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首页> 外文期刊>Journal of neurology >Sample size calculations for clinical trials targeting tauopathies: a new potential disease target
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Sample size calculations for clinical trials targeting tauopathies: a new potential disease target

机译：针对tauopathies的临床试验的样本量计算：新的潜在疾病靶标

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Disease-modifying therapies are being developed to target tau pathology, and should, therefore, be tested in primary tauopathies. We propose that progressive apraxia of speech should be considered one such target group. In this study, we investigate potential neuroimaging and clinical outcome measures for progressive apraxia of speech and determine sample size estimates for clinical trials. We prospectively recruited 24 patients with progressive apraxia of speech who underwent two serial MRI with an interval of approximately 2 years. Detailed speech and language assessments included the Apraxia of Speech Rating Scale and Motor Speech Disorders severity scale. Rates of ventricular expansion and rates of whole brain, striatal and midbrain atrophy were calculated. Atrophy rates across 38 cortical regions were also calculated and the regions that best differentiated patients from controls were selected. Sample size estimates required to power placebo-controlled treatment trials were calculated. The smallest sample size estimates were obtained with rates of atrophy of the precentral gyrus and supplementary motor area, with both measures requiring less than 50 subjects per arm to detect a 25 % treatment effect with 80 % power. These measures outperformed the other regional and global MRI measures and the clinical scales. Regional rates of cortical atrophy, therefore, provide the best outcome measures in progressive apraxia of speech. The small sample size estimates demonstrate feasibility for including progressive apraxia of speech in future clinical treatment trials targeting tau.
机译：正在开发针对tau病理学的疾病缓解疗法，因此应在原发性Tauopathies中进行测试。我们建议，进行性语言失用应被视为此类目标人群之一。在这项研究中，我们调查了进行性语言失用的潜在神经影像学和临床结局指标，并确定了用于临床试验的样本量估计值。我们前瞻性地招募了24名进行性语言失用的患者，他们接受了两次连续MRI检查，间隔约2年。详细的言语和语言评估包括言语失用评估量表和运动言语障碍严重性量表。计算心室扩张率和全脑，纹状体和中脑萎缩率。还计算了38个皮质区域的萎缩率，并选择了与对照最能区分患者的区域。计算了安慰剂对照治疗试验所需的样本量估计值。最小的样本量估计值是通过中央前回的萎缩率和辅助运动区获得的，这两种措施每只手臂需要少于50名受试者，才能以80％的功率检测25％的治疗效果。这些措施优于其他区域和全球MRI措施以及临床规模。因此，皮质萎缩的区域发生率可为进行性语言失用提供最佳的结局指标。小样本估计值表明在针对tau的未来临床治疗试验中包括进行性语言失语的可行性。

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