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EPI-743

DRUG11 trials

Sponsors

PTC Therapeutics, National Human Genome Research Institute (NHGRI), Yale University, Bambino Gesù Hospital and Research Institute, Edison Pharmaceuticals Inc

Conditions

Autism Spectrum DisorderFriedreich's AtaxiaGenetic DiseaseLeber's Hereditary Optic NeuropathyLeigh SyndromeMethylmalonic Aciduria and Homocystinuria,Cblc TypeMitochondrial DiseaseMitochondrial Diseases

Phase 1

EPI-743 for Metabolism or Mitochondrial Disorders
CompletedNCT01642056
National Human Genome Research Institute (NHGRI)Mitochondrial Disease, Myopathy, Neurology
Start: 2012-09-01End: 2019-09-24Updated: 2021-04-14
Open-Trial of EPI-743 for Adults With Tourette Syndrome
CompletedNCT01719523
Yale UniversityTourette Syndrome
Start: 2012-10-31End: 2013-10-31Updated: 2014-03-05

Phase 2

EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment
CompletedNCT01793090
Bambino Gesù Hospital and Research InstituteGenetic Disease, Methylmalonic Aciduria and Homocystinuria,Cblc Type, Retinopathy
Start: 2013-01-31End: 2017-02-28Updated: 2017-04-25
Phase 2 Study of EPI-743 for Treatment of Rett Syndrome
CompletedNCT01822249
Edison Pharmaceuticals IncRett Syndrome
Start: 2013-01-31End: 2014-01-31Updated: 2018-07-26
EPI-743 in Friedreich's Ataxia Point Mutations
CompletedNCT01962363
University of South FloridaFriedreich's Ataxia
Start: 2013-10-31End: 2016-06-30Updated: 2016-06-29
Phase 2 Study of EPI-743 in Children With Pearson Syndrome
TerminatedNCT02104336
Edison Pharmaceuticals IncPearson Syndrome
Start: 2014-08-31End: 2016-02-29Updated: 2020-11-18
An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder
WithdrawnNCT02226458
Edison Pharmaceuticals IncAutism Spectrum Disorder
Start: 2014-10-31End: 2016-01-31Updated: 2022-01-21
Protective Effects of EPI-743 on Noise-Induced Hearing Loss
CompletedNCT02257983
Edison Pharmaceuticals IncNoise-induced Hearing Loss
Start: 2014-10-31End: 2016-02-29Updated: 2020-11-18
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
CompletedNCT02352896
PTC TherapeuticsLeigh Syndrome
Start: 2014-01-31End: 2023-10-30Updated: 2024-10-23

Unknown Phase

EPI-743 for Mitochondrial Respiratory Chain Diseases
NCT01370447
PTC TherapeuticsMitochondrial Diseases
Updated: 2024-01-23
Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]
NCT02300753
Edison Pharmaceuticals IncLeber's Hereditary Optic Neuropathy
Updated: 2014-11-25