Neurocrine Launches 24-Week Phase 2 Trial of Crinecerfont in Under-4 CAH
NBIX•Neurocrine Biosciences has launched a 24-week, open-label Phase 2 trial of crinecerfont in 20 pediatric CAH patients aged three months to under four years to assess safety and tolerability under an FDA pediatric written request. The study includes PK/PD evaluations and targets supplemental U.S. label expansion.
1. Phase 2 Trial Initiation
Neurocrine Biosciences has commenced a Phase 2 clinical study of crinecerfont in pediatric patients aged three months to under four years diagnosed with classic congenital adrenal hyperplasia (CAH). This marks the first trial evaluating the agent in children below four years, following its adult and older pediatric approval as an adjunct to glucocorticoids.
2. Study Design and Objectives
The open-label, single-arm study spans 24 weeks and will enroll 20 participants to evaluate safety and tolerability, with secondary objectives assessing pharmacokinetics and pharmacodynamic effects on key hormone biomarkers. Crinecerfont’s mechanism targets ACTH reduction to control adrenal androgen excess without escalating glucocorticoid doses.
3. Regulatory and Global Efforts
This U.S. trial is conducted under an FDA Pediatric Written Request aimed at supporting a supplemental New Drug Application for indication expansion below four years of age. Separately, Neurocrine achieved target enrollment in a Phase 2 EU study of crinecerfont in newborns up to two years old, reinforcing its global pediatric development strategy.
