Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

Completed · Phase 2

Conditions studied: Leigh Syndrome

In brief

EPI-743 in Leigh syndrome participants that participated in previous EPI743-12-002 (NCT01721733) study.

Key facts

Study ID

NCT02352896

Run by

PTC Therapeutics

People needed

30

Starts

2014-01-31

Expected to finish

2023-10-30

Last updated by the study team

2024-10-23

Who can join

Age: 1 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of Leigh syndrome with genetic confirmation
• Completion of EPI743-12-002 protocol and initiation of treatment within 14 days of Month 12 visit in EPI743-12-002 study
• Participant or participant's guardian able to consent and comply with protocol requirements
• Continued abstention from supplements excluded in EPI743-12-002 study
• Botox® is allowed if approved by the sponsor

You may not qualify if…

• Allergy to EPI-743 or sesame oil
• Allergy to vitamin E
• Clinical history of bleeding or abnormal baseline prothrombin time (PT)/partial thromboplastin time (PTT)
• Hepatic insufficiency with liver function tests (LFTs) greater than two times upper limit of normal
• Renal insufficiency requiring dialysis
• End-stage cardiac failure
• Fat malabsorption syndromes precluding drug absorption
• Use of anticoagulant medications
• Participation in other clinical research studies/taking other experimental agents
• Participation in elective procedures that required sedation

Where it is running

• Gregory Enns — Stanford University, California, United States
• Akron Children's Hospital — Akron, Ohio, United States
• Baylor College of Medicine — Houston, Texas, United States
• Seattle Children's Hospital — Seattle, Washington, United States

Full record on ClinicalTrials.gov

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