Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents
Status:
Completed
Trial end date:
2018-07-01
Target enrollment:
Participant gender:
Summary
This single center open-label pilot study will enroll 15 non-ambulatory children with
Duchenne muscular dystrophy at least 8 years of age and who demonstrate pre-clinical
cardiomyopathy (defined as a cardiac ejection fraction >55% with abnormal LV strain by
cardiac MRI). They will receive (+)-epicatechin at one of three doses during an 8-week
dose-ranging study with assessments at baseline, 2 Weeks, 4weeks, and 8 weeks. The study will
determine optimal dosing for future cardiac efficacy studies based on serum / plasma
biomarker response using follistatin: myostatin ratio, nitrite/nitrate ratio, cardiac
troponins and cardiac BNP. Secondary endpoints will include additional biomarker assessments
by SOMAscanTM, cardiac functional evaluations by cardiac MRI (LV strain), and echocardiogram
(LV strain by speckle tracking) and measures of strength, range of motion and mobility, and
clinical safety assessments. Results of secondary endpoint analysis will be used to refine
design of subsequent clinical trials powered to detect changes in clinical outcomes.