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）是智力障碍的最常见遗传原因 700个出生。临床试验试图识别针对智力障碍的DS特定治疗方法 对于多种常见DS相关的合并症。在临床研究中，均获得 参与者的父母（或因智力残疾而需要DS的成年人需要的法定监护人） 通常需要参与个人的同意。许多DS的人可能不会 有能力做出有关临床试验参与的决定，但这从未进行过调查。有 现有的知识差距，因为缺乏指导使用同意参与的证据 DS个体的研究。目前针对DS患者的临床试验通常需要同意 对于所有参与者的参与，这阻止了无法提供同意的个人入学。这 可能会导致较高功能的DS个体的选择偏差。这反过来限制了 结果给所有具有DS的人，为旨在改善认知的疗法产生潜在的上限效应 并且是DS患者研究参与的障碍。该提议的总体目标是 开发一个基于证据的框架来指导对DS的个人的同意方法 参加临床试验。这将实现利用两个现有资源。第一个是对 大批对通过DS招募的研究参与感兴趣的DS的父母 连接和Lumind-IDSC基金会。第二个是使用经过验证的小儿工具，修改了 麦克阿瑟临床研究能力评估工具（MACCAT-CR）确定 有DS的个人可以决定参与临床研究。具体目的包括：目标1： 确定同意参与DS患者临床试验的父母/代表性偏好。 目标2：确定DS个人的能力，以决定临床试验参与 诊所恢复的DS儿童和成人样本。