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.

项目概要 对于婴儿来说，移动性是探索、沟通以及与护理人员互动的主要驱动力， 支持学习和一般发育，患有 DS 的婴儿从很小的时候就表现出发育迟缓。 在这些领域，人们普遍认为，护理人员尽早实施具有成本效益。 需要在婴儿期进行干预来解决这些延迟问题。 涉及体重支撑的安全带系统，允许婴儿在不活动时自由活动 但独立移动的安全带系统已广泛应用于各个年龄段的活动能力儿童。 障碍，但尚未在运动和活动支持婴儿参与方面进行系统研究。 与家里的物体和人的日常互动是早期学习的基础。 运动迟缓和行动障碍是患有这些疾病的婴儿参与这些关键环境的障碍 DS。拟议的工作将为家庭移动相关干预措施的首次临床试验奠定基础。 通过开发和完善纵向收集数据的程序，专门为 DS 婴儿量身定制 目标1是检验安全带系统的可行性和可接受性，进行纵向研究。 访问时间表、数据收集方法（亲自与虚拟）以及家庭标准化评估 为了实现这一目标，我们将实施一项详细的调查。 重点评估护理人员对安全带系统和研究参与的态度，收集以下方面的数据 研究入学率、保留率、面对面和虚拟数据收集方法的有效性、数据率 目标 2 是确定观察变量。 最适合作为未来临床试验评估有效性的主要结果指标 为了实现这一目标，我们将收集有关 DS 婴儿的安全带干预的观察数据。 婴儿姿势和运动、物体探索、沟通以及护理者-婴儿联合物体接触 这两个目标将通过对 15 名 DS 婴儿进行的纵向研究来实现。 和他们的照顾者进行了为期 6 个月的研究，在此期间我们将记录这些动物的自然历史 行为并检查它们与包括 DCC 推荐的标准化评估的关系 这种方法基于我们团队之前的工作，将允许。 我们设计一种基于家庭体重支持的安全带干预措施，其有效性可以评估 在随后的临床试验中，以家庭为基础的干预将提高干预研究的可及性。 那些在参与实验室研究方面面临障碍的家庭，从而增加了样本多样性， 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、纵向家庭数据收集以及对婴儿的评估管理 DS 及其家人有必要进行未来高影响力的 1 期临床试验。