Study of Atumelnant Safety and Effectiveness in Children With Classic Congenital Adrenal Hyperplasia Including a Long-Term Treatment Extension

What this Trial Is About

Researchers are evaluating the safety, effectiveness, and how the body processes and responds to atumelnant treatment in children with classic congenital adrenal hyperplasia CAH. This Phase 23 study includes a double-blind, randomized portion and an open-label extension to better understand treatment effects in pediatric participants aged 1 to under 18 years. The study is designed with multiple parts, including sequential cohorts based on age groups to carefully monitor safety and outcomes. Participants will receive atumelnant tablets once daily by mouth with doses adjusted based on their weight. The study is divided into three parts Part A involves open-label, semi-sequential cohorts for children aged 12 to under 18 and later for those aged 1 to 11 Part B is a randomized, double-blind, placebo-controlled phase evaluating active treatment versus placebo and Part C is an open-label extension where participants from Parts A and B may continue treatment. Safety reviews guide progression between cohorts. Throughout the study, participants will have their hormone levels monitored, including morning serum androstenedione and 17-hydroxyprogesterone, along with blood concentration measurements of atumelnant. The trial tracks changes in glucocorticoid doses and hormone levels over time up to 260 weeks in the extension phase. The study also assesses treatment compliance and overall safety, with participation lasting from initial screening through extended follow-up.

A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)