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

Primary Purpose

Congenital Adrenal Hyperplasia

Status
Withdrawn
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
NBI-77860
NBI-77860
NBI-77860
Sponsored by
Neurocrine Biosciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Adrenal Hyperplasia focused on measuring Adrenocortical function, Adrenal hyperplasia, Congenital, 21-hydroxylase deficiency, Adrenogenital Syndrome, Adolescents, Female, Hyperplasia, Adrenal Gland Diseases, Congenital Abnormalities, Adrenocortical Hyperfunction, Disorders of Sex Development, Endocrine System Diseases, Genetic Diseases, Inborn, Gonadal Disorders, Metabolic Diseases, Metabolism, Inborn Errors, Pathologic Processes, Steroid Metabolism, Inborn Errors, Urogenital Abnormalities, Corticotropin Releasing Factor

Eligibility Criteria

12 Years - 18 Years (Child, Adult)FemaleDoes not accept healthy volunteers

Inclusion Criteria:

  1. Have documentation of written informed consent, or written and witnessed assent from the subject and written informed consent from the subject's parent or legal guardian.
  2. Be in good general health.
  3. Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
  4. Be on a stable regimen of steroidal treatment for CAH for a minimum of 30 days before baseline (Night 1) that is expected to remain stable throughout the study.
  5. 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 30 days after the last dose of study drug, whichever is longer.
  6. Subjects of childbearing potential must have a negative pregnancy test at screening and negative urine pregnancy test at baseline (Night 1).
  7. Have a negative urine drug (for illegal drugs) and alcohol breath test at screening and baseline (Night 1).
  8. 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.
  9. Be willing to provide authorization for access to personal health information in conjunction with US Health Insurance Portability and Accountability Act (HIPAA).

Exclusion Criteria:

  1. Have a clinically significant unstable medical condition or chronic disease, or malignancy.
  2. Had a medically significant illness within 30 days of screening.
  3. Have a known or suspected differential diagnosis of any of the other known forms of classic CAH.
  4. Have a history that includes bilateral adrenalectomy, hypopituitarism, or other condition requiring daily therapy with orally administered glucocorticoids.
  5. Pregnant or lactating females.
  6. Have a history of epilepsy or serious head injury.
  7. Test positive at screening for hepatitis B, hepatitis C, or human immunodeficiency virus (HIV), or have a history of a positive result.
  8. Have a recent history (≤1 year) of alcohol or drug abuse, or current evidence of substance dependence or abuse criteria.
  9. 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.
  10. Have a blood loss ≥250 mL or donated blood within 56 days or donated plasma within 7 days before baseline.
  11. Self-report consumption of more than 6 caffeine-containing beverages a day within the last month before baseline.

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

NBI-77860 Dose Group1

NBI-77860 Dose Group 2

NBI-77860 Dose Group 3

Arm Description

NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours).

NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours). Dosing will not commence until all safety and PK results from the dose group 1 have been reviewed to ensure there are no safety concerns and that maximum tolerated dose (MTD) has not been reached.

NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours). Dosing will not commence until all safety and PK results from the dose group 2 have been reviewed to ensure there are no safety concerns and that maximum tolerated dose (MTD) has not been reached.

Outcomes

Primary Outcome Measures

Number of participants with adverse events following one oral dose of NBI-77860

Secondary Outcome Measures

Area Under Concentration Curve (AUC) of NBI-77860 and its metabolites following one oral dose of NBI-77860
Concentrations of 17-hydroxyprogesterone (17-OHP) following one oral dose of NBI-77860
Concentrations of adrenocorticotropin hormone (ACTH) following one oral dose of NBI-77860

Full Information

First Posted
January 20, 2015
Last Updated
June 9, 2015
Sponsor
Neurocrine Biosciences
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1. Study Identification

Unique Protocol Identification Number
NCT02349503
Brief Title
Safety, Pharmacokinetics and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia
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 Adrenal Hyperplasia
Study Type
Interventional

2. Study Status

Record Verification Date
June 2015
Overall Recruitment Status
Withdrawn
Study Start Date
February 2015 (undefined)
Primary Completion Date
October 2015 (Anticipated)
Study Completion Date
October 2015 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurocrine Biosciences

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Adrenal Hyperplasia
Keywords
Adrenocortical function, Adrenal hyperplasia, Congenital, 21-hydroxylase deficiency, Adrenogenital Syndrome, Adolescents, Female, Hyperplasia, Adrenal Gland Diseases, Congenital Abnormalities, Adrenocortical Hyperfunction, Disorders of Sex Development, Endocrine System Diseases, Genetic Diseases, Inborn, Gonadal Disorders, Metabolic Diseases, Metabolism, Inborn Errors, Pathologic Processes, Steroid Metabolism, Inborn Errors, Urogenital Abnormalities, Corticotropin Releasing Factor

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
NBI-77860 Dose Group1
Arm Type
Experimental
Arm Description
NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours).
Arm Title
NBI-77860 Dose Group 2
Arm Type
Experimental
Arm Description
NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours). Dosing will not commence until all safety and PK results from the dose group 1 have been reviewed to ensure there are no safety concerns and that maximum tolerated dose (MTD) has not been reached.
Arm Title
NBI-77860 Dose Group 3
Arm Type
Experimental
Arm Description
NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours). Dosing will not commence until all safety and PK results from the dose group 2 have been reviewed to ensure there are no safety concerns and that maximum tolerated dose (MTD) has not been reached.
Intervention Type
Drug
Intervention Name(s)
NBI-77860
Intervention Type
Drug
Intervention Name(s)
NBI-77860
Intervention Type
Drug
Intervention Name(s)
NBI-77860
Primary Outcome Measure Information:
Title
Number of participants with adverse events following one oral dose of NBI-77860
Time Frame
Up to 8 weeks
Secondary Outcome Measure Information:
Title
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)
Title
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)
Title
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)

10. Eligibility

Sex
Female
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Have documentation of written informed consent, or written and witnessed assent from the subject and written informed consent from the subject's parent or legal guardian. 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 for a minimum of 30 days before baseline (Night 1) 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 30 days 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 (Night 1). Have a negative urine drug (for illegal drugs) and alcohol breath test at screening and baseline (Night 1). 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. Pregnant or lactating females. Have a history of epilepsy or serious head injury. 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 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. Self-report consumption of more than 6 caffeine-containing beverages a day within the last month before baseline.
Facility Information:
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States

12. IPD Sharing Statement

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

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