An Efficacy and Safety Trial of Intravenous Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids

Novartis

Status and phase: Completed; Phase 3

Conditions: Osteoporosis

Treatments: Drug: Placebo; Drug: Zoledronic acid

Study type: Interventional

Funder types: Industry

Identifiers

NCT00799266

CZOL446H2337

2008-001252-52 (EudraCT Number)

Details and patient eligibility

About

This study was designed to evaluate the efficacy and safety of zoledronic acid compared to placebo in osteoporotic children treated with glucocorticoids

Full description

In March 2017, Novartis stopped enrollment as the study was not feasible to be conducted due to low enrollment and other recruitment challenges. Patients receiving the treatment continued to receive the treatment per protocol.

Enrollment: 34 patients

Sex: All

Ages: 5 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• A diagnosis of chronic rheumatologic conditions or inflammatory bowel disease or Duchenne muscular dystrophy requiring systemic glucocorticoids (i.v. or oral) within 12 months prior to screening
• Lumbar Spine BMDZ-score of -0.5 or worse
• Evidence of at least at least 1 vertebral compression fracture of Genant Grade 1 or higher (or radiographic signs of vertebral fracture) within 1 month from Screening visit OR One or more, low-trauma, lower extremity long-bone fracture which occurred sometime within the 2 years PRECEDING enrollment in the study OR Two or more, low-trauma, upper extremity long-bone fractures which occurred sometime within the 2 years PRECEDING enrollment in the study
• Consent/assent to study participation

Key Exclusion Criteria:

• History of primary bone disease (OI, Idiopathic Juvenile Osteoporosis, Rickets/Osteomalacia)
• Any medical condition that might have interfered with the evaluation of lumbar spine BMD, such as severe scoliosis or spinal fusion. Patients with less than 3 evaluable vertebrae by Dual Energy X-ray Absorptiometry (DXA) evaluation in the region of interest lumbar 1 (L1) to lumbar 4 (L4),
• Hypocalcemia and hypophosphatemia
• Serum 25-hydroxy vitamin D concentrations of <20 ng/mL or <50 nmol/L
• estimated glomerular filtration rate (GFR) <60 mL/min/1.73 m2
• serum creatinine increase between Visit 1 and Visit 2 >0.5 mg/dL (44.2 μmol/L)
• Uncontrolled symptoms of cardiac failure or arrhythmia
• Any prior use of bisphosphonates, or high dose sodium fluoride

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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34 participants in 2 patient groups, including a placebo group

Zoledronic acid

Experimental group

Description:

Twice yearly 0.05 mg/kg (max 5 mg) i.v infusion (at least 30 minutes) of zoledronic acid

Treatment:

Drug: Zoledronic acid

Placebo

Placebo Comparator group

Description:

Twice yearly i.v of infusion of Placebo (similar dosing as active drug)

Treatment:

Drug: Placebo