RANDOMIZED, PLACEBO-CONTROLLED TRIAL OF ORAL CALCIUM SUPPLEMENTATION FOR OSTEO

口服钙补充剂治疗 OSTEO 的随机、安慰剂对照试验

• 批准号: 8356674
• 负责人: KATHLEEN J MOTIL
• 金额: $ 2.18万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-12-01 至 2011-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8356674
• 关键词: Alkaline Phosphatase; Bone Mineral Contents; Calcium; Clinical; Clinical Research; Deposition; Excretory function; Food; Fracture; Funding; Grant; Growth; Individual; Intervention; Longevity; Measurement; Measures; Minerals; Monitor; N-telopeptide; National Center for Research Resources; Oral; Osteocalcin; Osteopenia; Placebos; Prevalence; Principal Investigator; Randomized; Recording of previous events; Records; Research; Research Infrastructure; Resources; Rett Syndrome; Safety; Source; Supplementation; Testing; Treatment Efficacy; United States National Institutes of Health; Venous; Woman; adverse outcome; bone; calcium metabolism; cost; girls; muscle form; pill; prevent; randomized placebo controlled trial; urinary; wasting

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. Primary support for the subproject and the subproject's principal investigator may have been provided by other sources, including other NIH sources. The Total Cost listed for the subproject likely represents the estimated amount of Center infrastructure utilized by the subproject, not direct funding provided by the NCRR grant to the subproject or subproject staff. ABSTRACT Osteopenia and bone fractures complicate the clinical course of girls and women with Rett syndrome (RTT). Our studies show that total body bone mineral content (BMC) is below -1 SD and -2 SD in 82% and 57% of these individuals, respectively (1). The purpose of this study is to determine the efficacy of oral calcium (Ca) supplementation in the treatment of osteopenia in RTT girls and women. We hypothesize that oral Ca supplementation will reverse the progression of bone mineral loss in RTT girls and women, particularly in younger than older individuals, by reducing bone mineral resorption and increasing bone mineral deposition. To test this hypothesis, we will measure BMC using duel energy x-ray absorptiometry (DXA) in 54 RTT girls and women randomized to receive oral Ca supplements or placebo. Urinary Ca excretion and venous markers of Ca metabolism will be measured. Bone fracture prevalence will be documented by medcal history and ambulatory status will be scored by clinicalexamination. Stadiometric and anthropometric measurements will be performed to characterize growth and regional muscle mass. Three-day food records and interval pill count will be obtained to monitor compliance. We anticipate that BMC z-scores will increase and urinary Ca wasting will decrease with oral Ca supplementation thereby reversing the progression of bone mineral loss in RTT girls and women. This information will be important to ascertain because of the longevity of RTT girls and women and the need to provide interventional strategies that prevent the adverse consequences associated with osteopenia. HYPOTHESIS The purpose of this study is to determine the therapeutic efficacy and safety of oral Ca supplementation in the treatment of osteopenia inRTT girls and women. We hypothesize that oral Ca supplementation will reverse the progression of bone mineral loss, particularly in younger than in older individuals, by decreasing bone mineral resorption and increasing bone mineral deposition in RTT girls and women. SPECIFIC AIMS 1. To determine if oral Ca supplements result in a greater increase in absolute BMC and BMC z-scores, measured byDXA, than placebo inRTT girls and women 2. To determine if Ca supplements result in a greater increase in absolute BMC and BMC z-scores in pre-pubertal RTT girls than in post-pubertal RTT women 3. To determine if oral Ca supplements result in a greater reduction in bone mineral resorption, measured by N-telopeptides and urinary Cacreatinine ratios, than placebo in RTT girls and women 4. To determine if oral Ca supplements result in increased bone mineral deposition, measured by osteocalcin and bone alkaline phosphatase, than placebo in RTT girls and women.

该子项目是利用资源的众多研究子项目之一 由 NIH/NCRR 资助的中心拨款提供。子项目的主要支持 并且子项目的首席研究员可能是由其他来源提供的， 包括其他 NIH 来源。 子项目可能列出的总成本 代表子项目使用的中心基础设施的估计数量， NCRR 赠款不直接向子项目或子项目工作人员提供资金。 抽象的 骨质减少和骨折使患有 Rett 综合征 (RTT) 的女孩和妇女的临床病程变得复杂。 我们的研究表明，82% 和 57% 的个体的全身骨矿物质含量 (BMC) 分别低于 -1 SD 和 -2 SD (1)。 本研究的目的是确定口服钙 (Ca) 补充剂治疗 RTT 女孩和妇女骨质减少的疗效。 我们假设口服钙补充剂将通过减少骨矿物质吸收和增加骨矿物质沉积来逆转 RTT 女孩和女性的骨矿物质流失的进程，特别是对于年轻人而言。 为了检验这一假设，我们将使用双能 X 射线吸收测定法 (DXA) 测量 54 名随机接受口服钙补充剂或安慰剂的 RTT 女孩和妇女的 BMC。 将测量尿钙排泄和钙代谢的静脉标志物。 骨折发生率将通过病史记录，动态状态将通过临床检查进行评分。 将进行空间测量和人体测量来表征生长和区域肌肉质量。 将获得三天的食物记录和间隔药片计数以监测依从性。 我们预计，通过口服钙补充剂，BMC z 分数将会增加，尿钙消耗将会减少，从而逆转 RTT 女孩和女性骨矿物质流失的进程。 由于 RTT 女孩和妇女的寿命较长，并且需要提供干预策略来预防与骨质减少相关的不良后果，因此确定这一信息非常重要。 假设 本研究的目的是确定口服钙补充剂治疗 RTT 女孩和妇女骨质减少的疗效和安全性。 我们假设口服钙补充剂将通过减少 RTT 女孩和女性的骨矿物质吸收和增加骨矿物质沉积来逆转骨矿物质损失的进展，特别是在年轻人中比老年人更是如此。 具体目标 1. 确定口服钙补充剂是否会导致 RTT 女孩和女性的绝对 BMC 和 BMC z 分数（通过 DXA 测量）比安慰剂有更大的增加 2. 确定钙补充剂是否会导致青春期前 RTT 女孩的绝对 BMC 和 BMC z 分数比青春期后 RTT 女性更大 3. 确定口服钙补充剂是否会导致 RTT 女孩和女性的骨矿物质吸收（通过 N-端肽和尿肌肌酐比率测量）比安慰剂更大程度地减少 4. 通过骨钙素和骨碱性磷酸酶测量，确定口服钙补充剂是否会导致 RTT 女孩和女性骨矿物质沉积增加，而不是安慰剂。