Safety and disease monitoring biomarkers in Duchenne muscular dystrophy: results from a Phase II trial

Biomark Med. 2021 Oct;15(15):1389-1396. doi: 10.2217/bmm-2021-0222. Epub 2021 Sep 17.

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.

Keywords: Duchenne muscular dystrophy; biomarker; cardiac troponin I; drug-induced liver injury; glutamate dehydrogenase.

Publication types

  • Clinical Trial, Phase II
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Alanine Transaminase / blood
  • Antibodies, Monoclonal, Humanized / administration & dosage
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Aspartate Aminotransferases / blood
  • Biomarkers / blood*
  • Child
  • Creatine Kinase / blood
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Drug Monitoring / methods*
  • Glutamate Dehydrogenase / blood
  • Humans
  • Male
  • Muscular Dystrophy, Duchenne / blood
  • Muscular Dystrophy, Duchenne / diagnosis
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Sensitivity and Specificity
  • Treatment Outcome
  • Troponin I / blood

Substances

  • Antibodies, Monoclonal, Humanized
  • Biomarkers
  • Troponin I
  • domagrozumab
  • Glutamate Dehydrogenase
  • Aspartate Aminotransferases
  • Alanine Transaminase
  • Creatine Kinase

Associated data

  • ClinicalTrials.gov/NCT02310763