ey0021.8-11 | Clinical Trials – New Treatments | ESPEYB21
K Sarafoglou
, MS Kim
, M Lodish
, EI Felner
, L Martinerie
, NJ Nokoff
, M Clemente
, PY Fechner
, MG Vogiatzi
, PW Speiser
, RJ Auchus
, GBG Rosales
, E Roberts
, GS Jeha
, RH Farber
, JL Chan
, Investigators Cahtalyst Pediatric Trial
Brief Summary: This phase 3, multinational, randomized clinical trial (CAHtalyst, NCT04806451) in pediatric patients with CAH, evaluated the efficacy of crinecerfont to improve androgen control and enable GC dose reduction to a physiological range.Comment: Congenital adrenal hyperplasia (CAH) comprises several rare autosomal recessive conditions resulting in disordered adrenal steroidogenesis. Pathogenic variants in the CYP21A2 gene encoding ste...