Treatment of Osteopenia in Children with Cerebral Palsy

脑瘫儿童骨质减少的治疗

• 批准号: 6575036
• 负责人: RICHARD C HENDERSON
• 金额: $ 14.55万
• 依托单位: UNIV OF NORTH CAROLINA CHAPEL HILL
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-09-25 至 2004-09-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6575036
• 关键词: bone density; cerebral palsy; children; clinical research; clinical trials; data management; diphosphonate; dosage; drug screening /evaluation; experimental designs; human data; human subject; osteopenia; patient oriented research; photon absorptiometry

DESCRIPTION (provided by applicant): Osteopenia resulting in fractures with minimal trauma is a common problem in many pediatric conditions such as osteogenesis imperfecta, juvenile idiopathic osteoporosis, muscular dystrophy, and myelodysplasia (spina bifida). As part of the North American Growth in Cerebral Palsy Project (NAGCePP) we have extensively studied one such group of children, those with cerebral palsy (CP), to better define the prevalence, causes, and consequences of osteopenia in this condition. Bisphosphonates are a group of medications utilized to treat osteoporosis in elderly adults. There are published reports of these agents used in assorted pediatric conditions, but with rare exception these are anecdotal, uncontrolled case reports involving at most a few children. We have recently completed a small placebo-controlled Pilot Trial to assess the safety and efficacy of intravenous bisphosphonates to treat low bone density in children with severe CP. At the conclusion of the 18-month study period bone density in the distal femur had increased 89% + 21% (mean + SE) compared to 9% + 6% in controls. No clinically significant adverse effects were identified. The Pilot Trial raises many questions with regards to dosing (frequency, duration, amount, route of administration), indications for treatment, long-term risks and benefits including the effect on fracture rate. The results of the Pilot Trial provide the justification for a larger scale Future Clinical Trial to answer these many questions. The purpose of this application is to support the Clinical Trial Planning Project, a process critical to the successful implementation and completion of the Future Clinical Trial. The Clinical Trial Planning Project will focus on building from both the Pilot Trial and the existing NAGCeP collaboration in several areas. Subjects: Develop additional recruiting strategies for larger numbers of subjects and further refine criteria for inclusion based on analysis of fracture risk data. Intervention: Select alternative drugs and dosing regimens that are more practical. Outcomes: Further evaluate and adapt DXA technology to this population with contractures, scoliosis, metallic implants, involuntary muscle spasms, retardation, and other characteristics that present unique challenges. Collaborative Resources & Infrastructure: Adapt and expand our existing NAGCePP network (eg database, safety monitoring) to incorporate the Future Clinical Trial.

描述（由申请人提供）： 在许多小儿疾病（例如骨生成不完美，少年特发性骨质疏松症，肌营养不良症和骨髓增生性肿瘤）（脊髓增生症（Spina bifida））中，导致最小创伤的骨质减少症是一个常见的问题。作为脑瘫项目（NAGCEPP）北美增长的一部分，我们已经广泛研究了一群这样的儿童，即患有脑瘫（CP）的儿童，以更好地定义在这种情况下骨质骨的普遍性，原因和后果。 双膦酸盐是一组用于治疗老年人骨质疏松症的药物。有关于各种儿科疾病中使用的这些药物的报告，但是除了极少数例外，这些都是轶事，不受控制的病例报告，其中最多几个儿童涉及。我们最近完成了一项小型安慰剂对照试验，以评估双膦酸酯静脉注射双膦酸盐治疗严重CP儿童低骨密度的安全性和功效。在股骨远端的18个月研究期结束时，骨密度增加了89％ + 21％（平均 + SE），而对照组的骨密度增加了9％ + 6％。未发现临床上显着的不良反应。 试点试验提出了许多关于给药的问题（频率，持续时间，数量，给药途径），治疗适应症，长期风险和收益，包括对断裂率的影响。 试点试验的结果为大规模的未来临床试验提供了理由，以回答这些许多问题。本申请的目的是支持临床试验计划项目，这对于成功实施和完成未来临床试验至关重要。 临床试验计划项目将着重于在多个领域的试验试验和现有的NAGCEP合作中建立。受试者：根据分析断裂风险数据的分析，为大量受试者制定额外的招聘策略，并进一步完善纳入标准。干预：选择更实用的替代药物和剂量方案。结果：进一步评估和调整DXA技术，以缔约方，脊柱侧弯，金属植入物，非自愿肌肉痉挛，延迟和其他特征，以提出独特的挑战。 协作资源与基础架构：适应和扩展我们现有的Nagcepp网络（例如数据库，安全监控），以纳入未来的临床试验。