Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).

NCT Number	NCT04806451
Lead Sponsor	Neurocrine Biosciences
Conditions	Congenital Adrenal Hyperplasia
Enrollment	103 participants
Start Date	2021-06-25
Primary Completion	2023-03-10 (estimated)
Study Completion	2027-08 (estimated)
Updated on ClinicalTrials.gov	2025-02-05