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

NCT07536269 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: NBI-74788-CAH2032
• Study Type: interventional
• Target: 20
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Congenital Adrenal Hyperplasia

Keywords

Congenital Adrenal Hyperplasia; CAH; Crinecerfont; Pediatric

Outcome Measures

Primary Outcomes (1)

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

  Day 1 up to 28 weeks

Study Arms (1)

Crinecerfont

EXPERIMENTAL

Participants with CAH will receive crinecerfont.

Drug: Crinecerfont

Interventions

Crinecerfont DRUG

Oral solution

Crinecerfont

Eligibility Criteria

• Age: 3 Months - 47 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Neurocrine Biosciences: lead

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital

Interventions

crinecerfont

Condition Hierarchy (Ancestors)

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

Study Officials

• Clinical Development Lead

  Neurocrine Biosciences

  STUDY DIRECTOR

Central Study Contacts

Neurocrine Medical Information Call Center

CONTACT

1-877-641-3461; medinfo@neurocrine.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 10, 2026
• First Posted: April 17, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): March 1, 2028
• Study Completion (Estimated): March 1, 2028
• Last Updated: April 17, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share