Bisphosphonate Therapy for Osteogenesis Imperfecta
研究概览
详细说明
The study is designed to evaluate the efficacy and safety of "Bisphosphonate Therapy for Osteogenesis Imperfecta (OI)." OI is an inherited disorder of collagen synthesis. Collagen is the major structural protein of the matrix of tendons, skin, and bones. Affected persons have low bone mineral density (and experience multiple fractures and progressive bony deformity). In its most severe form, the disorder is lethal in infancy. We plan to characterize the changes effected by oral bisphosphonate therapy and compare them to a regimen of intravenous bisphosphonate therapy in a group of children with OI.
Additionally, we have begun to treat patients with OI and other conditions of low bone mineralization for age who are not eligible for the standard protocol (too young, history of abdominal pain, etc.) with bisphosphonate. We also plan to screen the parents and siblings of our patients diagnosed with osteogenesis imperfecta, in order to determine if they also have osteoporosis.
研究类型
注册 (实际的)
阶段
- 第四阶段
参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:
Diagnosis of OI, as defined by genetic analysis revealing a defect of type I collagen, OR by bone mineral density (BMD) <2.5 standard deviations (SD) for age plus two of the following:
- Family history of OI
- Frequent fractures
- Blue sclerae
- Multiple wormian bones on skull x-ray
- Hearing disturbance
- Dentinogenesis imperfecta
- Age between 3 and 21 years at the start of the study period.
- Children must be able to swallow whole tablets
- Parents of children must be able to understand protocol and give informed consent.
Exclusion Criteria:
- Therapy with bisphosphonates during the past 12 months.
- Other "non-traditional" therapy for OI in the last 6 months, such as growth hormone or anabolic steroids.
- Other chronic diseases besides OI that interfere with bone morphology or gastrointestinal absorption
学习计划
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：随机化
- 介入模型：并行分配
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
有源比较器：Alendronate
1 mg/kg po qd rounded to nearest 10 or 20 mg dose
|
其他名称：
|
|
有源比较器：Pamidronate
3 mg/kg IV q4 months
|
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Bone Mineral Density
大体时间：2 years
|
By Dual-energy x-ray absorptiometry.
Results were reported as z-scores as well as as absolute values.
The Z-score indicates the number of standard deviations away from the mean.
A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher.
Higher Z scores indicate a better outcome", or similar, as accurate and appropriate.
|
2 years
|
合作者和调查者
调查人员
- 首席研究员：Linda A DiMeglio, MD, MPH、Indiana University School of Medicine
研究记录日期
研究主要日期
学习开始
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (估计)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.