Study of a corticotropin releasing factor-1 receptor antagonist for the treatment of congenital adrenal hyperplasia

Eunice Kennedy Shriver National Institute Of Child Health & Human Development

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Abstract

Excess glucocorticoid therapy is often needed to control the overproduction of adrenal androgens in congenital adrenal hyperplasia. High-affinity and selective small-molecule antagonists of CRH type 1 receptors in the pituitary gland are in development and might allow for lower glucocorticoid therapy. The mechanism of action has been validated in a previous proof-of-concept study and has been shown to be safe in Phase 1 clinical studies in healthy volunteers and Phase 2 dose finding studies of patients with CAH. Two Phase 2 studies of a CRH type 1 receptor antagonist in patients with CAH were completed this year. In both studies, patients with poor disease control at baseline had reductions in biomarkers; while patients with good disease control at baseline showed only minor changes. A randomized, double-blind, placebo-controlled study is planned which will explore the safety and efficacy of CRH type 1 receptor antagonist. This study represents the investigation of a novel therapy in the treatment of congenital adrenal hyperplasia.