Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old
Not Applicable
Not yet recruiting
- Conditions
- Congenital Adrenal Hyperplasia
- Interventions
- Registration Number
- NCT07187375
- Lead Sponsor
- Neurocrine Biosciences
- 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 6
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Crinecerfont Crinecerfont Participants with CAH will receive crinecerfont during an initial 14-day treatment period, followed by an optional 36-month open-label extension (OLE).
- Primary Outcome Measures
Name Time Method Plasma Concentration of Crinecerfont Days 7 and 15
- Secondary Outcome Measures
Name Time Method Number of Participants With Treatment-emergent Adverse Events (TEAEs) Up to Month 37