Towards Improved Clinical Trials in Cerebral Palsy

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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.