Safety and Disease Monitoring Biomarkers in Duchenne Muscular Dystrophy: Results From A Phase Ii Trial
Kathryn R. Wagner, M. Guglieri, Shashi K. Ramaiah, Lawrence Charnas, Shannon Marraffino, Michael Binks, Vishal S. Vaidya, Jeffrey Palmer, Richard A. Goldstein, Francesco Muntoni
Abstract
Aim: Evaluate the utility of glutamate dehydrogenase (GLDH) and cardiac troponin I as safety biomarkers, and creatine kinase and muscle injury panel as muscle health biomarkers in Duchenne muscular dystrophy. Patients & methods: Data were collected during a Phase II trial of domagrozumab. Results: GLDH was a more specific biomarker for liver injury than alanine aminotransferase. Cardiac troponin I elevations were variable and not sustained, limiting its applicability as a biomarker. Muscle injury panel biomarkers were no more informative than creatine kinase as a muscle health biomarker. Conclusion: Results support the use of GLDH as a specific biomarker for liver injury in patients with Duchenne muscular dystrophy. Clinical trial registration: ClinicalTrials.gov, NCT02310763.