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; 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.

该副本是利用资源的众多研究子项目之一 由NIH/NCRR资助的中心赠款提供。对该子弹的主要支持 而且，副投影的主要研究员可能是其他来源提供的 包括其他NIH来源。 列出的总费用可能 代表subproject使用的中心基础架构的估计量， NCRR赠款不直接向子弹或副本人员提供的直接资金。 抽象的 骨质减少和骨折使RETT综合征（RTT）的女孩和女性的临床过程变得复杂。 我们的研究表明，这些人的总体骨矿物质含量（BMC）分别为82％和57％的SD低于-1 SD和-2 SD（1）。 这项研究的目的是确定补充口服钙（CA）在RTT女孩和女性治疗骨质减少症中的功效。 我们假设通过减少骨矿物质的吸收和增加骨矿物质沉积，补充口服CA将逆转RTT女孩和妇女（尤其是年龄较大的年龄较大的年轻人）骨矿物质损失的进展。 为了检验这一假设，我们将使用54名RTT女孩和女性随机接受口服CA补充剂或安慰剂的Duel Energy X射线吸收法（DXA）测量BMC。 将测量Ca代谢的尿Ca排泄和静脉标记。 骨折的患病率将记录在Medcal病史中，而卧； 将进行体育测量和人体测量测量，以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丸计数以监控合规性。 我们预计BMC Z分数将增加，并且随着口服CA的补充而减少尿液CA，从而逆转了RTT女孩和女性骨矿物质损失的进展。 由于RTT女孩和妇女的寿命以及提供介入的策略，以防止与骨质减少症相关的不利后果，因此这些信息对于确定很重要。 假设 这项研究的目的是确定口服CA在治疗骨质减少症中的治疗功效和安全性。 我们假设补充口服CA可以通过减少RTT女孩和女性的骨矿物质吸收和增加骨矿物质的吸收和增加骨矿物质沉积来逆转骨矿物质损失的进展，尤其是在年轻人中的骨矿物质流失的进展。 具体目标 1。确定口服CA补充剂是否导致BYDXA测量的绝对BMC和BMC Z得分的增加，而安慰剂INRTT INRTT女性和女性 2。确定CA补充剂是否会导致Puerbertal RTT女孩中的绝对BMC和BMC Z得分的增加，而不是在Pustpubertal后RTT妇女中增加 3。确定口服Ca补充剂是否导致骨矿物质吸收的降低，通过N-甲状腺肽和尿氨基甲氨酸的比率比安慰剂中的安慰剂降低了 4。确定口服Ca补充剂是否导致骨矿物质沉积增加，而不是在RTT女孩和女性中，通过骨钙素和骨碱性磷酸酶测量。