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

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ClinicalTrials.gov Identifier: NCT03525886
Recruitment Status : Recruiting
First Posted : May 16, 2018
Last Update Posted : March 13, 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 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 three NBI-74788 dose cohorts, with each dose administered for 14 consecutive days. Cohort 3 will be conducted in parallel with cohort 2.

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 : 30 participants
Allocation: Non-Randomized
Intervention Model: Sequential 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 Adult Subjects With Congenital Adrenal Hyperplasia
Actual Study Start Date : April 10, 2018
Estimated Primary Completion Date : April 2019
Estimated Study Completion Date : April 2019


Arm Intervention/treatment
Experimental: NBI-74788 Dose Group 1
NBI-74788 administered orally for 14 consecutive days.
Drug: NBI-74788
Capsule, administered once daily.

Experimental: NBI-74788 Dose Group 2
NBI-74788 administered orally for 14 consecutive days. Dosing will not commence until safety and tolerability data from Dose Group 1 have been reviewed. Group 2 will be dosed in parallel with Group 3.
Drug: NBI-74788
Capsule, administered once daily.

Experimental: NBI-74788 Dose Group 3
NBI-74788 administered orally under an alternative dosing regimen for 14 consecutive days. Dosing will not commence until safety and tolerability data from Dose Group 1 have been reviewed. Group 3 will be dosed in parallel with Group 2.
Drug: NBI-74788
Capsule, administered once daily.




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:   18 Years to 50 Years   (Adult)
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.
  8. 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. 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 ≥550 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): NCT03525886


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

Locations
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United States, Colorado
Neurocrine Clinical Site Recruiting
Aurora, Colorado, United States, 80045
United States, Indiana
Neurocrine Clinical Site Recruiting
Indianapolis, Indiana, United States, 46202
United States, Michigan
Neurocrine Clinical Site Recruiting
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
Neurocrine Clinical Site Recruiting
Minneapolis, Minnesota, United States, 55454
United States, Pennsylvania
Neurocrine Clinical Site Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, Washington
Neurocrine Clinical Site Recruiting
Seattle, Washington, United States, 98105
Sponsors and Collaborators
Neurocrine Biosciences

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Responsible Party: Neurocrine Biosciences
ClinicalTrials.gov Identifier: NCT03525886     History of Changes
Other Study ID Numbers: NBI-74788-CAH2001
First Posted: May 16, 2018    Key Record Dates
Last Update Posted: March 13, 2019
Last Verified: March 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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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