A Histamine Pharmacodynamic Biomarker to Guide Treatment in Pediatric Asthma

指导小儿哮喘治疗的组胺药效生物标志物

基本信息

批准号：
10705453
负责人：
BRIDGETTE L. JONES
金额：
$ 7.13万
依托单位：
CHILDREN'S MERCY HOSP (KANSAS CITY, MO)
依托单位国家：
美国
项目类别：
财政年份：
2022
资助国家：
美国
起止时间：
2022-12-01 至 2024-05-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10705453
关键词：
Address African American Antihistamines Area Asthma Award Black race Caucasians Child Childhood Childhood Asthma Clinical Clinical Pharmacology Clinical Research Clinical Trials Conduct Clinical Trials Controlled Clinical Trials Cross-Sectional Studies Data Development Dose Double-Blind Method Effectiveness Enrollment Extrinsic asthma Family Frequencies Future Glean Goals Health Histamine Intervention Iontophoresis Knowledge Lasers Methods Monitor Outcome Parents Participant Patient Recruitments Patients Pediatric Research Pharmaceutical Preparations Physicians Placebo Control Population Heterogeneity Race Reporting Research Research Activity Research Design Research Personnel Safety Scientist Therapeutic Therapeutic Clinical Trial Therapeutic Uses Time Training Underrepresented Populations Work career clinical trial recruitment cohort community partnership comparative efficacy design discontinuation trial double-blind placebo controlled trial efficacy clinical trial evidence base improved parent grant participant enrollment participant retention pediatric drug development pharmacodynamic biomarker predictive marker recruit retention rate skills success therapeutically effective treatment response trial design

项目摘要

Project Summary The overarching goal of the parent award is to validate the Histamine Iontophoresis with Laser Doppler (HILD) flowimetry biomarker for prediction of treatment response to Levocetirizine among children with asthma. This research supplement will support both aims of the parent award as the parent award primary research activities include the conduct of a large clinical trial in children. Completion of safe and efficient clinical trials in children is essential for pediatric drug development. Over 20% of pediatric clinical trials fail or are discontinued early due to lack of enrollment and over 30% of trials do not meet initially defined recruitment goals. Barriers to participant recruitment and retention within pediatric clinical trials must be examined and addressed to increase the number of successful trials and the dissemination of important pediatric dosing, safety, and efficacy information. Overall, there is a lack of evidence-based strategies for pediatric trial recruitment and retention. We will address this gap, utilizing data from a real-world pediatric clinical trial. This supplement supports the success of future pediatric clinical trials by addressing three key areas. We will 1) compare the efficacy of three strategies for recruiting and retaining participants in pediatric clinical trials; 2) determine whether the efficacy of recruitment and retention strategies differs between Black/ African American children and White children; and 3) provide Ms. Ikerionwu with training and foundational knowledge in the conduct of pediatric clinical trials that supports her development as an aspiring physician-researcher conducting pediatric clinical trials. The objective of this application is to determine the most effective and efficient methods for enrolling and retaining children in a double blinded, placebo controlled clinical trial. We hypothesize that there will be superior strategies identified for pediatric participant recruitment and retention. This will be accomplished by Aim I to determine the most effective recruitment strategies for pediatric clinical trials. We hypothesize that differences exist in recruitment effectiveness between enrollment by face-to-face contact in clinical settings, tele-recruitment based on patients lists, and community partnership strategies and that face-to-face recruitment strategies within the clinical settings will have increased efficiency and effectiveness. In Aim II, we will determine barriers to retention in pediatric clinical trials. In both aims we will also determine differences in effective recruitment and retention strategies between children who identify as White/ Caucasian in comparison to children who identify as Black/ African American. The proposed work in this application will provide foundational knowledge and skills in the conduct of pediatric clinical trials and clinical research by a junior investigator from an under-represented background, thus leading to a future career as a physician scientist. The work will also provide evidenced based knowledge to inform successful recruitment and retention in diverse pediatric clinical trials.

项目概要家长奖的首要目标是验证激光多普勒组胺离子电渗疗法 (HILD) 用于预测哮喘儿童对左西替利嗪治疗反应的流量生物标志物。这研究补充将支持家长奖的两个目标，即家长奖的主要研究活动包括对儿童进行大型临床试验。完成安全高效的临床试验儿童对于儿科药物开发至关重要。超过 20% 的儿科临床试验失败或终止由于缺乏入组且超过 30% 的试验未达到最初确定的招募目标，因此提早进行。障碍必须检查并解决儿科临床试验中参与者的招募和保留问题，以增加成功试验的数量以及重要儿科剂量、安全性和有效性的传播信息。总体而言，儿科试验招募和保留缺乏循证策略。我们将利用真实儿科临床试验的数据来弥补这一差距。本补充支持通过解决三个关键领域来确保未来儿科临床试验的成功。我们将1）比较三种的功效招募和保留儿科临床试验参与者的策略； 2）判断是否有功效黑人/非裔美国儿童和白人儿童的招募和保留策略有所不同；和 3) 为 Ikerionwu 女士提供进行儿科临床试验的培训和基础知识，支持她发展成为一名有抱负的医生兼研究员，进行儿科临床试验。这该应用程序的目的是确定最有效和最高效的方法进行注册和让儿童参与双盲、安慰剂对照的临床试验。我们假设将会有确定了儿科参与者招募和保留的优越策略。这将通过以下方式完成我的目标是确定儿科临床试验最有效的招募策略。我们假设临床中面对面接触的招募效果存在差异设置、基于患者名单的电话招募、社区伙伴关系策略以及面对面的临床环境中的招募策略将提高效率和有效性。在目标二中，我们将确定儿科临床试验中保留的障碍。在这两个目标中，我们还将确定白人/白人儿童之间有效招募和保留策略的差异与被认定为黑人/非裔美国人的儿童相比。本申请中拟议的工作将提供进行儿科临床试验和临床研究的基础知识和技能来自代表性不足的背景的初级研究员，从而导致未来的职业生涯成为一名医生科学家。这项工作还将提供基于证据的知识，为成功的招聘和保留提供信息在各种儿科临床试验中。