OSTEOGENESIS IMPERFECTA/IV ZOLEDRONIC ACID VS IV PAMIDRONATE

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 本研究比较了两种药物治疗儿童严重成骨障碍的安全性和有效性。 年龄在1 - 17岁之间的III型和IV型成骨儿童，或在过去两年中有3次或3次以上轻微创伤骨折史的I型成骨儿童，或有需要手术的肢体畸形史的儿童都有资格参加。 其中一种药物，帕米膦酸盐，已经被证明可以减少骨质疏松症的骨折。 阿来膦酸盐是一种与帕米膦酸盐相同类型的新药，但它更强大，如果有效和安全，可能是一个很好的替代品。 本研究是唑来膦酸首次用于儿童成骨不全。 该研究持续13个月，包括10-15次访视。 在该年内，儿童每隔2个月或3个月接受一种研究药物治疗。 在整个研究期间进行体格检查、血液和尿液研究、X射线和骨密度测量。 除研究药物外，儿童还服用多种维生素和钙补充剂。 这是一项国际多中心研究，136名儿童将接受治疗。 一半的儿童将接受帕米膦酸盐治疗，另一半将接受唑来膦酸盐治疗。 一些接受唑来膦酸的儿童将有额外的实验室工作来研究这种药物在儿童中的药代动力学（身体处理）。

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.