This is a Phase 2, open-label, multiple-dose, study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 (crinecerfont) in pediatric participants (14 to 17 years of age) with a documented medical diagnosis of classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
Crinecerfont administered orally for 14 consecutive days.
Neurocrine Clinical Site
San Diego, California, United States
Neurocrine Clinical Site
Aurora, Colorado, United States
Neurocrine Clinical Site
Ann Arbor, Michigan, United States
Neurocrine Clinical Site
Minneapolis, Minnesota, United States
Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average)
Percent changes in 17-OHP were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The 2 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Time frame: Baseline, Day 14
Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (24-hour Period)
Percent changes in 17-OHP were assessed through the collection of samples for a 24-hour period prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The average of each participant's serial sampling values at each visit where serial sampling was performed was calculated and used to determine the percent change from baseline.
Time frame: Baseline, Day 14
Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) (Morning Window Averages)
Percent changes in ACTH were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Time frame: Baseline, Day 14
Percent Change From Baseline to Day 14 in Androstenedione (Morning Window Averages)
Percent changes in androstenedione were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Time frame: Baseline, Day 14
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Neurocrine Clinical Site
Philadelphia, Pennsylvania, United States
Neurocrine Clinical Site
Seattle, Washington, United States
Percent Change From Baseline to Day 14 in Testosterone (Morning Window Averages)
Percent change in testosterone were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Time frame: Baseline, Day 14