Phase 3 Trial of Crinecerfont in Pediatric Congenital Adrenal Hyperplasia

This phase 3 trial evaluated the efficacy of crinecerfont, an oral corticotropin releasing factor type 1 receptor antagonist, in pediatric patients with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study involved 103 participants randomly assigned to crinecerfont or placebo for 28 weeks. The primary endpoint was the change in androstenedione levels from baseline to week 4. Results showed a significant reduction in androstenedione levels with crinecerfont (197 ng/dL) compared to an increase with placebo (71 ng/dL) (P<0.001). By week 28, the glucocorticoid dose was reduced by 18.0% in the crinecerfont group while maintaining androstenedione control, whereas the placebo group saw a 5.6% increase. A physiologic glucocorticoid dose was achieved in 30% of crinecerfont treated participants versus none in the placebo group. The therapy demonstrated a favorable safety profile, supporting its potential as a glucocorticoid sparing treatment for pediatric CAH.

This phase 3 trial evaluated the efficacy of crinecerfont, an oral corticotropin releasing factor type 1 receptor antagonist, in pediatric patients with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study involved 103 participants randomly assigned to crinecerfont or placebo for 28 weeks. The primary endpoint was the change in androstenedione levels from baseline to week 4. Results showed a significant reduction in androstenedione levels with crinecerfont (197 ng/dL) compared to an increase with placebo (71 ng/dL) (P<0.001). By week 28, the glucocorticoid dose was reduced by 18.0% in the crinecerfont group while maintaining androstenedione control, whereas the placebo group saw a 5.6% increase. A physiologic glucocorticoid dose was achieved in 30% of crinecerfont treated participants versus none in the placebo group. The therapy demonstrated a favorable safety profile, supporting its potential as a glucocorticoid sparing treatment for pediatric CAH.