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

• Status: Active, not recruiting
• Trial end date: 2021-12-31
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Edison Pharmaceuticals Inc; PTC Therapeutics
• Treatments: Ubiquinone

Criteria

Inclusion Criteria:

1. Diagnosis of Leigh syndrome with genetic confirmation

2. Completion of EPI743-12-002 protocol and initiation of treatment within 14 days of Month 12 visit in EPI743-12-002 study

3. Participant or participant's guardian able to consent and comply with protocol requirements

4. Continued abstention from supplements excluded in EPI743-12-002 study

5. Botox® is allowed if approved by the sponsor

Exclusion Criteria:

1. Allergy to EPI-743 or sesame oil

2. Allergy to vitamin E

3. Clinical history of bleeding or abnormal baseline prothrombin time (PT)/partial thromboplastin time (PTT)

4. Hepatic insufficiency with liver function tests (LFTs) greater than two times upper limit of normal

5. Renal insufficiency requiring dialysis

6. End-stage cardiac failure

7. Fat malabsorption syndromes precluding drug absorption

8. Use of anticoagulant medications

9. Participation in other clinical research studies/taking other experimental agents

10. Participation in elective procedures that required sedation