OSTEOGENESIS IMPERFECTA/IV ZOLEDRONIC ACID VS IV PAMIDRONATE

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 这项研究比较了两种药物治疗严重成骨不全儿童的安全性和有效性。年龄在1至17岁之间的儿童，如有III型和IV型成骨，或I型成骨，并在过去两年内有3次或更多微创骨折史，或有肢体畸形史而需要手术，则符合条件。其中一种药物帕米磷酸钠已经被证明可以减少成骨不全时的骨折。阿来磷酸钠是一种与帕米磷酸钠相同类型的新药，但它更有效，如果有效和安全，它可能是一种很好的替代品。这项研究是第一次将唑来膦用于成骨不全的儿童。这项研究为期13个月，包括10-15次访问。在这一年中，儿童每隔2个月或3个月接受一种研究药物的治疗。在整个研究过程中，都要进行体检、血液和尿液检查、X光检查和骨密度测量。除了研究药物，儿童还服用多种维生素和钙补充剂。这是一项国际性、多中心的研究，136名儿童将在整个研究范围内接受治疗。一半的儿童将接受帕米磷酸钠的治疗，一半的儿童将接受唑来膦酸盐的治疗。一些接受唑来膦酸盐治疗的儿童将需要额外的实验室工作来研究这种药物在儿童体内的药代动力学(人体过程)。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This study compares the safety and effectiveness of two medications in children with severe osteogenesis imperfecta. Children between 1 and 17 years of age who have types III and IV osteogenesis, or who have type I osteogenesis with a history of 3 or more minimal trauma fractures in the past two years or a history of limb deformity requiring surgery are eligible. One of the medications, pamidronate, has already been shown to reduce fractures in osteogenesis imperfecta. Aledronate is a new drug of the same type as pamidronate, but it is more powerful and may be a good alternative if it is effective and safe. This study is the first time zoledronate is being used in children with osteogenesis imperfecta. This study lasts for 13 months and includes 10-15 visits. Children are treated with one of the study drugs at either 2 month or 3 month intervals during that year. Physical exams, blood and urine studies, x-rays and bone density measurements are made throughout the study. In addition to the study medication, children take multivitamins and calcium supplements. This is an international, multi-center study, and 136 children will be treated, study-wide. Half of the children will receive treatment with pamidronate and half will receive treatment with zoledronate. Some children who receive zoledronate will have additional lab work to study the pharmacokinetics (processing by the body) of this drug in children.