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

April 10, 2026 updated by: Neurocrine Biosciences

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

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Crinecerfont
Participants with CAH will receive crinecerfont.
Drug: Crinecerfont
Oral solution

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Participants With Treatment-emergent Adverse Events (TEAE)
Time Frame: Day 1 up to 28 weeks
Day 1 up to 28 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Neurocrine Biosciences

Investigators

  • Study Director: Clinical Development Lead, Neurocrine Biosciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

April 1, 2026

Primary Completion (Estimated)

March 1, 2028

Study Completion (Estimated)

March 1, 2028

Study Registration Dates

First Submitted

April 10, 2026

First Submitted That Met QC Criteria

April 10, 2026

First Posted (Actual)

April 17, 2026

Study Record Updates

Last Update Posted (Actual)

April 17, 2026

Last Update Submitted That Met QC Criteria

April 10, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NBI-74788-CAH2032

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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