Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 2:41 AM
Ignite Modification Date:
2025-12-25 @ 2:41 AM
NCT ID:
NCT01721733
Eligibility Criteria:
Inclusion Criteria:
* Clinical and MRI diagnosis of Leigh syndrome
* Moderate disease severity based on NPMDS score
* Age under 18 years
* Documented evidence of disease progression within 12 month of enrollment
* Availability of MRI that confirms necrotizing encephalopathy
* Patient or guardian able to consent and comply with protocol requirements
* Abstention from Coenzyme Q10, Vitamins C \& E, lipoic acid and Idebenone
Exclusion Criteria:
* Allergy to EPI-743, Vitamin E or sesame oil
* History of bleeding abnormalities or abnormal PT/PTT
* Diagnosis of concurrent inborn error of metabolism
* Previous tracheostomy
* Ventilator dependent or use of noninvasive ventilatory support w/in 1 month of enrollment
* LFTs greater than 2 times ULN
* Renal insufficiency
* End stage cardiac failure
* Fat malabsorption syndrome
* Use of anticoagulant medications
* Abstention from Botox for 6 months prior to enrollment and for duration of study
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
6 Years
Maximum Age:
17 Years
Study:
NCT01721733
Study Brief:
Protocol Section:
NCT01721733