NCT02104336 is a Phase 2 clinical trial of EPI-743 in Pearson Syndrome, sponsored by Edison Pharmaceuticals Inc.

Who is sponsoring NCT02104336?

NCT02104336 is sponsored by Edison Pharmaceuticals Inc.

What drugs are being tested in NCT02104336?

Interventions in NCT02104336: EPI-743.

What condition does NCT02104336 study?

NCT02104336 studies Pearson Syndrome.

Is NCT02104336 still recruiting?

NCT02104336 is currently terminated. Last updated 18 November 2020.

Last reviewed 2020-11-18 · How we verify

NCT02104336

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

Phase 2 Study of EPI-743 in Children With Pearson Syndrome

Terminated Phase 2 Last updated 18 November 2020

What this trial tests

Phase 2 trial testing EPI-743 in Pearson Syndrome in 2 participants. Terminated before completion.

Timeline

31 August 2014

Primary endpoint
30 November 2015

29 February 2016

Quick facts

Lead sponsor	Edison Pharmaceuticals Inc
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	2
Start date	31 August 2014
Primary completion	30 November 2015
Estimated completion	29 February 2016
Sites	4 locations across United States

Drugs / interventions tested

EPI-743 — full drug profile →

Conditions studied

Pearson Syndrome — all drugs for Pearson Syndrome →

Sponsor

Edison Pharmaceuticals Inc — full company profile →

Who can join

Under 18, any sex, with Pearson Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Treatment of Pediatric Subjects with Pearson syndrome

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Emerging therapies for mitochondrial diseases.
Hirano M, Emmanuele V, Quinzii CM. · · 2018 · cited 122× · PMID 29980632 · DOI 10.1042/ebc20170114
Mitochondrial disorders in children: toward development of small-molecule treatment strategies.
Koopman WJ, Beyrath J, Fung CW, Koene S, et al · · 2016 · cited 72× · PMID 26951622 · DOI 10.15252/emmm.201506131
Current and Emerging Clinical Treatment in Mitochondrial Disease.
Tinker RJ, Lim AZ, Stefanetti RJ, McFarland R. · · 2021 · cited 64× · PMID 33646563 · DOI 10.1007/s40291-020-00510-6
Clinical trials in mitochondrial disorders, an update.
Almannai M, El-Hattab AW, Ali M, Soler-Alfonso C, et al · · 2020 · cited 56× · PMID 33129691 · DOI 10.1016/j.ymgme.2020.10.002
Broadening the phenotypic spectrum of Pearson syndrome: Five new cases and a review of the literature.
Wild KT, Goldstein AC, Muraresku C, Ganetzky RD. · · 2020 · cited 30× · PMID 31825167 · DOI 10.1002/ajmg.a.61433
Challenges and opportunities to bridge translational to clinical research for personalized mitochondrial medicine.
Gropman AL, Uittenbogaard MN, Chiaramello AE. · · 2024 · cited 11× · PMID 38266483 · DOI 10.1016/j.neurot.2023.e00311
Therapies for Mitochondrial Disease: Past, Present, and Future.
Ball M, van Bergen NJ, Compton AG, Thorburn DR, et al · · 2025 · cited 3× · PMID 40714961 · DOI 10.1002/jimd.70065
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.

NCT02226458 — An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder · Phase 2 · withdrawn
NCT02257983 — Protective Effects of EPI-743 on Noise-Induced Hearing Loss · Phase 2 · completed
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 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
NCT02226458 — An Exploratory Open Label Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder · Phase 2 · withdrawn
NCT02257983 — Protective Effects of EPI-743 on Noise-Induced Hearing Loss · Phase 2 · completed

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 NCT02104336 (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: 18 November 2020

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/NCT02104336.

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