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，交流和与护理人员互动的主要驱动力 支持学习和一般发展。从生命的早期开始，患有DS暴露发育延迟的婴儿 跨这些领域。有宽度的协议，具有成本效益，照顾者的提前 需要在婴儿期的干预措施来解决这些延迟。这种干预的候选方法 涉及体重支持的线束系统，使婴儿在不在的时候自由移动 但是独立移动。线束系统已被广泛用于活动能力的所有年龄段的儿童 损伤但尚未系统地研究DS。运动和流动性支持婴儿参与 在每天与房屋中的物体和人们的互动中，这是早期学习的基础。 电机延误和移动性挑战代表了参与这些关键环境的障碍 DS。拟议的工作将为基于家庭的移动性干预措施的首次临床试验奠定了基础 专门针对患有DS的婴儿量身定制的，通过开发和完善程序，以纵向收集数据 家。目标1是检查线束系统的可行性和可接受性，纵向研究 请访问时间表，数据收集方法（个人与虚拟）以及内部标准化评估 DS及其家人的婴儿的程序。为了实现这一目标，我们将实施详细的调查 专注于评估护理人员的人参加安全带系统和研究参与，收集数据 研究注册，保留率，面对面的有效性和数据收集的虚拟方法，数据速率 完成和对标准措施的家庭管理。目标2是确定观察变量 最适合作为评估有效性的未来临床试验中的主要结果指标 DS婴儿的线束干预。为了实现这一目标，我们将收集有关的观察数据 婴儿姿势和运动，物体探索，交流和护理人员关节对象参与 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 他们的照顾者在一个6个月的时间内进行了，在此期间，我们将记录这些自然历史 行为并检查与他们与DCC推荐的标准化评估包括 发展，社会交流和语言。根据我们团队以前的工作，这种方法将允许 我们设计一个基于家庭的体重支持线束干预，可以评估其有效性 在随后的临床试验中。基于家庭的干预将增强对干预研究的可及性 面临基于实验室研究的障碍的家庭，从而增加样本多样性和 表示。该项目完成后，我们将制定可行且可接受的程序 用于安全带系统设置，纵向内部数据收集以及与患有婴儿的婴儿进行评估 DS及其家人必须进行未来的高影响力1期临床试验。