N Engl J Med

ENDO 2024: Novel agent shows promise in congenital adrenal hyperplasia trial

June 7, 2024

Among adults with CAH, the use of crinecerfont resulted in a greater decrease from baseline in the mean daily glucocorticoid dose, including a reduction to the physiologic range, compared with placebo.

• In the phase 3 CAHtalyst trial, 182 adults with CAH were randomly assigned in a 2:1 ratio to receive crinecerfont (n=122) or placebo (n=60) for 24 weeks. Glucocorticoid treatment was maintained at a stable level for 4 weeks to evaluate androstenedione levels, followed by glucocorticoid dose reduction and optimization over 20 weeks to achieve the lowest glucocorticoid dose that maintained androstenedione control (≤120% of the baseline value or within the reference range).
• Mean glucocorticoid dose at baseline was 17.6 mg/m^2 of body-surface area per day of hydrocortisone equivalents; mean androstenedione level was elevated at 620 ng/dL.
• At week 24, the change in the glucocorticoid dose (with androstenedione control) was -27.3% in the crinecerfont group and -10.3% in the placebo group (P<0.001). A physiologic glucocorticoid dose (with androstenedione control) was reported in 63% of the patients in the crinecerfont group and in 18% in the placebo group (P<0.001).
• At week 4, androstenedione levels decreased with crinecerfont (-299 ng/dL) but increased with placebo (45.5 ng/dL) (P<0.001).

Source:

Auchus RJ; CAHtalyst Adult Trial Investigators. (2024, June 1). N Engl J Med. Phase 3 Trial of Crinecerfont in Adult Congenital Adrenal Hyperplasia. https://pubmed.ncbi.nlm.nih.gov/38828955/