Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old

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).

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Congenital Adrenal Hyperplasia Adrenal Gland Diseases Adrenal Hyperplasia, Congenital Adrenogenital Syndrome Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disorders of Sex Development Endocrine System Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Gonadal Disorders Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Steroid Metabolism, Inborn Errors Urogenital Abnormalities Urogenital Diseases

Age range

0 year–23 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Neurocrine Clinical Site, Berlin, Germany

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

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.

Key Exclusion Criteria:

Have a known or suspected diagnosis of any of the other forms of classic CAH.
Have any condition besides CAH that requires chronic daily therapy with orally administered steroids.
Have any other clinically significant medical condition or chronic disease.

Note: Other protocol-defined inclusion and exclusion criteria may apply.

Treatment and study plan

Crinecerfont

Drug

Oral solution

Primary outcomes

Plasma Concentration of Crinecerfont

Time frame: Days 7 and 15

Secondary outcomes

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Time frame: Up to Month 37

Sponsors and collaborators

Lead sponsor

Neurocrine Biosciences

Industry

Registry information

Official study title

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

Important dates

Study start: 2025
Primary completion: 2029
Study completion: 2029
First posted: Sep 23, 2025
Registry last updated: Jun 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Active, Not Recruiting