ESPE Yearbook of Paediatric Endocrinology

ESPE Yearbook of Paediatric Endocrinology

ISSN 1662-4009 (online)

Published by BioScientifica

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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...
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Published by BioScientifica

ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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