ISSN 1662-4009 (online)

ey0019.8-8 | Clinical Trials – New Treatments | ESPEYB19

8.8. Crinecerfont lowers elevated hormone markers in adults with 21-hydroxylase deficiency congenital adrenal hyperplasia

AR Auchus , K Sarafoglou , PY Fechner , MG Vogiatzi , EA Imel , SM Davis , N Giri , J Sturgeon , E Roberts , JL Chan , RH Farber

J Clin Endocrinol Metab. 2022; 107(3): 801-812. PMID: 34653252 https://pubmed.ncbi.nlm.nih.gov/34653252/Brief Summary: This clinical trial evaluated the safety and efficacy of crinecerfont, a CRF1R antagonist, in suppressing adrenal androgen secretion in adult patients with classic congenital adrenal hyperplasia (CAH) during a treatment period of 14 days.Class...

ey0021.8-11 | Clinical Trials – New Treatments | ESPEYB21

8.11. Phase 3 Trial of Crinecerfont in Pediatric Congenital Adrenal Hyperplasia

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...