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Adrenal (Excluding Mineralocorticoids)

SAT-465 - Long-Term Crinecerfont Treatment Reduced ACTH and 17-Hydroxyprogesterone and Improved Clinical Outcomes in Children and Adolescents with Classic Congenital Adrenal Hyperplasia: 2-Year Results from Cahtalyst™ Pediatric

Saturday, June 13, 2026
9:30 AM - 4:30 PM CT
Location: ENDOExpo; Poster Floor
Disclosure(s):
Selma Feldman Witchel, MD: Neurocrine Biosciences: Consultant (); The Endocrine Society: Associate Editor, JES (), Editorial Board Member, JCEM Case Reports (); UpToDate: Section contributor ()
Patricia Y. Fechner, MD: No relevant disclosure to display


Background: Crinecerfont is a first-in-class corticotropin releasing factor type 1 receptor (CRF1) antagonist that is FDA-approved as an adjunct to glucocorticoid (GC) replacement to control androgens in patients with classic congenital adrenal hyperplasia (CAH) 4 years of age and older, including both the salt-wasting and simple virilizing forms. During the 6-month double-blind placebo-controlled (DBPC) period of the phase 3 CAHtalyst™ Pediatric trial (NCT04806451), crinecerfont substantially reduced excess adrenocorticotropic hormone (ACTH) and androgens, enabling subsequent reductions in GC doses, that were previously demonstrated to be sustained through 1 year of treatment.

Objective: To evaluate changes in ACTH, 17-hydroxyprogesterone (17-OHP), and clinical outcomes related to excess androgens in pediatric patients with CAH who received up to 2 years of crinecerfont.

Methods: Participants who completed 1 year of treatment in the DBPC and open-label periods of CAHtalyst™ Pediatric (NCT04806451) continued taking crinecerfont (25, 50, or 100 mg BID based on weight) in an ongoing open-label extension. At Month 12 and Month 24, mean changes from baseline in ACTH and 17-OHP were assessed. Outcomes related to excess androgens were analyzed at Month 12 and Month 24.

Results: At baseline, mean age (range) was 12 (4-17) years; mean GC dose was 16.4 mg/m2/d. Mean ACTH (329 pg/mL) and 17-OHP (8682 ng/dL) were elevated. At Month 12, mean ACTH and 17-OHP were reduced from baseline with crinecerfont (ACTH, -118 pg/mL; 17-OHP, -1698 ng/dL), with further reductions observed at Month 24 (ACTH, -157 pg/mL; 17-OHP, -1924 ng/dL), despite lower mean GC doses (Month 12, 13.5 mg/m2/d; Month 24, 13.2 mg/m2/d). Among pubertal male participants with androstenedione-to-testosterone ratio (A4/T) ≥0.5 at baseline, 31% (9/29) achieved A4/T < 0.5 at Month 12; 36% (9/25) achieved A4/T < 0.5 at Month 24. In 58 participants with a baseline acne visual analog scale (VAS; range 0-100) score >0 (mean score: 25.4), scores were reduced from baseline at Month 12 (-6.5), with further reductions observed at Month 24 (-11.0). In 29 female participants with a baseline hirsutism VAS score >0 (mean score 27.3), scores remained stable despite lower GC doses and continued pubertal stage progression (Month 12, -6.3; Month 24, +2.4). Crinecerfont was well tolerated; no new safety signals were observed.

Conclusion: In pediatric patients with CAH, crinecerfont enabled substantial reductions in ACTH and 17-OHP that were sustained for up to 2 years, despite reductions in GC doses. Moreover, improvements were observed in outcomes related to excess androgens (e.g., acne, hirsutism) among participants with these conditions at baseline. Crinecerfont addresses both treatment goals of decreasing androgens and reducing exposure to chronic supraphysiologic GC doses in patients with CAH, which can translate to improved clinical outcomes.

*Unless otherwise noted, all abstracts presented at ENDO must not be released to the press or the public until the date and time of presentation. For oral presentations, the abstracts are embargoed until the session begins. The Endocrine Society reserves the right to lift the embargo on specific abstracts that are selected for promotion prior to or during ENDO.*