Feasibility and outcome measures for infants with Down syndrome: Advancing clinical trial readiness for a harness-based mobility intervention

唐氏综合症婴儿的可行性和结果测量：推进基于安全带的活动干预的临床试验准备

• 批准号: 10656637
• 负责人: Nicole Baumer
• 金额: $ 47.75万
• 依托单位: BOSTON UNIVERSITY (CHARLES RIVER CAMPUS)
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-04-04 至 2025-03-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10656637
• 关键词: Address; Advanced Development; Age; Agreement; Attitude; Behavior; Behavior Therapy; Behavioral; Body Weight; Caregiver support; Caregivers; Case Study; Child; Clinical; Clinical Trials; Communication; Data; Data Collection; Development; Developmental Delay Disorders; Devices; Down Syndrome; Early Intervention; Effectiveness; Enrollment; Environment; Exhibits; Family; Family Research; Foundations; Future; Goals; Home; Hybrids; Impairment; Individual; Infant; Infant Care; Infant Development; Intervention; Intervention Studies; Joints; Laboratories; Language; Learning; Life; Literature; Locomotion; Longitudinal Studies; Measures; Mediating; Methodology; Methods; Motor; Movement; Muscle hypotonia; Natural History; Outcome; Outcome Measure; Parents; Participant; Perception; Persons; Phase I Clinical Trials; Play; Posture; Procedures; Recommendation; Research; Research Design; Sampling; Schedule; Social Interaction; Sorting; Standardization; Surveys; System; Time; Travel; Visit; Walking; Work; acceptability and feasibility; clinical care; clinical trial readiness; cost effective; design; effectiveness evaluation; evidence base; experience; infancy; portability; primary outcome; satisfaction; social communication; standardize measure; virtual

PROJECT SUMMARY For infants, mobility is a primary driver for exploration, communication, and interaction with caregivers that support learning and general development. From early in life, infants with DS exhibit developmental delays across these domains. There is widespread agreement that cost effective, caregiver-implemented early interventions during infancy are needed to address these delays. A candidate approach to such an intervention involves body weight supported harness systems that allow infants to move freely at a time when they are not yet independently mobile. Harness systems have been widely used in children of all ages with mobility impairments but have not been systematically studied in DS. Movement and mobility support infant participation in the everyday interactions with objects and people in their homes that are foundational for early learning. Early motor delays and mobility challenges represent an obstacle to participation in these key contexts for infants with DS. The proposed work will set the stage for the first clinical trial of a home-based mobility-related intervention specifically tailored for infants with DS by developing and refining procedures for collecting data longitudinally in the home. Aim 1 is to examine the feasibility and acceptability of the harness system, longitudinal study visit schedule, data collection methods (in person vs. virtual), and in-home standardized assessment procedures for infants with DS and their families. To achieve this aim, we will implement a detailed survey focused on assessing caregiver attitudes toward the harness system and study participation, gather data on study enrollment, retention, the effectiveness of in-person and virtual methods of data collection, rates of data completion, and home administration of standardized measures. Aim 2 is to identify observational variables best suited to serve as primary outcome measures in future clinical trials evaluating the effectiveness of the harness intervention for infants with DS. To achieve this aim, we will gather observational data on infant posture and locomotion, object exploration, communication, and caregiver-infant joint object engagement during everyday play at home. Both aims will be addressed through a longitudinal study of 15 infants with DS and their caregivers carried out over a 6-month period, during which we will document the natural history of these behaviors and examine their relations with INCLUDE DCC-recommended standardized assessments of development, social communication, and language. This approach, based on our team’s previous work, will allow us to design a home-based body weight supported harness intervention whose effectiveness can be evaluated in a subsequent clinical trial. Home-based intervention will enhance accessibility to intervention research for families who face barriers to laboratory-based research participation and thereby increase sample diversity and representation. Upon completion of this project, we will have developed the feasible and acceptable procedures for harness system setup, longitudinal in-home data collection, and assessment administration with infants with DS and their families necessary to conduct a future high-impact phase 1 clinical trial.

项目摘要 对于婴儿来说，移动性是探索、交流和与看护者互动的主要驱动力， 支持学习和全面发展。从生命早期开始，患有DS的婴儿表现出发育迟缓 在这些领域。人们普遍认为，成本效益高，无论何时实施， 需要在婴儿期进行干预，以解决这些延迟问题。这种干预的备选办法是 涉及体重支撑的安全带系统，该安全带系统允许婴儿在他们不能自由移动时自由移动。 但独立移动的。儿童呼吸机系统已广泛用于所有年龄段的儿童， 但尚未在DS中进行系统研究。运动和行动支持婴儿参与 在日常生活中与家中的物品和人的互动中，这是早期学习的基础。早期 运动延迟和移动性挑战是婴儿参与这些关键环境的障碍， DS.这项拟议中的工作将为首次基于家庭的移动相关干预的临床试验奠定基础 通过开发和完善纵向收集数据的程序， 家中目的1是检验安全带系统的可行性和可接受性，纵向研究 访视时间表、数据收集方法（亲自与虚拟）和家庭标准化评估 为患有DS的婴儿及其家人提供的程序。为达致这个目的，我们会进行一项详细的调查， 专注于评估护理人员对安全带系统和研究参与的态度，收集以下数据： 研究入组、保留、面对面和虚拟数据收集方法的有效性、数据收集率 完成和家庭管理的标准化措施。目标2是确定观测变量 最适合在未来的临床试验中作为评价有效性的主要结局指标 对患有DS的婴儿进行安全带干预。为达致这个目标，我们会收集以下观测数据： 婴儿姿势和运动、物体探索、交流和婴儿-婴儿关节物体接合 每天在家玩。这两个目标将通过对15名DS婴儿的纵向研究来解决 和他们的照顾者进行了6个月的时间，在此期间，我们将记录这些自然历史， 行为，并检查它们与纳入DCC推荐的标准化评估的关系， 发展、社会交流和语言。这种方法基于我们团队以前的工作， 我们设计了一个家庭为基础的体重支持挽具干预，其有效性可以评估 在随后的临床试验中。家庭干预将提高干预研究的可及性， 家庭面临的障碍，以实验室为基础的研究参与，从而增加样本的多样性， 表示.待这项计划完成后，我们便会制订出可行和可接受的程序， 用于安全带系统设置、纵向家庭数据收集和婴儿评估管理 DS和他们的家庭需要进行未来的高影响1期临床试验。