A Phase 2, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia

Neurocrine Biosciences1 site in 1 country8 target enrollmentDecember 12, 2019

ConditionsCAH - Congenital Adrenal Hyperplasia

InterventionsCrinecerfont

DrugsCrinecerfont

Overview

Phase: Phase 2
Intervention: Crinecerfont
Conditions: CAH - Congenital Adrenal Hyperplasia
Sponsor: Neurocrine Biosciences
Enrollment: 8
Locations: 1
Primary Endpoint: Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average)
Status: Completed
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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).

Registry

clinicaltrials.gov

Start Date

December 12, 2019

End Date

July 2, 2021

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Neurocrine Biosciences

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Be in good general health.
•Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
•Be on a stable regimen of steroidal treatment for CAH that is expected to remain stable throughout the study.
•Participants of childbearing potential must be instructed on the proper use of barrier methods of contraception and agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently from screening until the final study visit or a prespecified window after the last dose of study drug, whichever is longer.
•Participants of childbearing potential must have a negative pregnancy test at screening and negative urine pregnancy test at baseline.
•Have a negative urine drug (for illegal drugs) and alcohol breath test at screening and baseline.
•Be willing and able to adhere to the study regimen and study procedures described in the protocol and informed consent/assent form, including all requirements at the study center and return for the follow-up visit.

Exclusion Criteria

•Have a clinically significant unstable medical condition or chronic disease, or malignancy.
•Had a medically significant illness within 30 days of screening.
•Have a known or suspected differential diagnosis of any of the other known forms of classic CAH.
•Have a history that includes bilateral adrenalectomy, hypopituitarism, or other condition requiring daily therapy with orally administered glucocorticoids.
•Are pregnant or lactating females.
•Have a history of epilepsy or serious head injury.
•Have a known history of long QT syndrome or cardiac tachy-arrhythmia.
•Have hypersensitivity to any corticotropin releasing hormone antagonists.
•Test positive at screening for hepatitis B, hepatitis C, or human immunodeficiency virus (HIV), or have a history of a positive result.
•Have a recent history of alcohol or drug abuse, or current evidence of substance dependence or abuse criteria.

Arms & Interventions

Crinecerfont 50 milligrams (mg) Twice Daily (BID)

Crinecerfont administered orally for 14 consecutive days.

Intervention: Crinecerfont

Outcomes

Primary Outcomes

Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average)

Time Frame: Baseline, Day 14

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.

Secondary Outcomes

Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (24-hour Period)(Baseline, Day 14)
Percent Change From Baseline to Day 14 in Androstenedione (Morning Window Averages)(Baseline, Day 14)
Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) (Morning Window Averages)(Baseline, Day 14)
Percent Change From Baseline to Day 14 in Testosterone (Morning Window Averages)(Baseline, Day 14)