Classic Congenital Adrenal Hyperplasia

The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).

The purpose of this study is to evaluate the efficacy, safety, PK, and PD of atumelnant in adults with classic CAH due to 21-OHD.

Congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency is a chronic endocrine disorder, often diagnosed in the neonatal period. Severe forms may lead to genital virilization in afected girls and adrenal crises. Management includes lifelong hormone replacement therapy and, historically, early genital surgery, although recent guidelines recommend delaying non-urgent procedures to preserve patient autonomy.This study hypothesizes that pediatric management, particularly early surgical interventions, may influence long-term satisfaction and the expression of regret in adulthood. We will conduct an observational cross-sectional study including adult patients diagnosed with CAH during childhood. Data will be collected using standardized questionnaires assessing medical regret, quality of life, sexual function, and psychological outcomes, together with clinical characteristics and past medical and surgical history.The primary outcome is the prevalence of regret related to pediatric care. Secondary outcomes include quality of life, sexual satisfaction, psychological status, and adaptation of hormonal therapy from adolescence to adulthood. Patient-reported outcomes (regret, quality of life, sexual function, and psychological outcomes) will be assessed in female participants only, male participants will be included for the evaluation of hormonal treatment adaptation.

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of atumelnant (CRN04894).