product of alpha-tocotrienol

Also known as: Vincerinone (TM), Vatiquinone

Eligibility Criteria

Age8 Years - 65 Years
Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Emergency treatment for subjects with LHON who are actively losing site in one eye

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Doheny Eye Institute / UCLA

Los Angeles, California, 90033, United States

Location

MeSH Terms

Conditions

Optic Atrophy, Hereditary, Leber

Interventions

alpha-tocotrienol quinone

Condition Hierarchy (Ancestors)

Optic Atrophies, HereditaryOptic AtrophyOptic Nerve DiseasesCranial Nerve DiseasesNervous System DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesEye Diseases, HereditaryEye DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMitochondrial DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 21, 2014

First Posted

November 25, 2014

Last Updated

November 25, 2014

Record last verified: 2014-11

Locations