Inherited Retinal Degenerative Disease Clinical Trials Network.

摘要

The Foundation Fighting Blindness Clinical Research Institute (FFB CRI) [formerly National Neurovision Research Institute (NNRI)] the clinical arm of the Foundation Fighting Blindness (FFB), established the National Eye Evaluation Research (NEER) Network, composed of collaborative group of five Clinical Treatment and Evaluation Center (CTECs). The intent of this Network was to advance the science of therapeutic and preventive interventions for inherited orphan retinal degenerative diseases and dry age-related macular degeneration (AMD) through the conduct of clinical trials and other clinically relevant research. The scope of research carried out encompassed; (i) a Phase II clinical trial to evaluate the safety and efficacy of new therapeutic and preventive approaches, by repurposing an FDA-approved small molecule drug; (ii) natural history studies to develop standardized criteria to define disease stage, severity and progression; (iii) observational studies to enhance understanding of the natural history of these diseases for different genotypes and phenotypes; and (iv) evaluations of the reliability and validity of different available treatment outcomes measures to determine those that are most appropriate for various genotypes and phenotypes as well as for specific interventions.