This clinical trial is focused on studying a condition known as Classic Congenital Adrenal Hyperplasia (CAH), which is a genetic disorder affecting the adrenal glands. The study is evaluating a treatment called Crinecerfont, also known by its code name NBI-74788. Crinecerfont is being tested in two forms: an oral solution and a capsule. The purpose of the study is to assess the safety and effectiveness of Crinecerfont in reducing certain hormone levels in children with CAH.
Participants in the study will be randomly assigned to receive either Crinecerfont or a placebo, which is a substance with no active medication. The study will begin with a period where neither the participants nor the researchers know who is receiving the actual medication or the placebo. This is known as a double-blind study. After this initial phase, all participants will have the opportunity to receive Crinecerfont in an open-label phase, where everyone knows they are receiving the active treatment.
The study will last for up to 52 weeks, during which time participants will have regular visits to the study center to monitor their health and hormone levels. The main goal is to see if Crinecerfont can safely lower adrenal steroid levels while participants continue their usual treatment for CAH. This study is important for understanding how Crinecerfont can help manage CAH in children and improve their quality of life.



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