DESCRIPTION (provided by applicant): The Children's National Medical Center (CNMC) will utilize its extensive experience in neuroscience clinical trial development, its proven ability to accrue patients on neurologic clinical trials, its large patient base, and its clinical trial infrastructure to participate in the Network of Excellence in Neuroscience Clinical Trials (NEXT). CNMC will collaborate with other investigators within NEXT, as it has done in other neurologic clinical trials consortia over the past decade, to develop and ensure timely completion of biologically based translational/clinical research studies in both children and adults. CNMC will leverage synergistic support for the NEXT consortium through a series of pre-existing NIH- and DOD-funded clinical infrastructure grants and programs, including the only NIH CTSA hosted site within a children's hospital. CNMC also has highly developed core facilities, including third-generation sequencing and molecular diagnostic capabilities, biomarker discovery and testing facilities, and pharmacokinetic/pharmacodynamic experience, which will be available for CNMC activities within NEXT. CNMC has a strong leadership team, led by Roger J. Packer, MD (PI) and Eric Hoffman, PhD, who are experienced clinical trialists in neuroscience research and have not only led clinical trials, but have been instrumental in the development of national and international clinical trial consortia (including those for neuromuscular diseases). A host of experienced child neurologists, neuro-intensivists, clinical trialists, molecular geneticists, expets in pharmacokinetics and pharmacodynamics, and neurogeneticists are in place at CNMC to participate in trials opened through NEXT. The institution has a large patient base in a multitude of different neurologic diseases from which to accrue patients on NEXT studies. In addition to the over 18,000 unique patients with neurologic conditions seen at CNMC each year, CNMC works closely with multiple foundations/support groups, which have pledged to support NEXT efforts and refer patients to CNMC for open trials. CNMC'S Clinical Research Management Office is designed specifically to support and facilitate clinical trial completion. CNMC already has mechanisms in place through the Institutional Review Board, including agreement to participate in a federated IRB, to facilitate trial approval.

The inclusion of CNMC within NEXT will provide NEXT with a site that has specialized expertise in diverse areas of neuroscience and can accrue significant numbers of patients. It will provide NEXT with an excellent site to carry out clinical trials in th DC metropolitan region, as well as providing NEXT with a pediatrics-focused resource experienced in developing neurologic consortia. CNMC also has highly evolved genetic and biomarker-discovery core facilities to support NEXT studies.