Actively Recruiting
Phase 2 Open-Label Study to Evaluate Pharmacokinetics, Safety, and Tolerability of Crinecerfont in Children Under 2 With Congenital Adrenal Hyperplasia
Led by Neurocrine Biosciences · Updated on 2026-03-20
6
Participants Needed
3
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying crinecerfont, a drug, in children under 2 years old who have congenital adrenal hyperplasia (CAH), a condition caused by a deficiency in the 21-hydroxylase enzyme. The main focus is to understand how crinecerfont behaves in the body (pharmacokinetics) in this young patient group. This is a Phase 2 open-label study sponsored by Neurocrine Biosciences to evaluate safety, tolerability, and pharmacodynamics of the drug. Participants with CAH will receive crinecerfont as an oral solution during an initial 14-day treatment period. After this, they may choose to join a 36-month open-label extension where they continue to take the drug under observation. The study does not involve placebo or masking, and all participants receive the investigational drug. During the study, researchers will measure the plasma concentration of crinecerfont on days 7 and 15 to assess how the drug is processed in the body. They will also monitor participants for any treatment-emergent adverse events over up to 37 months. The total involvement may last for the initial treatment plus the extension period, with regular monitoring of safety and drug effects throughout.
CONDITIONS
Brief Title
Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Be a female or male between 0 to less than 2 years of age at screening
- Have a medically confirmed diagnosis of classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency
- Be on a clinically stable regimen of hydrocortisone (and fludrocortisone, if applicable) treatment
You will not qualify if you...
- Have a known or suspected diagnosis of any other form of classic congenital adrenal hyperplasia
- Have any condition besides congenital adrenal hyperplasia that requires chronic daily oral steroid therapy
- Have any other clinically significant medical condition or chronic disease
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 14 days
Participants receive crinecerfont oral solution for an initial 14-day treatment period.
Visits on Days 7 and 15 for plasma concentration measurements
Duration - Up to 36 months
Participants may continue crinecerfont during an optional 36-month open-label extension.
Follow-up visits as scheduled during extension
Trial Site Locations
Total: 3 locations
1
Neurocrine Clinical Site
Berlin, Germany, 13353
Actively Recruiting
2
Neurocrine Clinical Site
Düsseldorf, Germany, 40225
Actively Recruiting
3
Neurocrine Clinical Site
Heidelberg, Germany, 69120
Actively Recruiting
Research Team
N
Neurocrine Medical Information Call Center
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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