DEtermining Capacity and Informing Down syndrome Assent Strategies (DECIDAS)

确定能力并告知唐氏综合症同意策略 (DECIDAS)

• 批准号: 10696233
• 负责人: Daniel Combs
• 金额: $ 23.03万
• 依托单位: UNIVERSITY OF ARIZONA
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-09-08 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10696233
• 关键词: 6 year old; 7 year old; Adult; Advocacy; Age; Agreement; Authorization documentation; Birth; Child; Childhood; Clinic; Clinical Assessment Tool; Clinical Research; Clinical Trials; Cognition; Collaborations; Competence; Consent; Data; Decision Making; Down Syndrome; Enrollment; Ethicists; Exclusion; Family member; Foundations; Funding; Genetic; Goals; Hepatitis; Hypothyroidism; Impaired cognition; Individual; Informed Consent; Intellectual functioning disability; Knowledge; Legal; Legal Guardians; Obstructive Sleep Apnea; Parents; Participant; Persons; Population; Prevalence; Process; Registries; Research; Resources; Rights; Risk; Sampling; Schools; Selection Bias; Surveys; United States; United States National Institutes of Health; authority; common rule; comorbidity; congenital heart disorder; decision-making capacity; design; empowerment; evidence base; improved; interest; marginalization; mental age; neglect; preference; prevent; programs; recruit; research study; tool

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.

唐氏综合症 (DS) 是智力障碍最常见的遗传原因，患病率为 1% 临床试验已尝试确定针对智力障碍和 DS 的特定治疗方法。 对于多种常见的 DS 相关合并症，两者均获得了临床研究的许可。 参与者的父母（或法定监护人，如果患有 DS 的成年人因智力障碍而需要监护人） 通常不需要征得参与个人的同意。许多患有 DS 的人可能不需要。 有能力做出有关临床试验参与的决定，但这从未被调查过。 现有的知识差距，因为缺乏证据来指导使用同意参与 当前针对 DS 患者的临床试验通常需要获得同意。 所有参与者都可以参与，这可以防止无法同意的个人参加。 可能会导致对功能较高的 DS 个体的选择偏差，这反过来又限制了该方法的普遍性。 对所有 DS 患者的结果，为旨在改善认知的治疗创造了潜在的天花板效应 并且是 DS 患者参与研究的障碍 该提案的总体目标是 制定一个基于证据的框架来指导 DS 患者同意的方法 这将利用两个现有资源来实现。 大量 DS 患者的家长，他们对通过 DS 招募的研究参与感兴趣 Connect 和 LuMind-IDSC 基金会第二个是使用经过验证的儿科工具（经过修改）。 麦克阿瑟临床研究能力评估工具（MacCAT-CR），以确定 患有 DS 的个体做出参与临床研究的决定的具体目标包括： 目标 1： 确定家长/代表是否同意参加 DS 患者的临床试验。 目标 2：确定 DS 患者做出有关参与临床试验的决定的能力 临床采集的 DS 儿童和成人样本。