Phase 3 N=34 Randomized Quadruple-blind Treatment

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

Osteoporosis

Bottom Line

View on ClinicalTrials.gov: NCT00799266 ↗

Enrolled (actual)

Serious AEs

17.7%

Results posted

Jul 2019

Primary outcome: Primary: Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 12 — 0.582; 0.168 Z-score — p=0.0392

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Zoledronic acid (Drug); Placebo (Drug)
Age: Pediatric · 5+ yrs
Sex: All
Sponsor: Novartis Pharmaceuticals
Primary completion: Mar 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 12	0.582; 0.168	0.0392 sig
SECONDARY Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 6	0.447; 0.157	0.1322
SECONDARY Mean Change From Baseline in Lumbar Spine BMC at Month 6 and 12	4.110; 2.131; 6.450; 4.295	0.0409 sig
SECONDARY Mean Change From Baseline in Total Body BMC at Month 6 and 12	129.272; 95.214; 220.805; 140.064	0.3827
SECONDARY Mean Change From Baseline in Serum P1NP at Months 6 and 12	-134.285; 77.497; -230.966; 150.166	0.0631
SECONDARY Mean Change From Baseline in BSAP at Months 6 and 12	-7.413; 3.810; -13.984; 6.450	0.2129
SECONDARY Mean Change From Baseline in Serum NTX at Months 6 and 12	-13.746; 7.192; -20.134; 7.440	0.0254 sig
SECONDARY Mean Change From Baseline in Serum TRAP-5b at Months 6 and 12	-1.561; 0.313; -1.728; 0.109	0.2178
SECONDARY Number of Participants With New Vertebral Fractures at Month 12	0; 2	0.2258
SECONDARY Mean Change From Baseline in Vertebral Morphometry at Month 12	-0.018; -0.0003	0.3180
SECONDARY Percentage of Patients With Reduction in Pain at Months 3, 6, 9 and 12	37.5; 53.8; 37.5; 50.0; 33.3; 46.2	0.5226
SECONDARY Mean Change From Baseline in 2nd Metacarpal Cortical Width at Month 12	-0.01; 0.03	0.5165
SECONDARY Urinary Concentration of Zoledronic Acid at Month 12	1643.3	—
SECONDARY Safety of Zoledronic Acid for the Treatment of Osteoporotic Children Treated With Glucocorticoids	83.3; 75.0; 27.8; 6.3	—

Summary

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

Eligibility 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 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

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Data sourced from ClinicalTrials.gov (NCT00799266). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.