Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
Trial ID or NCT#
NCT01721733
Status
RECRUITING
Purpose
The purpose of this study is to evaluate the effects of EPI-743 in children with Leigh syndrome on disease severity, neuromuscular function, respiratory function, disease morbidity and mortality and disease associated biomarkers.
Official Title
A Phase 2B Randomized, Placebo Controlled, Double Blind Clinical Trial of EPI-743 in Children With Leigh Syndrome
Eligibility Criteria
Ages Eligible for Study: 6 Years to 17 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
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
Investigator(s)
Gregory Enns
Contact us to find out if this trial is right for you.
Contact
Gregory Enns, MD
650-498-5798
View on ClinicalTrials.gov
