Decisional Capacity and Informed Consent in Fragile X Syndrome

脆性 X 综合征的决策能力和知情同意

基本信息

批准号：
8437309
负责人：
Donald B Bailey
金额：
$ 62.7万
依托单位：
RESEARCH TRIANGLE INSTITUTE
依托单位国家：
美国
项目类别：
财政年份：
2012
资助国家：
美国
起止时间：
2012-09-26 至 2017-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8437309
关键词：
Adult Adverse effects Affect Behavior Blood Caregivers Clinical Treatment Clinical Trials Cognitive Consent Data Daughter Decision Aid Decision Making Developmental Disabilities Developmental Process Educational Assessment Ethics Family Female Female Adolescents Fragile X Syndrome Generations Gold Health Communication Heart Rate Human Impairment Individual Individual Differences Informed Consent Inherited Institutional Review Boards Intellectual functioning disability Intervention Learning Life Life Cycle Stages Literature Measures Molecular Nature Neurocognitive Parental Consent Parents Participant Perception Pharmacologic Substance Process Program Development Randomized Controlled Trials Reading Research Research Personnel Saliva Son Specialist Surveys Tablets Technology Testing Videotape base comparative efficacy consent capacity design digital evidence based guidelines experience male member mild neurocognitive impairment neuroimaging neuropsychological novel psychologic research study skills standard measure

项目摘要

DESCRIPTION (provided by applicant): Fragile X syndrome (FXS) is the most common inherited form of intellectual disability. The typical male has an IQ of 50 to 55. Some are severely affected (low IQ, nonverbal), while others are mildly affected (mild intellectual disabiliy, can read, very sociable). Females are generally less affected than males but have an even broader range in function. A few females have severe intellectual and adaptive impairments, but most have a normal or borderline intellectual ability; about one-third have mild cognitive impairment. This wide range in cognitive ability almost certainly leads to variable ability to make choices and live independently. This application focuses on a particular form of choice making-decisional capacity-the ability to make a meaningful decision about whether to participate in research. Individuals with FXS have served as participants in research studies for more than 30 years. For the most part, this research has been relatively noninvasive, limited to parent surveys and neuropsychological or psycho-educational assessments. Some studies have drawn blood, collected saliva, measured heart rate, or used neuroimaging, but until recently there has been a paucity of treatment research. This scenario has changed dramatically in the last 3 years. Advances in understanding the molecular basis of FXS have led to a new generation of targeted treatment approaches, and clinical trials are underway using a variety of compounds. The possibility of side effects and the potential for significant changes in behavior and learning abilty elevate to a new level the importance of obtaining meaningful consent. Researchers and IRB members need data to help guide decisions about involving individuals with FXS in the consent process. Only a few studies have investigated decisional capacity of individuals with intellectual and developmental disabilities, and none have studied individuals with FXS. Virtually nothing is known about the extent to which individuals with FXS are involved in decisions about research participation, and no studies have investigated decisional capacity in FXS. This project will assess the range of decisional capacity in FXS, identify factors associated with variability in decisional capacity, and develop and evaluate a tablet-based decision aid to assist in obtaining informed consent. The study team includes experts in FXS, human research protection professionals, specialists in developing materials for informed decision making, a leading researcher in FXS clinical trials, and researchers who have developed and evaluated gold-standard measures for characterizing decisional capacity. The study will provide important information about the nature of decisional capacity in FXS and will provide guidance for researchers, IRB members, and pharmaceutical companies seeking to include individuals with FXS as study participants. PUBLIC HEALTH RELEVANCE: This project will be the first to study the ability of individuals with fragile X syndrome to provide informed consent to participate in research. Given the advent of clinical trials and treatment possibilities for these individuals, understanding their decisiona capacity will be important for researchers, clinicians, IRB members, and pharmaceutical companies. This project will assess the range of decisional capacity in FXS, identify factors associated with variability in decisional capacity, and develop and evaluate a tablet-based decision aid to assist in obtaining informed consent.

描述（由申请人提供）：脆性 X 综合征 (FXS) 是最常见的智力障碍遗传形式。典型男性的智商为 50 到 55。有些人受到严重影响（智商低，非语言能力），而另一些人则受到轻微影响（轻度智力障碍，可以阅读，非常善于交际）。女性通常比男性受到的影响较小，但功能范围更广。少数女性有严重的智力和适应性障碍，但大多数女性智力正常或处于边缘水平；大约三分之一有轻度认知障碍。这种广泛的认知能力几乎肯定会导致不同的制造能力选择并独立生活。该应用程序侧重于一种特定形式的选择——决策能力——就是否参与研究做出有意义的决定的能力。 FXS 患者参与研究已有 30 多年。在很大程度上，这项研究相对无创，仅限于家长调查和神经心理学或心理教育评估。一些研究采取了抽血、收集唾液、测量心率或使用神经影像学的方法，但直到最近还缺乏治疗研究。这种情况在过去三年里发生了巨大的变化。对 FXS 分子基础了解的进展催生了新一代的靶向治疗方法，并且正在使用各种化合物进行临床试验。副作用的可能性以及行为和学习能力发生重大变化的可能性将获得有意义的同意的重要性提升到了一个新的水平。研究人员和 IRB 成员需要数据来帮助指导有关 FXS 患者参与同意过程的决策。只有少数研究调查了智力和发育障碍人士的决策能力，并且没有一项研究对 FXS 患者进行研究。事实上，人们对 FXS 患者参与研究决策的程度一无所知，也没有研究调查 FXS 的决策能力。该项目将评估 FXS 决策能力的范围，确定与决策能力变异性相关的因素，并开发和评估基于平板电脑的决策辅助工具，以协助获得知情同意。研究团队包括 FXS 专家、人类研究保护专业人员、开发知情决策材料的专家、FXS 临床试验的领先研究人员，以及开发和评估表征决策能力的黄金标准措施的研究人员。该研究将提供有关 FXS 决策能力性质的重要信息，并将为寻求将 FXS 患者纳入研究参与者的研究人员、IRB 成员和制药公司提供指导。公共健康相关性：该项目将是第一个研究脆性 X 综合征患者提供知情同意参与研究的能力的项目。鉴于这些人的临床试验和治疗可能性的出现，了解他们的决策能力对于研究人员、临床医生、IRB 成员和制药公司来说非常重要。该项目将评估 FXS 决策能力的范围，确定与决策能力变异性相关的因素，并开发和评估基于平板电脑的决策辅助工具，以协助获得知情同意。