SUN-454 Rationale and Methods for a Phase II Trial Evaluating Osilodrostat in Pediatric Patients with Cushing's Disease

ABSTRACT: Abstract Background: Cushing’s disease (CD) is a rare disease associated with significant morbidity and mortality, with 2-5 new cases per million per year, of which 10% occur in children (Stratakis CA. Endocrinol Metab Clin North Am 2012;41:793-803). In children, CD presents with a combination of weight gain and slowed linear growth. First-line surgery is the treatment of choice for most patients, with a success rate of 60-98% in pediatric patients if performed by an expert pituitary surgeon (Keil MF. J Clin Endocrinol Metab 2013;98:2667-78). When surgery is not successful, patients require additional pharmacologic interventions, which are currently limited in children. Osilodrostat is a potent oral 11β-hydroxylase inhibitor in clinical development for the treatment of CD. This Phase II, m

SUBMITTER: Savage M 

PROVIDER: S-EPMC6553121 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature