A neuroprotective therapy is the single most important unmet medical need in Parkinson's disease. Several promising agents in the laboratory have been tested in the clinic, but none has been established in clinical trials to have a disease modifying effect despite positive results because of potential confounding symptomatic or pharmacologic effects. The delayed start design was developed to try to avoid a symptomatic confound when testing a putative neuroprotective therapy. In this study design, patients are randomly assigned to study drug or placebo in the first phase of the study, and both groups receive the active drug in the second phase. If benefits seen at the end of phase I persist through the end of phase II, they cannot be readily explained by a symptomatic effect (as patients in both groups are receiving the same medication) and benefits in the early start group must relate to the early initiation of the treatment. Although the precise mechanism responsible for such an effect can be debated, positive results in a delayed start study indicate that patients who receive early treatment have a better outcome than those where the treatment is delayed. We are using the delayed start design to assess the potential disease modifying effects of rasagiline in a prospective double blind controlled trial (the ADAGIO study). We here describe the rationale for the study and baseline characteristics of the 1,176 patients who have been enrolled into the trial.

译文

神经保护疗法是帕金森氏病中最重要的未满足的医疗需求。实验室中的几种有前途的药物已经在临床上进行了测试,但是在临床试验中,尽管由于潜在的混淆症状或药理作用而获得了积极的结果,但没有一个具有疾病缓解作用。开发了延迟启动设计,以避免在测试假定的神经保护疗法时出现症状混淆。在此研究设计中,患者在研究的第一阶段被随机分配到研究药物或安慰剂,两组在第二阶段均接受活性药物。如果在I期结束时看到的益处持续到II期结束时,则不能很容易地用症状效应来解释 (因为两组患者都接受相同的药物治疗),并且早期开始组的益处必须与早期开始治疗有关。尽管可以讨论导致这种作用的确切机制,但延迟开始研究中的积极结果表明,接受早期治疗的患者比延迟治疗的患者有更好的结果。在一项前瞻性双盲对照试验 (ADAGIO研究) 中,我们正在使用延迟启动设计来评估雷沙吉兰的潜在疾病修饰作用。我们在这里描述研究的基本原理和已纳入试验的1,176患者的基线特征。

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