Journal article
Randomized Controlled Trial Evaluating the Use of Zoledronic Acid in Duchenne Muscular Dystrophy
M Zacharin, A Lim, J Gryllakis, A Siafarikas, C Jefferies, J Briody, N Heather, J Pitkin, J Emmanuel, KJ Lee, X Wang, PJ Simm, CF Munns
Journal of Clinical Endocrinology and Metabolism | ENDOCRINE SOC | Published : 2021
Abstract
Context: Patients with glucocorticoid-dependent Duchenne muscular dystrophy (DMD) have increased fracture risk and reduced bone mineral density (BMD), often precipitating mobility loss. Objective: To investigate use of zoledronic acid (ZA) in DMD in improving BMD. Methods: Two arm, parallel, randomized controlled trial, set in pediatric hospitals across Australia and New Zealand. Sixty-Two (31 per arm) boys with glucocorticoid-dependent DMD between 6 and 16 years were included. Five ZA infusions (0.025 mg/kg at months 0, and 3, and 0.05 mg/kg at months 6, 12, and 18), plus calcium and vitamin D, were compared with calcium and vitamin D alone. The main outcome measures were change in lumbar s..
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Funding Acknowledgements
This study has received funding support from Novartis for provision of zoledronic acid and a grant of $50 000 to set up the study. Adverse effects if any were reported to Novartis, but the company had no other input into the study. This trial was also funded by various philanthropic groups; namely an Altum grant from the Muscular Dystrophy Association, and project grants from Thrasher Foundation and Foundation for Children. The Australasian Paediatric Endocrine Group also provided funding for the study with 2 investigator awards that contributed to the study.