Clinical Trial: Safety, Pharmacokinetics and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

Official Title: A Phase 1, Open-Label, Single-Dose, Sequential Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital

Brief Summary: 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.

Detailed Summary:
Sponsor: Neurocrine Biosciences

Current Primary Outcome: Number of participants with adverse events following one oral dose of NBI-77860 [ Time Frame: Up to 8 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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) ]


Original Secondary Outcome: Same as current

Information By: Neurocrine Biosciences

Dates:
Date Received: January 20, 2015
Date Started: February 2015
Date Completion: October 2015
Last Updated: June 9, 2015
Last Verified: June 2015