DARRAS, BASIL T
BOSTON CHILDREN'S HOSPITAL
NINDS NEXT: Childrens Hospital Boston Clinical Research Site
Spinal muscular atrophy (SMA)
National Institute of Neurological Disorders and Stroke, clinical research site, Pediatric Hospitals, Boston, Pediatric Neurology
DESCRIPTION (provided by applicant): Barriers and challenges to the conduct of clinical trials are obstacles to the improvement of scientific knowledge and the advancement of diagnosis and treatments for neurologic disorders. The innovative aspects of the NEXT model are aimed at overcoming these barriers. The Department of Neurology at Children’s Hospital Boston has the resources, personnel, patient population, institutional support, research expertise, and dedication that make it the logical choice for a NEXT Clinical Research Site (CRS) focused on pediatric neurology. The Specific Aims of this proposal are: (1) to develop the Children’s Hospital Boston NEXT CRS as a shared infrastructure platform and assign roles for CRS leadership and staff; (2) to identify and recruit eligible patients for NEXT and NINDS trials and establish the extent of available disease-specific patient populations; (3) to submit trial applications for Network review, be named lead CRS on a future NEXT study, and demonstrate experience and expertise in the conduct of clinical research; (4) to collaborate with the NEXT Data Coordinating Center and demonstrate expertise in publication and dissemination of research results; (5) to abide by standardized Network policies and implement and optimize parameters of institutional, divisional, departmental, and individual collaborative agreements; and (6) to train new clinical investigators in skills required for an independent career in clinica research and identify means by which the CRS will maximize co-investigator use of local clinical research educational and support services.
Children’s Hospital Boston NEXT Clinical Research Site will lead advances in pediatric neurology in multicenter clinical research trials, undertaken within standardized parameters that will improve the translation of scientific findings into effective treatments. A future trial of biomarkers in spinal muscular atrophy will guide therapeutic developments and provide a model for NEXT clinical trials in other neurologic disorders.