Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old

The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to <4 years of age with CAH.

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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

3 month–47 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

Have a medically confirmed diagnosis of classic CAH (salt wasting or simple virilizing) due to 21-hydroxylase deficiency (21-OHD)
Have received at least 3 months of hydrocortisone treatment before Day 1, with a stable dose for at least 4 weeks before screening.
Have a body weight of at least 4.5 kilograms (kg) at screening.
Have a newborn screen that is otherwise normal except for elevated 17-OHP or any other abnormality on newborn screen that was cleared upon evaluation by a pediatric specialist.

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

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

Time frame: Day 1 up to 28 weeks

Study contacts

Contact information is provided by the study sponsor or research team.

Neurocrine Medical Information Call Center

CONTACT

[email protected]

1-877-641-3461

Sponsors and collaborators

Lead sponsor

Neurocrine Biosciences

Industry

Registry information

Official study title

A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Crinecerfont in Pediatric Participants 3 Months to <4 Years of Age With Classic Congenital Adrenal Hyperplasia

Important dates

Study start: 2026
Primary completion: 2028
Study completion: 2028
First posted: Apr 17, 2026
Registry last updated: Apr 17, 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.

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