Neurocrine Biosciences Inc
released topline data from the Phase 3 CAHtalyst Pediatric Study of crinecerfont in children and adolescents with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency.
CAH is an inherited condition that affects the body's adrenal glands, the cone-shaped organs on top of the kidneys. In a person with CAH, the adrenal glands are very large and cannot produce certain chemicals, including cortisol, which helps protect the body during stress or illness and helps the body regulate the amount of sugar in the blood.
The Phase 3 Pediatric study met its primary endpoint, demonstrating that treatment with crinecerfont resulted in a statistically significant decrease in serum androstenedione from baseline at Week 4 versus placebo following a glucocorticoid (GC) stable period.
Approximately 30% of participants receiving crinecerfont achieved a reduction to a physiologic GC dose while maintaining androgen control compared to 0% of participants receiving a placebo.
The study also met the other key secondary endpoint, demonstrating a statistically significant decrease in serum 17-hydroxyprogesterone from baseline at Week 4 versus placebo.
Crinecerfont was generally well tolerated.
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