NCT02226458 (Autism) is a Phase 2 clinical trial of EPI-743 in Autism Spectrum Disorder, sponsored by Edison Pharmaceuticals Inc.

Who is sponsoring NCT02226458?

NCT02226458 is sponsored by Edison Pharmaceuticals Inc.

What drugs are being tested in NCT02226458?

Interventions in NCT02226458: EPI-743.

What condition does NCT02226458 study?

NCT02226458 studies Autism Spectrum Disorder.

Is NCT02226458 still recruiting?

NCT02226458 is currently withdrawn. Last updated 21 January 2022.

Last reviewed 2022-01-21 · How we verify

NCT02226458: Autism

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder

Withdrawn Phase 2 Last updated 21 January 2022

What this trial tests

Phase 2 trial testing EPI-743 in Autism Spectrum Disorder. Withdrawn.

Timeline

31 October 2014

Primary endpoint
30 November 2015

31 January 2016

Quick facts

Lead sponsor	Edison Pharmaceuticals Inc
Phase	Phase 2
Status	Withdrawn
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Start date	31 October 2014
Primary completion	30 November 2015
Estimated completion	31 January 2016

Drugs / interventions tested

EPI-743 — full drug profile →

Conditions studied

Autism Spectrum Disorder — all drugs for Autism Spectrum Disorder →

Sponsor

Edison Pharmaceuticals Inc — full company profile →

Who can join

Adults 3 to 14, any sex, with Autism Spectrum Disorder. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The investigators hypothesize that EPI-743 may provide clinical benefit to children with Autism Spectrum Disorder.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

Evaluating the efficacy of vatiquinone in preclinical models of Leigh syndrome and GPX4 deficiency.
Kayser EB, Mulholland M, Olkhova EA, Chen Y, et al · · 2025 · cited 2× · PMID 39930437 · DOI 10.1186/s13023-025-03582-x

Verify or expand the search:

Other trials of EPI-743

Trials testing the same drug.

NCT02257983 — Protective Effects of EPI-743 on Noise-Induced Hearing Loss · Phase 2 · completed
NCT02104336 — Phase 2 Study of EPI-743 in Children With Pearson Syndrome · Phase 2 · terminated
NCT02352896 — Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome · Phase 2 · completed
NCT01642056 — EPI-743 for Metabolism or Mitochondrial Disorders · Phase 1, PHASE2 · completed
NCT01370447 — EPI-743 for Mitochondrial Respiratory Chain Diseases · no longer available

Other recruiting trials for Autism Spectrum Disorder

Currently open trials in the same condition.

NCT07324057 — The Effects of Preoperative Intranasal Administration of Dexmedetomidine and Esketamine on Negative Postoperative Behavi · NA · recruiting
NCT07493096 — Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal Disor · recruiting
NCT07505290 — Efficacy of the Korean PEERS® for Preschoolers (PEERS®-PS-K) Social Skills Intervention: A Randomized Controlled Trial f · NA · active not recruiting
NCT07524192 — A Self-Instructional Online Program for Early Childhood and Elementary Teachers Supporting Autistic Children and Childre · NA · recruiting
NCT07432776 — Effect of an 8-Week Pickleball Program for Adults With Autism: A Feasibility Trial With a Delayed-Control Design · EARLY_PHASE1 · recruiting

Other Edison Pharmaceuticals Inc trials

Trials by the same sponsor.

NCT02462603 — Safety and Biomarker Study of PTC-589 in Participants With Parkinson's Disease · Phase 2 · completed
NCT02360436 — Analysis of the Glutathione Cycle in Children With Rett Syndrome · withdrawn
NCT02257983 — Protective Effects of EPI-743 on Noise-Induced Hearing Loss · Phase 2 · completed
NCT02104336 — Phase 2 Study of EPI-743 in Children With Pearson Syndrome · Phase 2 · terminated

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT02226458 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Edison Pharmaceuticals Inc
Last refreshed: 21 January 2022

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02226458.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing