Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia
Primary Purpose
CAH - Congenital Adrenal Hyperplasia
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
NBI-74788
Sponsored by
About this trial
This is an interventional treatment trial for CAH - Congenital Adrenal Hyperplasia
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.
- 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.
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 ≥250 mL or donated blood within 56 days or donated plasma within 7 days before baseline.
Sites / Locations
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
NBI-74788
Arm Description
NBI-74788 administered orally for 14 consecutive days.
Outcomes
Primary Outcome Measures
Number of participants with adverse events following dosing of NBI-74788
Secondary Outcome Measures
Area under the plasma concentration versus time curve (AUC) of NBI-74788 and its metabolites following dosing of NBI-74788
Concentrations of 17-hydroxyprogesterone (17-OHP) following dosing of NBI-74788
Concentrations of biomarkers following dosing of NBI-74788
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04045145
Brief Title
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
June 2022
Overall Recruitment Status
Completed
Study Start Date
September 11, 2019 (Actual)
Primary Completion Date
July 2, 2021 (Actual)
Study Completion Date
July 2, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurocrine Biosciences
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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 approximately 12 pediatric female and male subjects (14 to 17 years of age) with a documented medical diagnosis of classic 21-hydroxylase deficiency congenital adrenal hyperplasia (CAH).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
CAH - Congenital Adrenal Hyperplasia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
NBI-74788
Arm Type
Experimental
Arm Description
NBI-74788 administered orally for 14 consecutive days.
Intervention Type
Drug
Intervention Name(s)
NBI-74788
Intervention Description
NBI-74788 administered orally for 14 consecutive days.
Primary Outcome Measure Information:
Title
Number of participants with adverse events following dosing of NBI-74788
Time Frame
Up to 7 Weeks
Secondary Outcome Measure Information:
Title
Area under the plasma concentration versus time curve (AUC) of NBI-74788 and its metabolites following dosing of NBI-74788
Time Frame
From baseline up to 7 weeks
Title
Concentrations of 17-hydroxyprogesterone (17-OHP) following dosing of NBI-74788
Time Frame
From baseline up to 7 weeks
Title
Concentrations of biomarkers following dosing of NBI-74788
Time Frame
From baseline up to 7 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
14 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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.
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.
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 ≥250 mL or donated blood within 56 days or donated plasma within 7 days before baseline.
Facility Information:
Facility Name
Neurocrine Clinical Site
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Neurocrine Clinical Site
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Neurocrine Clinical Site
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Neurocrine Clinical Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55454
Country
United States
Facility Name
Neurocrine Clinical Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Neurocrine Clinical Site
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia
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