Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
Trial ID or NCT#
NCT01721733
Status
NOT 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: Yes
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
