Expanding the Observer-Reported Communication Ability (ORCA) Measure: Measuring the communication ability of individuals with rare, neurodevelopmental disorders

扩大观察者报告的沟通能力（ORCA）测量：测量患有罕见神经发育障碍的个体的沟通能力

• 批准号: 10399549
• 负责人: Christina K. Zigler
• 金额: $ 71.73万
• 依托单位: DUKE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-05-01 至 2026-04-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10399549
• 关键词: Expanding; Observer; Reported; Communication; Ability

PROJECT SUMMARY / ABSTRACT: Children with neurodevelopmental disorders (NDDs) typically have severe deficits in domains of daily functioning like communication, which families consistently list as one of their top clinical priorities. However, existing clinical outcome assessments (COAs) have major limitations, including the inability to discriminate among individuals with low communication ability and/or are primarily nonverbal. Further, there are no current caregiver-reported measures designed for clinical trials that have high quality validity evidence for individuals with NDDs. To address this critical need, our team designed the Observer-Reported Communication Ability (ORCA) measure to assess expressive, receptive, and pragmatic forms of communication, regardless of the modalities used by a child (e.g., vocalizations, gestures, assistive devices). Qualitative and quantitative evidence support its validity for individuals with Angelman syndrome, and excitingly, the ORCA is likely to be generalizable to other NDD populations. This proposed study will allow us to use rigorous development methods to expand the ORCA measure to a broad range of NDD populations and, using existing COAs, provide investigators with a comprehensive set of tools to evaluate changes in communication ability in NDD populations in pediatric clinical trials. Our study team includes experts in COA development, communication, clinical trials, and neuropsychology. Importantly, the team also includes a Stakeholder Engagement Group (SEG) consisting of 16 leaders in the field; 12 parents/patient advocates and 4 clinicians who will provide access to 13 distinct NDD populations for the studies planned in this proposal. An External Technical Advisory Committee (ETAC) will provide additional guidance and consists of clinicians, methodologists, speech-language pathologists, industry experts, and patient advocates. In the 2-year UG3 planning phase, we will conduct qualitative interviews with 8-12 parents for each of our 13 NDDs and with 8-12 communication experts to identify the core concepts of communication for each of the NDDs (Aim 1). We will use the results from the qualitative interviews and data collected in seven ongoing natural history studies to develop targeted plans for the UH3 implementation phase to expand and validate the ORCA measure for each of the 13 NDDs (Aim 2). In the 3-year UH3 phase, we will expand the ORCA measure and conduct, as needed, additional qualitative interviews and longitudinal quantitative studies to evaluate the reliability, validity and responsiveness of the ORCA measure alongside other communication COAs (Aim 3). Finally, we will have approximately 1500 caregivers of typically developing children from birth to <6 years of age complete the expanded ORCA measure twice (6-months apart) to estimate age-based norm scores and examine responsiveness of the ORCA measure over time. With constant feedback from the SEG and the ETAC, we will provide an expanded ORCA measure to the research field that can capture communication ability across a diverse range of NDDs, and an endpoint framework for researchers to evaluate the efficacy of interventions using an outcome that is important to families of children and adolescents with NDDs.

项目总结/摘要： 患有神经发育障碍（NDD）的儿童通常在日常功能方面存在严重缺陷 比如沟通，家庭一直将其列为最重要的临床优先事项之一。然而，现有的临床 结果评估（COA）有很大的局限性，包括无法区分个人， 沟通能力低和/或主要是非语言。此外，目前还没有任何政府报告的措施， 专为临床试验而设计，这些试验对NDD患者具有高质量的有效性证据。 为了满足这一关键需求，我们的团队设计了观察者报告的沟通能力（ORCA）测量， 评估表达、接受和实用的交流形式，无论孩子使用何种方式 (e.g.,发声、手势、辅助设备）。定性和定量证据支持其有效性， Angelman综合征的个体，令人兴奋的是，ORCA很可能推广到其他NDD 人口。这项拟议的研究将使我们能够使用严格的开发方法来扩大ORCA措施 广泛的NDD人群，并使用现有的COA，为研究者提供一套全面的 评估儿科临床试验中NDD人群沟通能力变化的工具。 我们的研究团队包括COA开发，沟通，临床试验和神经心理学专家。 重要的是，该小组还包括一个利益攸关方参与小组，由16名该领域的领导人组成; 12 父母/患者倡导者和4名临床医生，他们将为研究提供13个不同的NDD人群 在这个提案中。外部技术咨询委员会（ETAC）将提供额外的指导， 由临床医生、方法学家、言语语言病理学家、行业专家和患者倡导者组成。 在为期2年的UG 3规划阶段，我们将为13个NDD中的每个NDD与8-12名家长进行定性访谈 并与8-12名传播专家合作，确定每个国家发展目标的核心传播概念（目标1）。 我们将使用定性访谈的结果和七项正在进行的自然历史研究中收集的数据， 为UH 3实施阶段制定有针对性的计划，以扩展和验证ORCA措施， 13个国家发展计划（目标2）。在为期3年的UH 3阶段，我们将扩大ORCA措施，并根据需要进行， 额外的定性访谈和纵向定量研究，以评估信度，效度和 ORCA措施的响应性以及其他沟通COA（目标3）。最后，我们将有 大约1500名从出生到6岁以下发育正常的儿童的照顾者完成了扩大的 ORCA测量两次（间隔6个月），以估计基于年龄的标准评分，并检查 ORCA随时间推移进行测量。随着SEG和ETAC的不断反馈，我们将提供一个扩展的 ORCA测量研究领域，可以捕获跨各种NDD的通信能力， 一个终点框架，供研究人员使用一个重要的结果来评估干预措施的有效性， NDD儿童和青少年的家庭。