← Back to Search

Antioxidant

EPI-743 for Leigh Syndrome

Phase 2
Waitlist Available
Led By Bruce Cohen, MD
Research Sponsored by PTC Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to month 36
Awards & highlights

Study Summary

This trial is testing the safety and effectiveness of a drug called EPI-743 in people with Leigh syndrome.

Eligible Conditions
  • Leigh Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to month 36
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to month 36 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Disease Severity as Measured by Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) Sections 1-3 Score
Number of Participants With Dose-Limiting Serious Adverse Events (SAEs)
Secondary outcome measures
Awake Oxygen Saturation Levels
Barry-Albright Dystonia Scale Score
Bayley Scales of Infant Development-III Score (Participants Age 0-3)
+7 more

Side effects data

From 2019 Phase 1 & 2 trial • 20 Patients • NCT01642056
50%
Blood creatine phosphokinase increased
20%
Vomiting
20%
Alanine aminotransferase increased
20%
Aspartate aminotransferase increased
15%
Activated partial thromboplastin time prolonged
15%
Hypothyroidism
10%
Hospitalisation
10%
Blood alkaline phosphatase increased
10%
Hypernatraemia
10%
Cough
10%
Upper respiratory tract irritation
10%
Polycythaemia
10%
Hypoglycaemia
5%
Prothrombin time prolonged
5%
Platelet count decreased
5%
Goitre
5%
Hyperammonaemia
5%
Amylase increased
5%
Red blood cell sedimentation rate increased
5%
Hyperbilirubinaemia
5%
Proteinuria
5%
Rash
5%
Status epilepticus
5%
Adrenal insufficiency
5%
Gastroenteritis viral
5%
Hyperthyroidism
5%
Neutrophil count decreased
5%
Nasal congestion
5%
Electrocardiogram QT prolonged
5%
International normalised ratio increased
5%
Hyperkalaemia
5%
Hypercholesterolaemia
5%
Lipase increased
5%
White blood cell count decreased
5%
Wheezing
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
EPI-743

Trial Design

1Treatment groups
Experimental Treatment
Group I: EPI-743Experimental Treatment1 Intervention
Participants will receive EPI-743 at a dose of 15 milligrams/kilogram (mg/kg) up to a total 200 mg 3 times daily (TID) orally with meal or by using nasogastric (NG) or gastrostomy feeding tubes with liquid food for at least 36 months.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
EPI-743
2014
Completed Phase 2
~170

Find a Location

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor
74 Previous Clinical Trials
6,200 Total Patients Enrolled
2 Trials studying Leigh Syndrome
95 Patients Enrolled for Leigh Syndrome
Bruce Cohen, MDPrincipal InvestigatorAkron Children's Hospital
2 Previous Clinical Trials
36 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many participants can the clinical trial accommodate?

"At this current juncture, no more participants are being recruited for this trial. It was initially posted on January 31st 2014 and last modified November 17th 2022. If you're searching for other studies related to Leigh Syndrome, 1382 clinical trials are actively enrolling patients; there are 4 trials concerning EPI-743 that welcome new enrollees as well."

Answered by AI

Are there any precedent studies that have utilized EPI-743 in their research?

"EPI-743 was first tested in 2010 at CHOC Children's Clinic and has since seen 9 studies come to fruition. Currently, 4 investigations into the drug are running with a concentration of sites located in Akron, Ohio."

Answered by AI

Is enrollment for this research effort currently available?

"According to clinicaltrials.gov, the recruitment phase for this trial closed on November 17th 2022; however, there are still 1,386 similar studies actively recruiting participants at this time."

Answered by AI

Is eligibility for this investigation open to me?

"This experiment is targeting 30 children between one year and eighteen suffering from Leigh Syndrome. To be considered for the trial, applicants must meet these criteria."

Answered by AI

Is this research break-through and unprecedented?

"Since 2010, PTC Therapeutics has sponsored studies on EPI-743. The initial research was conducted with 94 participants and led to the drug receiving Phase 2 approval. At present, 4 trials are underway in 27 cities across 13 countries."

Answered by AI

Could you outline the safety profile of EPI-743 for those under its care?

"Although EPI-743 has not yet been clinically validated, there is enough evidence to suggest its safety merits a score of 2 out of 3."

Answered by AI

Are individuals aged 30+ eligible to join the experiment?

"This medical trial is seeking participants aged between one year and eighteen years."

Answered by AI

To what extent is this trial accessible through healthcare facilities in the region?

"Currently, this trial is accepting patients from 4 medical centres situated in Akron, Stanford University and Houston among other sites. To limit travel time during the study's duration, it is recommended to choose a facility close to your location."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
2014. "Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02352896.
~3 spots leftby Apr 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top