Project Details

E.W. "Al" Thrasher

Status: Funded - Open

Clinical trial of Zoledronic acid (Aclasta) in children and adolescents with Duchenne muscular dystrophy

Margaret Zacharin, MBBS, FRACP


BACKGROUND: Duchenne Muscular Dystrophy (DMD) affects 1 in 3,500 boys worldwide. Treatment usually includes use of long term corticosteroids which help reduce inflammation but cause side effects of severe and unremitting bone loss.  Fractures of vertebrae as well as long bones are reported in between 40-70% of DMD patients with 20-50% of affected boys losing their ability to walk after a fracture.

GAP:  Bisphosphonates are a class of drug taken up into bone where they reduce corticosteroid induced bone loss. This benefit has been shown in studies of children, adolescents and adults with bone fragility conditions. In boys with DMD data are lacking concerning beneficial effects of bisphosphonates. This is the first randomized controlled trial of this type of intervention in the world, for boys who have DMD.

HYPOTHESIS:  It is hypothesized that Zoledronic acid doses administered intravenously at 3-6 monthly intervals in combination with calcium and vitamin D will demonstrate superior effect, relative to vitamin D and calcium therapy alone for improvement in lumbar spine bone mineral density, reduced pain and fracture incidence and improved quality of life in boys with DMD.

METHODS: This is an open label 2 year trial of intravenous Zoledronic acid plus calcium and vitamin D, comparing the effect of the drug with use of calcium and vitamin D alone. It aims to show that Zoledronic acid can increase bone mineral density, improve pain and reduce fracture rate after 24 months of treatment and is superior to standard treatment. Boys between 6 to 16 years with DMD  treated with  corticosteroid therapy will be recruited and  randomised to either Group A (Zoledronic Acid, calcium and vitamin D) or Group B (calcium and vitamin D alone).

RESULTS: Pending

IMPACT: The results of this trial, if positive,  would have far reaching consequences in terms of potential reduction in morbidity, hospitalization and immobilization of affected boys and would inform practice worldwide, providing an improved standard clinical care for these patients. If zoledronic acid becomes standard preventive care for boys with DMD, it will lead to improved patient quality of life and improve family burden of care for boys with DMD and their families.

Drug Therapy, Bone, Human, Randomized Clinical Trial

Supervising Institution:
Murdoch Childrens Research Institute

Project Location:
Victoria, New South Wales

Award Amount: