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NCT01370447
EPI-743 for Mitochondrial Respiratory Chain Diseases
NO LONGER AVAILABLE
Last updated 23 January 2024
What this trial tests
trial testing EPI-743 in Mitochondrial Diseases. No longer available.
Quick facts
| Lead sponsor | PTC Therapeutics |
|---|---|
| Status | NO LONGER AVAILABLE |
| Study type | EXPANDED_ACCESS |
| Sites | 16 locations across United States |
Drugs / interventions tested
- EPI-743 — full drug profile →
Conditions studied
- Mitochondrial Diseases — all drugs for Mitochondrial Diseases →
Sponsor
PTC Therapeutics — full company profile →
Who can join
1 and older, any sex, with Mitochondrial Diseases. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
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Oxidative stress in the eye and its role in the pathophysiology of ocular diseases.
Böhm EW, Buonfiglio F, Voigt AM, Bachmann P, et al · · 2023 · cited 169× · PMID 38006824 · DOI 10.1016/j.redox.2023.102967 -
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 -
Moving towards clinical trials for mitochondrial diseases.
Pitceathly RDS, Keshavan N, Rahman J, Rahman S. · · 2021 · cited 52× · PMID 32618366 · DOI 10.1002/jimd.12281 -
Oxidative Stress: A Suitable Therapeutic Target for Optic Nerve Diseases?
Buonfiglio F, Böhm EW, Pfeiffer N, Gericke A. · · 2023 · cited 41× · PMID 37508003 · DOI 10.3390/antiox12071465 -
Current Drug Repurposing Strategies for Rare Neurodegenerative Disorders.
Shah S, Dooms MM, Amaral-Garcia S, Igoillo-Esteve M. · · 2021 · cited 32× · PMID 34992533 · DOI 10.3389/fphar.2021.768023
Verify or expand the search:
- PubMed search for NCT01370447
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
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
- 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
Other recruiting trials for Mitochondrial Diseases
Currently open trials in the same condition.
- NCT07450690 — Exercise Training Effects on Muscle Function in Adults With Mitochondrial Myopathy · NA · recruiting
- NCT06573866 — Enhancement of Quality of Work And Life · NA · recruiting
- NCT06535646 — Digital Health Technology for People With Mitochondrial Disease · NA · recruiting
- NCT06504433 — The Natural History of Mitochondrial Diseases · recruiting
- NCT04322734 — Transgenerational Metabolic-Immune Biomarkers of Neurological and Neurodevelopmental Disorders · recruiting
Other PTC Therapeutics trials
Trials by the same sponsor.
- NCT06302348 — A Study of Sepiapterin in Participants With Phenylketonuria (PKU) · Phase 3 · recruiting
- NCT05515536 — A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia · Phase 3 · active not recruiting
- NCT05485987 — A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia · Phase 2 · completed
- NCT05218655 — A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease · Phase 3 · completed
- NCT05358717 — A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD) · 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 NCT01370447 (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 PTC Therapeutics
- Last refreshed: 23 January 2024
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/NCT01370447.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing