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Crinecerfont, a CRF1 Receptor Antagonist, Lowers Adrenal Androgens in Adolescents With Congenital Adrenal Hyperplasia.
Newfield, Ron S; Sarafoglou, Kyriakie; Fechner, Patricia Y; Nokoff, Natalie J; Auchus, Richard J; Vogiatzi, Maria G; Jeha, George S; Giri, Nagdeep; Roberts, Eiry; Sturgeon, Julia; Chan, Jean L; Farber, Robert H.
Afiliação
  • Newfield RS; Pediatric Endocrinology, University of California San Diego and Rady Children's Hospital, San Diego, CA 92123, USA.
  • Sarafoglou K; Department of Pediatrics, Division of Endocrinology, University of Minnesota Medical School, Minneapolis, MN 55454, USA.
  • Fechner PY; Departments of Pediatrics, Division of Pediatric Endocrinology, University of Washington School of Medicine, Seattle Children's, Seattle, WA 98105, USA.
  • Nokoff NJ; Department of Pediatric Endocrinology, University of Colorado Anschutz Medical Campus, Children's Hospital Colorado, Aurora, CO 80045, USA.
  • Auchus RJ; Departments of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology and Diabetes, University of Michigan Medical School, Ann Arbor, MI 48109, USA.
  • Vogiatzi MG; Division of Endocrinology and Diabetes, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
  • Jeha GS; Neurocrine Biosciences, Inc., San Diego, CA 92130, USA.
  • Giri N; Neurocrine Biosciences, Inc., San Diego, CA 92130, USA.
  • Roberts E; Neurocrine Biosciences, Inc., San Diego, CA 92130, USA.
  • Sturgeon J; Neurocrine Biosciences, Inc., San Diego, CA 92130, USA.
  • Chan JL; Neurocrine Biosciences, Inc., San Diego, CA 92130, USA.
  • Farber RH; Neurocrine Biosciences, Inc., San Diego, CA 92130, USA.
J Clin Endocrinol Metab ; 108(11): 2871-2878, 2023 10 18.
Article em En | MEDLINE | ID: mdl-37216921
ABSTRACT
CONTEXT Crinecerfont, a corticotropin-releasing factor type 1 receptor antagonist, has been shown to reduce elevated adrenal androgens and precursors in adults with congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD), a rare autosomal recessive disorder characterized by cortisol deficiency and androgen excess due to elevated adrenocorticotropin.

OBJECTIVE:

To evaluate the safety, tolerability, and efficacy of crinecerfont in adolescents with 21OHD CAH.

METHODS:

This was an open-label, phase 2 study (NCT04045145) at 4 centers in the United States. Participants were males and females, 14 to 17 years of age, with classic 21OHD CAH. Crinecerfont was administered orally (50 mg twice daily) for 14 consecutive days with morning and evening meals. The main outcomes were change from baseline to day 14 in circulating concentrations of ACTH, 17-hydroxyprogesterone (17OHP), androstenedione, and testosterone.

RESULTS:

8 participants (3 males, 5 females) were enrolled; median age was 15 years and 88% were Caucasian/White. After 14 days of crinecerfont, median percent reductions from baseline to day 14 were as follows ACTH, -57%; 17OHP, -69%; and androstenedione, -58%. In female participants, 60% (3/5) had ≥50% reduction from baseline in testosterone.

CONCLUSION:

Adolescents with classic 21OHD CAH had substantial reductions in adrenal androgens and androgen precursors after 14 days of oral crinecerfont administration. These results are consistent with a study of crinecerfont in adults with classic 21OHD CAH.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Hiperplasia Suprarrenal Congênita / Androgênios Limite: Adolescent / Adult / Female / Humans / Male Idioma: En Revista: J Clin Endocrinol Metab Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Hiperplasia Suprarrenal Congênita / Androgênios Limite: Adolescent / Adult / Female / Humans / Male Idioma: En Revista: J Clin Endocrinol Metab Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos