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

• Status: Recruiting
• Trial end date: 2024-04-01
• Target enrollment: 0
• Participant gender: All

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 patients 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 treatment with crinecerfont. Duration of participation is approximately 14 months.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Neurocrine Biosciences

Criteria

Inclusion Criteria:

• Be willing and able to adhere to the study procedures, including all requirements at the study center, and return for the follow-up visit.

• Have a medically confirmed diagnosis of 21-hydroxylase deficiency CAH.

• Be on a stable regimen of steroidal treatment for CAH.

• Have elevated androgen levels.

• Patients of childbearing potential must be abstinent or agree to use appropriate birth control during the study.

Exclusion Criteria:

• Have a diagnosis of any of the other forms of classic CAH.

• Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.

• Have a clinically significant unstable medical condition or chronic disease other than CAH.

• Have a history of cancer unless considered to be cured.

• Have a known history of clinically significant arrhythmia or abnormalities on ECG.

• Have a known hypersensitivity to any corticotropin-releasing hormone antagonist.

• Have received an investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.

• Have current substance dependence or substance (drug) or alcohol abuse.

• Have had a significant blood loss or donated blood or blood products within 8 weeks prior to the study.