Phase 2 Teriparatide for Tx of Idiopathic Osteoporosis in Premenopausal Women

特立帕肽治疗绝经前女性特发性骨质疏松症的 2 期临床试验

• 批准号: 8020203
• 负责人: Elizabeth J Shane
• 金额: $ 22.85万
• 依托单位: COLUMBIA UNIVERSITY HEALTH SCIENCES
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-09-15 至 2015-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8020203
• 关键词: Phase; Teriparatide; Tx; Idiopathic; Osteoporosis

DESCRIPTION (provided by applicant): IOP in premenopausal women is defined as osteoporosis that occurs in young, otherwise healthy women with intact gonadal function and no secondary cause of bone loss. IOP in premenopausal women is an uncommon disorder. The estimated prevalence in the United States, based on women with documented low trauma fractures or low femoral neck bone mineral density (BMD), is approximately 113,000. There is no FDA-approved therapy for premenopausal women with IOP, many of whom have sustained multiple fractures or have extremely low BMD. This proposal seeks support for a Phase 2 clinical trial to investigate the efficacy and safety of teriparatide, human recombinant PTH(1-34), for IOP in premenopausal women. It is based upon previous work of the investigators in which they defined microarchitectural abnormalities in this disorder: thin cortices, trabecular bone loss, fewer trabecular plates, and reduced stiffness or strength. The study will test the central hypothesis that anabolic therapy with teriparatide, a drug that increases osteoblast-mediated bone formation, will safely increase areal and volumetric BMD in premenopausal women with IOP. In this Phase 2 clinical trial, which is randomized and placebo-controlled, the investigators will characterize effects of teriparatide on areal and volumetric BMD, microarchitecture, remodeling, stiffness, mineralization and collagen properties in women with IOP. The investigators will use both state-of-the-art and novel approaches to skeletal macro- and micro-imaging that have never been applied to the therapy of IOP. These new approaches will provide a rational basis for a much needed therapeutic approach to IOP in premenopausal women and will have high impact upon clinical practice. By investigating the therapy of IOP and improving the health of young women with unexplained osteoporosis, this proposal addresses a key goal of the FDA's Office of Orphan Product Development OPD grant program, namely to support the clinical development of products for use in rare diseases or conditions where no current therapy exists.

描述（由申请人提供）： 绝经前女性的IOP被定义为骨质疏松症，发生在具有完整性性腺功能的年轻，健康的女性中，没有造成骨质流失的次要原因。 绝经前女性的IOP是一种罕见的疾病。 美国估计的患病率是基于记录的低创伤性骨折或股骨颈部骨矿物质密度（BMD）低约113,000的女性。 IOP的绝经前女性没有FDA批准的疗法，其中许多人患有多个骨折或BMD极低。 该提案寻求支持第二阶段临床试验，以调查teriparatide，人类重组PTH（1-34）的疗效和安全性，以促进绝经前妇女的IOP。 它基于研究人员以前的工作，在该研究中，他们定义了这种疾病中的微构造异常：薄皮质，小梁骨质流失，小梁板的较少以及刚度降低的刚度或力量。 该研究将检验一个中心假设，即使用Teriparatide（增加成骨细胞介导的骨形成）的代谢疗法将安全增加IOP的绝经前妇女的面积和体积BMD。 在这一阶段2临床试验中，该试验是随机和安慰剂对照的，研究人员将表征teriparatide对IOP女性对女性的面积和体积BMD，微结构，重塑，僵硬，矿化和胶原蛋白特性的影响。 研究人员将使用从未应用于IOP治疗的最先进和新颖的骨骼宏观和微观成像。 这些新方法将为绝经前妇女使用急需的治疗方法提供合理的基础，并将对临床实践产生很大的影响。 通过研究IOP的疗法并改善无法解释的骨质疏松症的年轻女性的健康，该提案解决了FDA孤儿产品开发OPD赠款计划的关键目标，即支持在不存在当前治疗的情况下用于稀有疾病或疾病的临床开发。