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In 2001, the National Institute of Neurological Disorders and Stroke (NINDS) created the NIH Exploratory Trials in Parkinson Disease (NET-PD) program to evaluate therapies to slow the progression of PD.
The program was created based on the 2000 Parkinson Agenda, which called for neuroprotection clinical trials. Specifically, the agenda called for the initiation of randomized, controlled, clinical trials to test potential neuroprotectants. Expert advice regarding implementation of this initiative was solicited at the Therapeutic Initiatives in Parkinson Disease meeting convened by NINDS on October 13, 2000, which included representatives of several NIH Institutes and national experts in PD and clinical research in neurodegenerative disorders.
NET-PD was sponsored by NINDS and coordinated by scientists at the University of Rochester, New York, and the Division of Biostatistics, School of Public Health at the University of Texas Health Science Center. The program included a statistical coordinating center, a clinical coordinating center, and a network of more than 50 clinical sites in the United States and Canada.
NET-PD investigators and the advisory groups selected 5 compounds (or potential therapies) for study: CoQ10, GPI 1485, minocycline, creatine, and pioglitazone.
Futility trials—which identify compounds unlikely to have therapeutic benefit—were used to narrow the list of candidate compounds for future efficacy trials. Of the 5 compounds evaluated, creatine was shown to have the most promising characteristics as a potential neuroprotective agent. Therefore, NINDS recommended that NET-PD evaluate creatine in a large, long-term trial (called Long-term Study 1 or LS-1) of individuals with early PD.
- NET-PD studies broke new ground in clinical methodology.
- NET-PD developed the futility trial model.
- NET-PD was a new model of clinical trials for NINDS—consisting of small pilot trials followed by a large definitive trial.
- NET-PD successfully completed 5 pilot trials, most within one year.