Phase 3 Study of Atumelnant Versus Placebo in Adults With Classic Congenital Adrenal Hyperplasia Due to 21-OHD

What this Trial Is About

Researchers are investigating atumelnant in adults aged 18 to under 75 years with classic congenital adrenal hyperplasia CAH caused by 21-hydroxylase deficiency 21-OHD. This Phase 3, global, randomized, double-blind, placebo-controlled study aims to assess the effectiveness, safety, pharmacokinetics, and pharmacodynamics of atumelnant in participants who have been on stable glucocorticoid GC therapy for at least two months. Eligible participants enter a screening period lasting 3 to 6 weeks to confirm suitability for the study. After screening, participants are randomly assigned in a 21 ratio to receive either 80 mg of atumelnant orally once daily, with a possible dose increase to 120 mg at Week 20, or a matching placebo once daily for 32 weeks. The study compares these two groups to evaluate the effects of atumelnant alongside usual GC treatment. During the study, participants will have regular assessments including blood tests to measure hormone levels such as androstenedione A4 and 17-hydroxyprogesterone 17-OHP at baseline and specified weeks. Researchers will monitor participants hormone control, GC doses, safety, and adherence. The main outcome is the proportion of participants achieving morning post-GC A4 levels within the normal range while on physiologic GC replacement at Week 32. The total participation duration includes the screening and 32-week treatment periods.

A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia