Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old
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
2 other identifiers
interventional
6
1 country
3
Brief Summary
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).
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 Sep 2025
Typical duration for phase_2
3 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
September 18, 2025
CompletedFirst Posted
Study publicly available on registry
September 23, 2025
CompletedStudy Start
First participant enrolled
September 30, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 6, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 4, 2029
March 20, 2026
March 1, 2026
3.9 years
September 18, 2025
March 17, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Plasma Concentration of Crinecerfont
Days 7 and 15
Secondary Outcomes (1)
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Up to Month 37
Study Arms (1)
Crinecerfont
EXPERIMENTALParticipants with CAH will receive crinecerfont during an initial 14-day treatment period, followed by an optional 36-month open-label extension (OLE).
Interventions
Eligibility Criteria
You may qualify if:
- 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.
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 (3)
Neurocrine Clinical Site
Berlin, 13353, Germany
Neurocrine Clinical Site
Düsseldorf, 40225, Germany
Neurocrine Clinical Site
Heidelberg, 69120, Germany
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
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
September 18, 2025
First Posted
September 23, 2025
Study Start
September 30, 2025
Primary Completion (Estimated)
September 6, 2029
Study Completion (Estimated)
October 4, 2029
Last Updated
March 20, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will not share