Endocrine Abstracts

Introduction: Classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD) is a rare autosomal recessive disorder characterized by deficiency of cortisol and oftentimes aldosterone, with elevated adrenocorticotropic hormone (ACTH) and steroid precursors that are shunted toward excess androgen production. A phase 2 study of adults with classic 21OHD demonstrated that crinecerfont–an oral, non-steroidal, selective corticotropin-releasing factor type...

Objective: To describe the baseline characteristics of individuals enrolled in CAHtalyst Pediatric (NCT04806451), a randomized, double-blind, placebo-controlled, Phase 3 study evaluating the safety and efficacy of crinecerfont (a corticotropin-releasing factor type 1 receptor [CRF1] antagonist) in children and adolescents with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (21-OHD CAH).Methods: Key eligibility criteria...