- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03525886
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasia
April 5, 2022 updated by: Neurocrine Biosciences
A Phase 2, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adult Subjects With Congenital Adrenal Hyperplasia
This is a Phase 2, open-label, multiple-dose, dose-escalation study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 in up to 30 adult female and male subjects (18 to 50 years of age) with a documented medical diagnosis of classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH).
The study will include a sequential-cohort design with four NBI-74788 dosing regimens, with each regimen administered for 14 days.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
18
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Colorado
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Aurora, Colorado, United States, 80045
- Neurocrine Clinical Site
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Indiana
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Indianapolis, Indiana, United States, 46202
- Neurocrine Clinical Site
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Michigan
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Ann Arbor, Michigan, United States, 48109
- Neurocrine Clinical Site
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Minnesota
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Minneapolis, Minnesota, United States, 55454
- Neurocrine Clinical Site
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- Neurocrine Clinical Site
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Washington
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Seattle, Washington, United States, 98105
- Neurocrine Clinical Site
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
16 years to 48 years (Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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.
- Subjects 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.
- Subjects 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.
- Be willing to provide authorization for access to personal health information in conjunction with US Health Insurance Portability and Accountability Act (HIPAA).
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 (≤1 year) of alcohol or drug abuse, or current evidence of substance dependence or abuse criteria.
- Used any anticoagulants or antiplatelet therapies within 30 days before screening.
- Have an active bleeding disorder.
- Used any other investigational drug within 30 days before initial screening, or plans to use an investigational drug (other than the study drug) during the study.
- Have a blood loss ≥550 mL or donated blood within 56 days or donated plasma within 7 days before baseline.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Cohort 1 (50 mg QHS)
NBI-74788 50 mg once daily at bedtime (QHS) administered orally for 14 consecutive days.
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Capsule, administered daily.
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Experimental: Cohort 2 (100 mg QHS)
NBI-74788 100 mg once daily at bedtime (QHS) administered orally for 14 consecutive days.
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Capsule, administered daily.
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Experimental: Cohort 3 (100 mg QPM)
NBI-74788 100 mg once daily in the evening (QPM) administered orally for 14 consecutive days.
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Capsule, administered daily.
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Experimental: Cohort 4 (100 mg BID)
NBI-74788 100 mg twice daily (BID) administered orally for 14 consecutive days.
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Capsule, administered daily.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percent Change From Baseline to Day 14 in 17-hydroxyprogesterone (17-OHP) Morning Window Averages
Time Frame: Baseline and Day 14
|
Percent changes in 17-OHP were assessed through the collection of samples from 0600 hours to 1000 hours both prior to study drug administration (i.e., at baseline) and after 14 days of study drug dosing.
The 3 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
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Baseline and Day 14
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percent Change From Baseline to Day 14 in Androstenedione Morning Window Averages
Time Frame: Baseline and Day 14
|
Percent changes in androstenedione were assessed through the collection of samples from 0600 hours to 1000 hours prior to study drug administration (baseline) and after 14 days of study drug dosing.
The 3 samples collected at each visit during this morning window were averaged and used to determine the change and percent change from baseline.
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Baseline and Day 14
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Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) Morning Window Averages
Time Frame: Baseline and Day 14
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Percent changes in ACTH were assessed through the collection of samples from 0600 hours to 1000 hours prior to study drug administration (baseline) and after 14 days of study drug dosing.
The 3 samples collected at each visit during this morning window were averaged and used to determine the change and percent change from baseline.
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Baseline and Day 14
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
April 10, 2018
Primary Completion (Actual)
April 7, 2020
Study Completion (Actual)
April 7, 2020
Study Registration Dates
First Submitted
May 2, 2018
First Submitted That Met QC Criteria
May 14, 2018
First Posted (Actual)
May 16, 2018
Study Record Updates
Last Update Posted (Actual)
May 3, 2022
Last Update Submitted That Met QC Criteria
April 5, 2022
Last Verified
April 1, 2022
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Metabolic Diseases
- Endocrine System Diseases
- Gonadal Disorders
- Disorders of Sex Development
- Urogenital Abnormalities
- Congenital Abnormalities
- Genetic Diseases, Inborn
- Metabolism, Inborn Errors
- Adrenal Gland Diseases
- Steroid Metabolism, Inborn Errors
- Hyperplasia
- Adrenal Hyperplasia, Congenital
- Adrenogenital Syndrome
- Adrenocortical Hyperfunction
Other Study ID Numbers
- NBI-74788-CAH2001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
No
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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