Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old

Neurocrine Biosciences

Status and phase: Enrolling; Phase 2

Conditions: Congenital Adrenal Hyperplasia

Treatments: Drug: Crinecerfont

Study type: Interventional

Funder types: Industry

Identifiers

NCT07187375

2024-514127-42-00 (EU Trial (CTIS) Number)

NBI-74788-CAH2011

Details and patient eligibility

About

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).

Enrollment: 6 estimated patients

Sex: All

Ages: Under 23 months old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• 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.

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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6 participants in 1 patient group

Crinecerfont

Experimental group

Description:

Participants with CAH will receive crinecerfont during an initial 14-day treatment period, followed by an optional 36-month open-label extension (OLE).

Treatment:

Drug: Crinecerfont