A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH

A Phase 2 Study to Evaluate the Safety, Efficacy and Pharmacokinetics of SPR001 (Tildacerfont) in Children Aged 2 to 17 Years With Congenital Adrenal Hyperplasia (CAH)

An investigation of the safety and efficacy of tildacerfont in participants with CAH.

This is a Phase 2 open-label study with up to 10 cohorts that will evaluate the safety, efficacy, and PK of different tildacerfont dosing regimens potentially up to 200mg QD for 12 weeks in children with classic CAH, and up to 400mg BID for 4 weeks in children and adults with classic CAH.

Inclusion Criteria:

• Male and female subjects aged 2+
• Diagnosis of CAH due to 21-hydroxylase deficiency (OHD) and/or elevated 17- hydroxyprogesterone (OHP) requiring ongoing GC replacement since diagnosis
• Stable dose of GC replacement for at least 1 month prior to screening

Exclusion Criteria:

• History of bilateral adrenalectomy or hypopituitarism
• Clinically significant unstable medical conditions, illness, or chronic diseases
• History of active bleeding disorders