Phase 0 and window of opportunity clinical trial design in neuro-oncology: a RANO review.

ABSTRACT: Glioblastoma is a devastating disease with poor prognosis. Few effective chemotherapeutics are currently available, and much effort has been expended to identify new drugs capable of slowing tumor progression. The phase 0 trial design was developed to facilitate early identification of promising agents for cancer that should undergo accelerated approval. This design features an early in-human study that enrolls a small number of patients who receive subtherapeutic doses of medication with the goals of describing pharmacokinetics through drug blood level measurements and determining intratumoral concentrations of the investigational compound as well as pharmacodynamics by studying the biochemical and physiological effects of drugs. In neuro-oncology, however, the presence of the blood-brain

SUBMITTER: Vogelbaum MA 

PROVIDER: S-EPMC7690357 | biostudies-literature | 2020 Nov

REPOSITORIES: biostudies-literature