Clinical trial of zoledronic acid in children and adolescents with Duchenne muscular dystrophy

Phase 3

Completed

• Conditions: Duchenne muscular dystrophy; osteopaenia; Neurological - Other neurological disorders; Musculoskeletal - Osteoporosis; Human Genetics and Inherited Disorders - Other human genetics and inherited disorders

• Registration Number: ACTRN12610000507088
• Lead Sponsor: Royal Children's Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2010-06-18
• Last Update Posted: 13 January 2020

Recruitment & Eligibility

• Status: Completed
• Sex: Male
• Target Recruitment: 60

Inclusion Criteria

All boys between 6-16 years with confirmed Duchenne Muscular Dystrophy (DMD) and who are receiving glucocorticoid therapy (this is universally prednisolone)

Exclusion Criteria

Genant Grade 3 or greater vertebral compression
1. Any prior use of osteoporosis or bone-modifying therapy, such as bisphosphonates, sodium fluoride, calcitonin, calcitriol, Gonadotrophin releasing hormone agonists or Growth Hormone (GH).
2. Patients who have received testosterone therapy may only be included in the trial if this therapy was given as part of physiological replacement in the setting of documented hormonal deficiencies
3. Any prior history of malignancy
4. Any medical condition that might interfere with the evaluation of LS BMD, such as severe scoliosis or spinal fusion. Patients with less than 3 evaluable vertebrae by DEXA evaluation in the region of interest (ROI) L1-L4, as confirmed by the central imaging laboratory, will not be considered eligible for this study.
5. Hypocalcemia and hypophosphatemia: any value (age-matched) below the normal range at screening
6. Vitamin D deficiency (serum 25-hydroxy vitamin D concentrations of < 50 nmol/L) at screening
7. Renal impairment: Glomerulr filtration rate (GFR) < 35 ml/min/1.73 m2 at screening based on the Schwartz formula.
8. A serum creatinine increase between Visit 1 and Visit 2 greater than 44.2 mmol/L
9. History of hyperparathyroidism, hypothyriodism or hyperthyroidism within 1 year of screening
10.History of sarcoidosis, primary bone disease (osteogenesis imperfecta, idiopathic juvenile
osteoporosis, rickets/osteomalacia)., Kawasaki’s disease or Henoch-Schonlein Purpura.
11. Diagnosis of active uveitis (symptomatic or asymptomatic) at the time of enrollment of the study.
12. Any subject involved in another study, if an investigational agent is deemed by investigators to possible interfere with this study agent. ( for example use of a different bisphosphonate or a statin, a drug that utilizes the same biochemical pathways )

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change in lumbar spine (LS) areal bone mineral density (aBMD) Z-score as assessed by dual Xray absorptiometry (DEXA), with volumetric calculation for each subject to account for size variability [Bone mineral apparent density (BMAD)]. [0,12 and 24 months]