This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
103
CRF type 1 receptor antagonist
Non-active dosage form
Neurocrine Clinical Site
Birmingham, Alabama, United States
Neurocrine Clinical Site
Los Angeles, California, United States
Neurocrine Clinical Site
Orange, California, United States
Neurocrine Clinical Site
San Diego, California, United States
Neurocrine Clinical Site
San Francisco, California, United States
Change From Baseline in Serum Androstenedione at Week 4
Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
Time frame: Baseline, Week 4
Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4
Time frame: Baseline, Week 4
Percent Change From Baseline in Glucocorticoid Daily Dose at Week 28
Time frame: Baseline, Week 28
Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 28
Time frame: Week 28
Change From Baseline in Body Mass Index (BMI) Standard Deviation Score (SDS) at Week 28
Time frame: Baseline, Week 28
Change From Baseline in Mean 24-hour Salivary 17-OHP at Week 28
Time frame: Baseline, Week 28
Change From Baseline in the Ratio of Bone Age to Chronological Age (BA:CA) at Week 28
Time frame: Baseline, Week 28
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Neurocrine Clinical Site
Aurora, Colorado, United States
Neurocrine Clinical Site
Hartford, Connecticut, United States
Neurocrine Clinical Site
Washington D.C., District of Columbia, United States
Neurocrine Clinical Site
Atlanta, Georgia, United States
Neurocrine Clinical Site
Indianapolis, Indiana, United States
...and 36 more locations