Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old
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
1 other identifier
interventional
20
1 country
11
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jul 2026
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 10, 2026
CompletedFirst Posted
Study publicly available on registry
April 17, 2026
CompletedStudy Start
First participant enrolled
July 1, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 1, 2028
September 16, 2026
September 1, 2026
1.7 years
April 10, 2026
September 14, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Participants With Treatment-emergent Adverse Events (TEAE)
Day 1 up to 28 weeks
Study Arms (1)
Crinecerfont
EXPERIMENTALParticipants with CAH will receive crinecerfont.
Interventions
Eligibility Criteria
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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
Neurocrine Clinical Site
San Francisco, California, 94143, United States
Neurocrine Clinical Site
Aurora, Colorado, 80045, United States
Neurocrine Clinical Site
Hartford, Connecticut, 06106, United States
Neurocrine Clinical Site
Atlanta, Georgia, 30329, United States
Neurocrine Clinical Site
Chicago, Illinois, 60618, United States
Neurocrine Clinical Site
Indianapolis, Indiana, 46202, United States
Neurocrine Clinical Site
Ann Arbor, Michigan, 48109, United States
Neurocrine Clinical Site
Minneapolis, Minnesota, 55454, United States
Neurocrine Clinical Site
Philadelphia, Pennsylvania, 19104, United States
Neurocrine Clinical Site
Dallas, Texas, 75235, United States
Neurocrine Clinical Site
Seattle, Washington, 98101, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Clinical Development Lead
Neurocrine Biosciences
Central Study Contacts
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
July 1, 2026
Primary Completion (Estimated)
March 1, 2028
Study Completion (Estimated)
March 1, 2028
Last Updated
September 16, 2026
Record last verified: 2026-09
Data Sharing
- IPD Sharing
- Will not share