The NINDS intends to form a network to perform pilot clinical trials for neurological diseases. The purpose of this network is twofold: 1) to facilitate the early clinical testing of promising compounds for different neurological diseases. The focus here is on the timely translation of pre-clinical results into clinical studies and the efficient conduct of these clinical studies. Pilot clinical trials are defined as studies required to gather the information needed to conduct a definitive efficacy study and fulfill the relevant regulatory requirements. Thus, the term pilot clinical trial includes Phase I and II studies, although it is anticipated that the majority of studies would be Phase II. The interventions studied may be new compounds or compounds that are already FDA approved for other indications. Pilot trials include but are not limited to: studies to refine the intervention strategy - dosage, duration, delivery system, pharmacokinetics and dynamics; studies to define and refine the target population; and collection of preliminary data for establishing measures of efficacy and safety. 2) to build and operate a network that will serve multiple different neurological diseases through shared resources and facilitate the study of rare neurological diseases by reaching out to a large number of sites serving different patient populations.
