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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-09-30

Study Completion Date

2029-10-04

Brief Summary

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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).

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Detailed Description

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Conditions

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Congenital Adrenal Hyperplasia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Crinecerfont

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

Group Type EXPERIMENTAL

Crinecerfont

Intervention Type DRUG

Oral solution

Interventions

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Crinecerfont

Oral solution

Intervention Type DRUG

Eligibility Criteria

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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.

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.

Minimum Eligible Age

0 Years

Maximum Eligible Age

23 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No