A Multicenter, Randomized, Double-blind, Placebo Controlled Efficacy and Safety Trial of Intravenous Zoledronic Acid Twice Yearly Compared to Placebo in Osteoporotic Children Treated With Glucocorticoids.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 34
- 试验地点
- 1
- 主要终点
- Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 12
研究概览
简要总结
This study was designed to evaluate the efficacy and safety of zoledronic acid compared to placebo in osteoporotic children treated with glucocorticoids
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Zoledronic acid
Twice yearly 0.05 mg/kg (max 5 mg) i.v infusion (at least 30 minutes) of zoledronic acid
干预措施: Zoledronic acid (Drug)
Placebo
Twice yearly i.v of infusion of Placebo (similar dosing as active drug)
干预措施: Placebo (Drug)
结局指标
主要结局
Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 12
时间窗: Month 12
Lumbar Spine Bone Mineral Density (BMD) Z-score was determined by the central imaging vendor before first treatment and at Month 12. The methods to be used to measure Lumbar Spine BMD Z-score were described in the respective DXA Manuals provided by central imaging vendor. Positive changes from baseline indicated an improvement in condition.
次要结局
- Mean Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score at Month 6(Month 6)
- Mean Change From Baseline in Lumbar Spine BMC at Month 6 and 12(Month 6, Month 12)
- Mean Change From Baseline in Total Body BMC at Month 6 and 12(Month 6, Month 12)
- Mean Change From Baseline in Serum P1NP at Months 6 and 12(Month 6, Month 12)
- Mean Change From Baseline in BSAP at Months 6 and 12(Month 6, Month 12)
- Mean Change From Baseline in Serum NTX at Months 6 and 12(Month 6, Month 12)
- Mean Change From Baseline in Serum TRAP-5b at Months 6 and 12(Month 6, Month 12)
- Number of Participants With New Vertebral Fractures at Month 12(Month 12)
- Mean Change From Baseline in Vertebral Morphometry at Month 12(Month 12)
- Percentage of Patients With Reduction in Pain at Months 3, 6, 9 and 12(Month 3, Month 6, Month 9 and Month 12)
- Mean Change From Baseline in 2nd Metacarpal Cortical Width at Month 12(Month 12)
- Urinary Concentration of Zoledronic Acid at Month 12(Month 12)
- Safety of Zoledronic Acid for the Treatment of Osteoporotic Children Treated With Glucocorticoids(Baseline through Month 12)