Defining inclusion criteria and endpoints for clinical trials: a prospective cross-sectional study in CRB1-associated retinal dystrophies.
Mays TalibMary J van SchooneveldJan WijnholdsMaria M van GenderenNicoline E Schalij-DelfosHerman E TalsmaRalph J FlorijnJacoline B Ten BrinkFrans P M CremersAlberta A H J ThiadensL Ingeborgh van den BornCarel B HoyngMagda A Meester-SmoorArthur A BergenCamiel J F BoonPublished in: Acta ophthalmologica (2021)
Despite the generally severe course of CRB1-RDs, symptom onset and central visual function are variable, even at advanced ages. Phenotypes may vary within the same family. Imaging and functional studies in a prospective longitudinal setting should clarify which endpoints may be most appropriate in a clinical trial.