NCT01728064 is a Phase 2 clinical trial of EPI-743 400 mg in Friedreich's Ataxia, sponsored by PTC Therapeutics.

Who is sponsoring NCT01728064?

NCT01728064 is sponsored by PTC Therapeutics.

What drugs are being tested in NCT01728064?

Interventions in NCT01728064: Placebo, EPI-743 400 mg, EPI-743 200 mg.

What condition does NCT01728064 study?

NCT01728064 studies Friedreich's Ataxia.

Is NCT01728064 still recruiting?

NCT01728064 is currently completed. Last updated 31 August 2020.

Last reviewed 2020-08-31 · How we verify

NCT01728064

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

Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

Completed Phase 2 Last updated 31 August 2020

What this trial tests

Phase 2 trial testing Placebo in Friedreich's Ataxia in 60 participants. Completed in 29 February 2016.

Timeline

31 December 2012

Primary endpoint
31 October 2015

29 February 2016

Quick facts

Lead sponsor	PTC Therapeutics
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	60
Start date	31 December 2012
Primary completion	31 October 2015
Estimated completion	29 February 2016
Sites	3 locations across United States

Drugs / interventions tested

Placebo
EPI-743 400 mg — full drug profile →
EPI-743 200 mg — full drug profile →

Conditions studied

Friedreich's Ataxia — all drugs for Friedreich's Ataxia →

Sponsor

PTC Therapeutics — full company profile →

Who can join

Adults 18 to 45, any sex, with Friedreich's Ataxia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this study is to examine the effects of EPI-743 on visual function and neurologic function in patients with Friedreich's ataxia.

Publications & conference data

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

Iron homeostasis and ferroptosis in human diseases: mechanisms and therapeutic prospects.
Ru Q, Li Y, Chen L, Wu Y, et al · · 2024 · cited 365× · PMID 39396974 · DOI 10.1038/s41392-024-01969-z
Emerging therapies for mitochondrial diseases.
Hirano M, Emmanuele V, Quinzii CM. · · 2018 · cited 122× · PMID 29980632 · DOI 10.1042/ebc20170114
Mitochondrial diseases: from molecular mechanisms to therapeutic advances.
Wen H, Deng H, Li B, Chen J, et al · · 2025 · cited 111× · PMID 39788934 · DOI 10.1038/s41392-024-02044-3
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
Glutathione as a Redox Biomarker in Mitochondrial Disease-Implications for Therapy.
Enns GM, Cowan TM. · · 2017 · cited 50× · PMID 28467362 · DOI 10.3390/jcm6050050
Test-retest reliability of the Friedreich's ataxia rating scale.
Rummey C, Zesiewicz TA, Perez-Lloret S, Farmer JM, et al · · 2020 · cited 14× · PMID 32779859 · DOI 10.1002/acn3.51118
Exploring the anti-inflammatory activities, mechanism of action and prospective drug delivery systems of tocotrienol to target neurodegenerative diseases.
Mathew AM, Bhuvanendran S, Nair RS, K Radhakrishnan A. · · 2023 · cited 4× · PMID 39291146 · DOI 10.12688/f1000research.131863.1
Novel Therapeutic Approaches in Inherited Neuropathies: A Systematic Review.
Hustinx M, Shorrocks AM, Servais L. · · 2023 · cited 4× · PMID 37376074 · DOI 10.3390/pharmaceutics15061626

Verify or expand the search:

Other recruiting trials for Friedreich's Ataxia

Currently open trials in the same condition.

NCT07095062 — Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome. · NA · 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 NCT01728064 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 9 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: 31 August 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/NCT01728064.

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