DEtermining Capacity and Informing Down syndrome Assent Strategies (DECIDAS)

唐氏综合症(DS)是导致智力残疾的最常见的遗传原因，患病率为1/4 700个新生儿。临床试验试图确定DS特异性治疗智力障碍的方法以及 用于多种常见的DS相关并发症。在临床研究中，两项许可都来自 参与者的父母(或法定监护人，如成人因智力残疾而需要一名患有DS的人) 此外，通常还需要得到参与个人的同意。许多患有DS的人可能不会 有能力就参与临床试验做出决定，但这从未被调查过。的确有 现有的知识差距，因为缺乏证据来指导使用同意来参与 对患有DS的个体进行研究。目前针对DS患者的临床试验通常需要获得许可 对于所有参与者的参与，这防止了无法提供同意的个人的登记。这 可能会导致对患有DS的高功能个体的选择偏见。这反过来又限制了 结果对所有DS患者来说，为旨在改善认知的治疗创造了潜在的天花板效应 对于患有DS的人来说，这是学习参与的障碍。这项提议的首要目标是 开发基于证据的框架，以指导对DS患者同意的方法 参与临床试验。这将利用现有的两项资源来实现。第一个是对一个 通过DS招募的对参与研究感兴趣的DS患者的大量父母- Connect和Lumind-IDSC基金会。第二种是使用经过验证的儿科工具，即改良的 麦克阿瑟临床研究能力评估工具(MacCAT-CR)，以确定 DS患者可以决定是否参与临床研究。具体目标包括：目标1： 确定父母/代表同意参与DS患者的临床试验的偏好。 目的2：确定DS患者对参与临床试验做出决定的能力 门诊招募的患有DS的儿童和成人样本。

Down Syndrome (DS) is the most common genetic cause of intellectual disability, with a prevalence of one in 700 births. Clinical trials have attempted to identify DS-specific treatments for intellectual disability as well as for the multiple common DS-associated comorbidities. In clinical research studies, both permission from the participant’s parent (or legal guardian in the case of adults with DS requiring one due to intellectual disability) as well as assent from the participating individual are generally required. Many individuals with DS may not have capacity to make decisions about clinical trial participation, but this has never been investigated. There is an existing knowledge gap as there is a lack of evidence to guide the use of assent for participation in research studies in individuals with DS. Current clinical trials for individuals with DS typically require assent for participation from all participants, which prevents enrollment of individuals unable to provide assent. This may lead to a selection bias for higher functioning individuals with DS. This in turn limits generalizability of results to all individuals with DS, creates a potential ceiling effect for therapies intended to improve cognition and is a barrier to study participation for individuals with DS. The overarching goal of this proposal is to develop an evidence-based framework to guide the approach to assent for individuals with DS to participate in clinical trials. This will be achieved leveraging two existing resources. The first is a survey of a large pool of parents of individuals with DS who are interested in research participation recruited through DS- Connect and the LuMind-IDSC foundation. The second is the use of a validated pediatric tool, the modified MacArthur Competence Assessment Tool for Clinical Research (MacCAT-CR), to determine the ability of individuals with DS to make decisions about participation in clinical research. Specific Aims include: Aim 1: To determine parent/representative preferences for assent to participate in clinical trials in individuals with DS. Aim 2: To determine the capacity of individuals with DS to make decisions about clinical trial participation in a clinic-recruited sample of children and adults with DS.