A clinical research organization at the University of Rochester—the Center for Health + Technology—has received a $2.9 million award from the U.S. Department of Defense. The award will be used to develop new outcomes measures for use in therapeutic studies in a range of chronic diseases, officials say.
These tools are expected to provide insight into therapies during clinical trials. CHeT designs and conducts multicenter clinical trials with collaborators worldwide to advance the development of new therapies.
The study, titled Longitudinal Endpoint Optimization to Provide Assessments of Relevant Drugs, aims to improve clinical trial readiness by developing research instruments for mitochondrial disease, cardiomyopathy, rheumatoid arthritis and dystonia. Currently, there are few effective treatments available for these chronic diseases.
The study is being funded by the Defense Department’s Peer Reviewed Medical Research Program, which was created to enhance well-being and function of military personnel, their families, and civilians while improving U.S. combat readiness and performance, officials say.
“Patient-reported outcomes instruments improve clinical trial efficiency, detect early changes in patient health, focus on the symptomatic areas that are most important to specific populations, ensure the relevance of future treatments to patients, and provide a mechanism for patients to provide direct input into a treatment’s effectiveness,” says Chad Heatwole M.D., principal investigator of the grant, director of CHeT, and a neurologist at the University of Rochester Medical Center.
For 15 years, Heatwole and his team have developed and validated more than 100 disease-specific, patient-reported outcome instruments. Collectively, these instruments are translated and validated in 12 languages and are used worldwide as sensitive and clinically relevant outcomes measures in therapeutic trials, officials say. They cover adult and pediatric populations with neurological conditions, neuromuscular diseases, movement disorders, inflammatory bowel diseases and cancer.
With the LEOPARD study, qualitative patient interviews with military personnel and civilians, data from large cross-sectional studies, factor analysis, expert review, and longitudinal reliability testing will be used to determine important symptoms to patients with these diseases, and to develop and validate four disease-specific instruments. Additionally, the research will identify at-risk demographic subgroups for increased symptomatic burden. It will also pinpoint opportunities for early intervention in high-risk populations.
URMC will partner with the Uniformed Services University and Walter Reed National Military Medical Center, which will serve as a recruitment site for the study.
“The state-of-the-art patient-reported outcomes measures developed through this research will expedite therapy development for military personnel, veterans, civilians, and families affected by these four common and undertreated diseases,” says Heatwole.
URMC cardiologist Jeffrey Alexis M.D., rheumatologist John Looney, M.D., neurologist Ray Dorsey, M.D., and biostatistics professor Brent Johnson along with Walter Reed National Military Medical Center and Uniformed Services University clinical faculty Mark Haigney M.D., Francis O’Connor M.D., and Glen Cook M.D. will collaborate on the study.
CHeT operates five units with expertise in clinical trials coordination, clinical materials services, data modeling and predictive analytics, tech research and innovation, and patient-reported outcome measures. Since its inception, the center has completed more than 130 clinical trials, worked with over 40,000 research participants and has coordinated trials that have led to seven Food and Drug Administration approvals.
The LEOPARD study is likely to add to its wealth of data on patient-reported outcomes measures, a fast-growing area in clinical research and recognized as a valuable tool in the patient-centric approach.