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Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Pediatric Subjects With Congenital Adrenal Hyperplasia

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ClinicalTrials.gov Identifier: NCT04045145
Recruitment Status : Recruiting
First Posted : August 5, 2019
Last Update Posted : September 11, 2019
Sponsor:
Information provided by (Responsible Party):
Neurocrine Biosciences

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).

Condition or disease Intervention/treatment Phase
CAH - Congenital Adrenal Hyperplasia Drug: NBI-74788 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Estimated Study Start Date : September 2019
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : June 2020


Arm Intervention/treatment
Experimental: NBI-74788
NBI-74788 administered orally for 14 consecutive days.
Drug: NBI-74788
NBI-74788 administered orally for 14 consecutive days.




Primary Outcome Measures :
  1. Number of participants with adverse events following dosing of NBI-74788 [ Time Frame: Up to 7 Weeks ]

Secondary Outcome Measures :
  1. 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 ]
  2. Concentrations of 17-hydroxyprogesterone (17-OHP) following dosing of NBI-74788 [ Time Frame: From baseline up to 7 weeks ]
  3. Concentrations of biomarkers following dosing of NBI-74788 [ Time Frame: From baseline up to 7 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   14 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Be in good general health.
  2. Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
  3. Be on a stable regimen of steroidal treatment for CAH that is expected to remain stable throughout the study.
  4. 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.
  5. Subjects of childbearing potential must have a negative pregnancy test at screening and negative urine pregnancy test at baseline.
  6. Have a negative urine drug (for illegal drugs) and alcohol breath test at screening and baseline.
  7. 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:

  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. Are pregnant or lactating females.
  6. Have a history of epilepsy or serious head injury.
  7. Have a known history of long QT syndrome or cardiac tachy-arrhythmia.
  8. Have hypersensitivity to any corticotropin releasing hormone antagonists.
  9. Test positive at screening for hepatitis B, hepatitis C, or human immunodeficiency virus (HIV), or have a history of a positive result.
  10. Have a recent history (≤1 year) of alcohol or drug abuse, or current evidence of substance dependence or abuse criteria.
  11. Used any anticoagulants or antiplatelet therapies within 30 days before screening.
  12. Have an active bleeding disorder.
  13. 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.
  14. Have a blood loss ≥250 mL or donated blood within 56 days or donated plasma within 7 days 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): NCT04045145


Contacts
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Contact: Cheryl Chen 858-617-7744 cechen@neurocrine.com

Locations
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United States, California
Neurocrine Clinical Site Recruiting
San Diego, California, United States, 92123
Sponsors and Collaborators
Neurocrine Biosciences

Additional Information:
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Responsible Party: Neurocrine Biosciences
ClinicalTrials.gov Identifier: NCT04045145     History of Changes
Other Study ID Numbers: NBI-74788-CAH2008
First Posted: August 5, 2019    Key Record Dates
Last Update Posted: September 11, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Hyperplasia
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