This is a Phase 1, multicenter, open-label, single-dose study to evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-77860 in subjects with congenital adrenal hyperplasia (CAH). The study will be conducted in approximately 15 adolescent females (12-18 years of age) with a documented medical diagnosis of classic 21-hydroxylase deficiency CAH. The study will include three independent dose cohorts of NBI-77860 (approximately 5 subjects per dose cohort). Ascending doses will be evaluated as part of a sequential-cohort design.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Unnamed facility
Ann Arbor, Michigan, United States
Unnamed facility
Seattle, Washington, United States
Number of participants with adverse events following one oral dose of NBI-77860
Time frame: Up to 8 weeks
Area Under Concentration Curve (AUC) of NBI-77860 and its metabolites following one oral dose of NBI-77860
Time frame: Night 1 and Days 2, 7, 14, 21 and 35 (or early termination)
Concentrations of 17-hydroxyprogesterone (17-OHP) following one oral dose of NBI-77860
Time frame: Screening, Night 1, Day 2 (10, 12 and 24 hours postdose) and 35 (or early termination)
Concentrations of adrenocorticotropin hormone (ACTH) following one oral dose of NBI-77860
Time frame: Screening, Night 1, Day 2 (10, 12 and 24 hours postdose) and 35 (or early termination)
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