The following is a summary of “Crinecerfont, a CRF1 Receptor Antagonist, Lowers Adrenal Androgens in Adolescents With Congenital Adrenal Hyperplasia,” published in the November 2023 issue of Endocrinology by Newfield, et al.

Crinecerfont, a corticotropin-releasing factor type 1 receptor antagonist, has effectively reduced elevated adrenal androgens in adults with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD). CAH is a rare autosomal recessive disorder characterized by cortisol deficiency and androgen excess. For a study, researchers sought to assess the safety, tolerability, and efficacy of crinecerfont in adolescents with 21OHD CAH.

The open-label phase 2 study was conducted at four centers in the United States. Male and female participants, aged 14 to 17 years, with classic 21OHD CAH were enrolled. Crinecerfont was orally administered at 50 mg twice daily for 14 consecutive days, with doses taken during morning and evening meals. The primary outcomes included changes from baseline to day 14 in circulating concentrations of ACTH, 17-hydroxyprogesterone (17OHP), androstenedione, and testosterone.

Eight participants (3 males, 5 females) were enrolled, with a median age of 15 years, and 88% identified as Caucasian/White. Following 14 days of crinecerfont administration, the median percent reductions from baseline to day 14 were as follows: ACTH, -57%; 17OHP, -69%; and androstenedione, -58%. Among female participants, 60% (3/5) experienced a ≥50% reduction from baseline testosterone.

Adolescents with classic 21OHD CAH demonstrated significant reductions in adrenal androgens and androgen precursors after 14 days of oral crinecerfont administration. The findings aligned with previous studies in adults with classic 21OHD CAH, supporting the potential efficacy of crinecerfont in this patient population.

Source: academic.oup.com/jcem/article/108/11/2871/7175445