Open AccessSafety and disease monitoring biomarkers in Duchenne muscular dystrophy: results from a Phase II trial
Author(s) -
Kathryn R. Wagner,
M. Guglieri,
Shashi K. Ramaiah,
Lawrence Charnas,
Shan Marraffino,
Michael Binks,
Vishal S. Vaidya,
Jeffrey Palmer,
Richard A. Goldstein,
Francesco Muntoni
Publication year - 2021
Publication title -
biomarkers in medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.652
H-Index - 44
eISSN - 1752-0371
pISSN - 1752-0363
DOI - 10.2217/bmm-2021-0222
Subject(s) - medicine , duchenne muscular dystrophy , biomarker , creatine kinase , muscular dystrophy , troponin , troponin i , creatine , pathology , myocardial infarction , biochemistry , biology
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 .