An International, Multicenter, Open-label, Efficacy and Safety Trial of Intravenous Zoledronic Acid in Infants Less Than One Year of Age, With Severe Osteogenesis Imperfecta
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- The primary objective of this trial is to assess the change in lumbar spine bone mineral density Z-score at month 24 relative to baseline in zoledronic acid treated infants compared to historical controls.
研究概览
简要总结
The investigators have currently finished conducting an international multi-center trial that compares the efficacy and safety of pamidronate and zoledronate in the treatment of moderate to severe forms of Osteogenesis Imperfecta (OI). This trial has included only children above one year of age. The aim of the current study is to extend the observations of that currently finished study to infants below 1 year of age. Moreover, it is possible to administer zoledronate in a single short infusion instead of the three-day cycles with Pamidronate, therefore decreasing patient and family burdens with shorter stays in the hospital.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- — 至 12 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children, male or female 2 weeks to < 12 months of age, at least at 38 weeks gestational age.
- •Any child with phenotypic OI type II, III or IV.
- •Any child classified as OI type I, V-VIII that has had at least two or more previous fractures of long bone, or vertebral compression fractures, and a low bone mineral density.
- •No previous treatment with bisphosphonates.
- •Negative urine protein as measured by dipstick. One repeat assessment of the urine protein will be allowed.
排除标准
- •Blood oxygen saturation of less than 90% in room air.
- •Serum creatinine level greater than 56 µmol/L.
- •Any clinically significant clinical laboratory abnormalities at screening.
- •Treatment with any investigational drug within the past 30 days.
- •Patients who are unlikely to be able to complete the study or comply with the visit schedule.
- •Any disease or planned therapy which will interfere with the procedures or data collection of this trial.
研究组 & 干预措施
Treatment Arm (only)
Zoledronic acid infusion
干预措施: Zoledronic Acid (Drug)
结局指标
主要结局
The primary objective of this trial is to assess the change in lumbar spine bone mineral density Z-score at month 24 relative to baseline in zoledronic acid treated infants compared to historical controls.
时间窗: 3 times during 10 visits within 2 years
次要结局
- To assess the effect of zoledronic acid on the number of clinical fractures &/or vertebral compressions that occur over a two year period compared to untreated historical controls in infants.(3 times during 10 visits within 2 years)
研究者
Francis H. Glorieux
Principal Investigator
Shriners Hospitals for Children