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Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
Trial ID or NCT#
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
- - 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
- - 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)
Contact us to find out if this trial is right for you.
CONTACT
Gregory Enns, MD
(650) 498-5798
View on ClinicalTrials.gov
About this Clinical Trial
Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
Your Message Will Go To
Gregory Enns, MD650-498-5798