A Phase 2, Open-Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability, and Pharmacodynamics of Crinecerfont in Pediatric Subjects 0 to <2 Years of Age With Congenital Adrenal Hyperplasia
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
The main objective for this study is to evaluate the pharmacokinetics (PK) of crinecerfont in pediatric participants 0 to \<2 years of age with congenital adrenal hyperplasia (CAH).
Eligibility
Participation Requirements
Sex: All
Maximum Age: 1
Healthy Volunteers: f
View:
• Be a female or male between 0 to \<2 years of age at screening.
• Have a medically confirmed diagnosis of classic CAH (salt wasting or simple virilizing) due to 21-hydroxylase deficiency (21-OHD).
• Be on a clinically stable regimen of hydrocortisone (and fludrocortisone, if applicable) treatment.
Locations
Other Locations
Germany
Neurocrine Clinical Site
RECRUITING
Berlin
Neurocrine Clinical Site
RECRUITING
Heidelberg
Contact Information
Primary
Neurocrine Medical Information Call Center
medinfo@neurocrine.com
1-877-641-3461
Time Frame
Start Date: 2025-09-30
Estimated Completion Date: 2029-10-04
Participants
Target number of participants: 6
Treatments
Experimental: Crinecerfont
Participants with CAH will receive crinecerfont during an initial 14-day treatment period, followed by an optional 36-month open-label extension (OLE).
Related Therapeutic Areas
Sponsors
Leads: Neurocrine Biosciences