Endocrine Abstracts

More than 90% of classic congenital adrenal hyperplasia (CAH) patients have defects in the cytochrome P450 enzyme steroid 21-hydroxylase, resulting in the inability to produce cortisol as well as the overproduction of androgens and androgen precursors such as 17-hydroxyprogesterone (17-OHP). Management of classic CAH can be challenging since patients are often unable to adequately balance the supraphysiologic doses of exogenous glucocorticoids required to suppress excess andro...

Background: Prader-Willi syndrome (PWS) is a rare disease characterized by hyperphagia and abnormal food-related behaviors that contribute to severe morbidity and early mortality and to a significant burden on patients and caregivers. There is no approved treatment for hyperphagia in PWS. Patients with PWS have increased circulating levels of the orexigenic hormone acylated ghrelin (AG) with a relative deficit of unacylated ghrelin (UAG). Livoletide (AZP-531) is a first-in-cla...

Background: Hot flushes (HF) affect 70% of menopausal women and can be debilitating. Oestrogen administration is effective but not without risk. Neurokinin B signalling is increased in menopausal women, and is likely critical in the aetiology of their HF. We therefore hypothesised that a neurokinin 3 receptor (NK3R) antagonist could attenuate menopausal flushing.Design: This single-centre, phase 2, randomised, double-blind, placebo-controlled, crossover ...

Background: Hot flushes (HF) affect 70% of menopausal women and can be debilitating. Oestrogen administration is effective but not without risk. Neurokinin B signalling is increased in menopausal women, and is likely critical in the aetiology of their HF. We therefore hypothesised that a neurokinin 3 receptor (NK3R) antagonist could attenuate menopausal flushing.Design: This single-centre, phase 2, randomised, double-blind, placebo-controlled, crossover ...

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...