Phase 3 Trial of Crinecerfont in Adult Congenital Adrenal Hyperplasia

This phase 3 trial evaluated the efficacy of crinecerfont, an oral corticotropin releasing factor type 1 receptor antagonist, in adults with congenital adrenal hyperplasia (CAH) due to 21 hydroxylase deficiency. The study involved 182 patients randomly assigned to crinecerfont or placebo for 24 weeks. The primary endpoint was the percent change in daily glucocorticoid dose while maintaining androstenedione control. Results showed a significant reduction in glucocorticoid dose with crinecerfont (27.3%) compared to placebo (10.3%), with 63% of crinecerfont treated patients achieving a physiologic glucocorticoid dose versus 18% in the placebo group. Crinecerfont also significantly lowered androstenedione levels. The therapy demonstrated a favorable safety profile, supporting its potential as a glucocorticoid-sparing treatment for CAH.

This phase 3 trial evaluated the efficacy of crinecerfont, an oral corticotropin releasing factor type 1 receptor antagonist, in adults with congenital adrenal hyperplasia (CAH) due to 21 hydroxylase deficiency. The study involved 182 patients randomly assigned to crinecerfont or placebo for 24 weeks. The primary endpoint was the percent change in daily glucocorticoid dose while maintaining androstenedione control. Results showed a significant reduction in glucocorticoid dose with crinecerfont (27.3%) compared to placebo (10.3%), with 63% of crinecerfont treated patients achieving a physiologic glucocorticoid dose versus 18% in the placebo group. Crinecerfont also significantly lowered androstenedione levels. The therapy demonstrated a favorable safety profile, supporting its potential as a glucocorticoid-sparing treatment for CAH.