Tanner Caroline M, Cummings Steven R, Schwarzschild Michael A, Brown Ethan G, Dorsey E Ray, Espay Alberto J, Galifianakis Nicholas B, Goldman Samuel M, Litvan Irene, Luthra Nijee, McFarland Nikolaus R, Mitchell Kyle T, Standaert David G, Bauer Douglas C, Greenspan Susan L, Beck James C, Lyles Kenneth W
Department of Neurology, Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.
Parkinson's Disease Research Education and Clinical Center, San Francisco Veteran's Affairs Health Care System, San Francisco, CA, USA.
NPJ Parkinsons Dis. 2021 Mar 1;7(1):16. doi: 10.1038/s41531-021-00162-1.
The Trial of Parkinson's And Zoledronic acid (TOPAZ, https://clinicaltrials.gov/ct2/show/NCT03924414 ) is a unique collaboration between experts in movement disorders and osteoporosis to test the efficacy of zoledronic acid, an FDA-approved parenteral treatment for osteoporosis, for fracture prevention in people with neurodegenerative parkinsonism. Aiming to enroll 3,500 participants age 65 years or older, TOPAZ is one of the largest randomized, placebo-controlled clinical trials ever attempted in parkinsonism. The feasibility of TOPAZ is enhanced by its design as a U.S.- wide home-based trial without geographical limits. Participants receive information from multiple sources, including specialty practices, support groups and websites. Conducting TOPAZ in participants' homes takes advantage of online consent technology, the capacity to confirm diagnosis using telemedicine and the availability of research nursing to provide screening and parenteral therapy in homes. Home-based clinical research may provide an efficient, convenient, less expensive method that opens participation in clinical trials to almost anyone with parkinsonism.
帕金森病与唑来膦酸试验（TOPAZ，https://clinicaltrials.gov/ct2/show/NCT03924414 ）是运动障碍和骨质疏松症领域专家之间的一次独特合作，旨在测试唑来膦酸（一种经美国食品药品监督管理局批准用于治疗骨质疏松症的肠胃外给药药物）对神经退行性帕金森综合征患者预防骨折的疗效。TOPAZ计划招募3500名65岁及以上的参与者，是帕金森病领域有史以来规模最大的随机、安慰剂对照临床试验之一。TOPAZ作为一项全美范围内不受地域限制的居家试验，其设计增强了试验的可行性。参与者从多个渠道获取信息，包括专科医疗机构、支持小组和网站。在参与者家中开展TOPAZ试验利用了在线同意技术、通过远程医疗确认诊断的能力以及研究护理人员上门提供筛查和肠胃外治疗的便利。居家临床研究可能提供一种高效、便捷、成本较低的方法，使几乎任何帕金森病患者都能参与临床试验。
