Endocrine Abstracts

Introduction: Classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (21OHD CAH) is a rare genetic disorder resulting in impaired cortisol biosynthesis, increased steroid precursors, and excess androgen production. Inhibition of adrenocorticotropic hormone (ACTH) release via antagonism of the corticotropin-releasing factor-1 (CRF1) receptor could reduce adrenal androgen production, thereby reducing the amount of exogenous glucocorticoids required for androgen ...

Introduction: Corticotropin-releasing factor type 1 (CRF1) receptor antagonists, such as crinecerfont, have recently been investigated for the treatment of classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD), a rare autosomal disease characterized by cortisol deficiency, elevated adrenocorticotropic hormone (ACTH), and excess androgen production. In a study of adults with 21OHD, treatment with crinecerfont for 14 days led to median ...

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...

Objectives: To describe the baseline characteristics of individuals enrolled in CAHtalyst (NCT04490915), 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 adults with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (21-OHD).Methods: The study included adults (age ≥18 years) with cl...

Introduction: Classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (CAH), a genetic condition characterized by cortisol deficiency and excess adrenal androgens, typically requires management with supraphysiological glucocorticoid (GC) doses. This multinational Phase 3 study (CAHtalyst™) evaluated whether crinecerfont, an oral CRF1 antagonist, can reduce GC dose while maintaining androgen control in adults with CAH.Metho...