A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Crinecerfont in Pediatric Participants 3 Months to <4 Years of Age With Classic Congenital Adrenal Hyperplasia

Neurocrine Switzerland GmbH

Summary

The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to \<4 years of age with CAH.

Eligibility

Age range: 0–3 years
Sex: All
Healthy volunteers: No

Detailed criteria

Key Inclusion Criteria: * Have a medically confirmed diagnosis of classic CAH (salt wasting or simple virilizing) due to 21-hydroxylase deficiency (21-OHD) * Have received at least 3 months of hydrocortisone treatment before Day 1, with a stable dose for at least 4 weeks before screening. * Have a body weight of at least 4.5 kilograms (kg) at screening. * Have a newborn screen that is otherwise normal except for elevated 17-OHP or any other abnormality on newborn screen that was cleared upon evaluation by a pediatric specialist. Key Exclusion Criteria: * Have a known or suspected diagnosis…

Interventions

Drug
Crinecerfont
Oral solution

Summary

Eligibility

Interventions

Locations (10)