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

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ClinicalTrials.gov Identifier: NCT02349503
Recruitment Status : Withdrawn
First Posted : January 29, 2015
Last Update Posted : June 11, 2015
Sponsor:
Information provided by (Responsible Party):
Neurocrine Biosciences

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.

Condition or disease Intervention/treatment Phase
Congenital Adrenal Hyperplasia Drug: NBI-77860 Phase 1

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : February 2015
Estimated Primary Completion Date : October 2015
Estimated Study Completion Date : October 2015


Arm Intervention/treatment
Experimental: NBI-77860 Dose Group1
NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours).
Drug: NBI-77860
Experimental: NBI-77860 Dose Group 2
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.
Drug: NBI-77860
Experimental: NBI-77860 Dose Group 3
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.
Drug: NBI-77860



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

Secondary Outcome Measures :
  1. 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) ]
  2. 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) ]
  3. 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) ]


Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02349503


Locations
United States, Michigan
Ann Arbor, Michigan, United States, 48109
United States, Washington
Seattle, Washington, United States, 98105
Sponsors and Collaborators
Neurocrine Biosciences

Responsible Party: Neurocrine Biosciences
ClinicalTrials.gov Identifier: NCT02349503     History of Changes
Other Study ID Numbers: NBI-77860-1401
First Posted: January 29, 2015    Key Record Dates
Last Update Posted: June 11, 2015
Last Verified: June 2015

Keywords provided by Neurocrine Biosciences:
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

Additional relevant MeSH terms:
Hyperplasia
Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Congenital Abnormalities
Genetic Diseases, Inborn
Steroid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases
Adrenal Gland Diseases
Endocrine System Diseases
Gonadal Disorders
Epinephrine
Racepinephrine
Epinephryl borate
Adrenergic alpha-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Adrenergic beta-Agonists
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Anti-Asthmatic Agents
Respiratory System Agents