Towards Improved Clinical Trials in Cerebral Palsy

描述（由申请人提供）：候选人Jilda Vargus-Adams， MD MS，是辛辛那提大学医学院/辛辛那提儿童医院医学中心PM&R和儿科部门的儿科物理医生和助理教授。该奖项将使她在以患者为导向的儿童脑瘫（CP）临床有效性研究方面建立独立的职业生涯，为儿童脑瘫的干预提供临床试验的经验基础。她将通过为期五年的培训、指导和研究计划来实现这一长期目标。候选人将在培训计划中获得四个具体目标：1)扩展她的研究技能，特别关注临床试验，包括实验设计，实际问题，共识形成技术和分析；2)增强她在CP结果测量方面的经验，包括结果测量理论、常用CP仪器的使用和管理以及步态分析；3)通过课程学习和指导，加强科研写作和科研提案能力；4)提高她对负责任的研究行为的理解。候选人将在杰出教师的指导下追求这些职业目标。她的赞助者是一位在临床试验中建立的儿科研究员和成功的职业导师。共同发起人代表与研究和职业发展计划直接相关的临床和研究专业知识。建议的研究计划侧重于完善一组用于CP儿童临床试验的结果测量。候选人建议：1)在ICF框架内进行前瞻性的、逐步的过程，以确定和表征一组CP患者的结果测量指标；2)进行纵向临床研究，以确定一组结果测量指标的有效性、可靠性和响应性；3)设计一项严格的随机对照临床试验，对CP儿童进行干预，并提交资金申请。对CP结果测量的深入了解将有助于未来临床试验和荟萃分析的标准化、实施、解释和效率，并有助于常规临床实践；此外，它将独特地为CP临床试验的候选人的职业生涯做好准备。这项工作将通过告知，首先，更好地设计和实施CP临床试验，然后，改善CP儿童的临床决策，从而改善公共卫生。

DESCRIPTION (provided by applicant): The candidate, Jilda Vargus-Adams, MD MS, is a pediatric physiatrist and Assistant Professor in the Departments of PM&R and Pediatrics at the University of Cincinnati College of Medicine/Cincinnati Children's Hospital Medical Center. This award will establish her independent career in patient-oriented research into clinical effectiveness in childhood cerebral palsy (CP) by preparing her to conduct clinical trials that provide an empirical basis for interventions in childhood CP. She will accomplish this long-term goal through a five-year program of training, mentoring, and research. The candidate will obtain four specific objectives in the training program: 1) expand her research skills with specific attention to clinical trials including experimental design, practical issues, consensus formation techniques, and analysis; 2) enhance her experience in outcome measurement for CP including outcome measurement theory, use and administration of common CP instruments, and gait analysis; 3) strengthen her skills in scientific writing and grant proposals through coursework and mentoring; and 4) improve her understanding of the responsible conduct of research. The candidate will pursue these career objectives under the guidance of distinguished faculty. Her sponsor is an established pediatric researcher in clinical trials and successful career mentor. Co-sponsors represent clinical and research expertise directly relevant to the research and career development plan. The proposed research plan focuses on refining a panel of outcome measures for use in clinical trials with children with CP. The candidate proposes to: 1) conduct a prospective, stepwise process to identify and characterize a panel of outcome measures for patients with CP within an ICF framework, 2) perform a longitudinal clinical study to define the validity, reliability, and responsiveness of a battery of outcome measures, and 3) design a rigorous randomized controlled clinical trial of an intervention for children with CP and submit it for funding. Deeper understanding of CP outcome measures will facilitate the standardization, conduct, interpretation, and efficiency of future clinical trials and meta-analyses, as well as be useful in routine clinical practice; furthermore, it will uniquely prepare the candidate for a career as a clinical trialist in CP. This work will improve public health by informing, first, the better design and implementation of CP clinical trials and, thereafter, improved clinical decision making for children with CP.