NCT04806451

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

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 103 (actual)
• Sponsor: Neurocrine Biosciences · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

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).

Conditions

• Congenital Adrenal Hyperplasia

Interventions

Type	Name	Description
DRUG	Crinecerfont	CRF type 1 receptor antagonist
DRUG	Placebo	Non-active dosage form

Timeline

• Start date: 2021-06-25
• Primary completion: 2023-03-10
• Completion: 2027-08-01
• First posted: 2021-03-19
• Last updated: 2025-02-05
• Results posted: 2025-02-05

Locations

46 sites across 10 countries: United States, Belgium, Canada, France, Germany, Greece, Italy, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study