Nevanimibe HCl for the Treatment of Classic CAH

A Multicenter Dose-Titration Open-Label Study of Nevanimibe Hydrochloride for the Treatment of Classic Congenital Adrenal Hyperplasia

This is a multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH). Following a Screening Period of approximately 2-14 weeks, eligible subjects will enter a Baseline Period of approximately 2-8 weeks and then a 16-week Treatment Period. It is anticipated that the overall duration of the study per subject will range from 24-42 weeks.

Inclusion Criteria:

• Documented historical diagnosis of classic CAH due to 21-hydroxylase deficiency and/or historical documentation of elevated 17-OHP
• Chronic glucocorticoid replacement therapy for at least 6 consecutive months prior to screening
• Stable glucocorticoid and mineralocorticoid regimen for at least 4 weeks prior to screening and throughout the treatment period of the study

Exclusion Criteria:

• Nonclassic CAH
• Other causes of adrenal insufficiency
• HIV, hepatitis B, or hepatitis C
• AST or ALT >2x ULN, bilirubin or serum creatinine >1.5x ULN