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Trial record 15 of 108 for:    "21-hydroxylase deficiency"

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Recruitment Status : Withdrawn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

Tracking Information
First Submitted Date  ICMJE January 20, 2015
First Posted Date  ICMJE January 29, 2015
Last Update Posted Date June 11, 2015
Study Start Date  ICMJE February 2015
Estimated Primary Completion Date October 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 23, 2015)
Number of participants with adverse events following one oral dose of NBI-77860 [ Time Frame: Up to 8 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02349503 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 23, 2015)
  • 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) ]
  • 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) ]
  • 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) ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety, Pharmacokinetics and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Congenital Adrenal Hyperplasia
Intervention  ICMJE Drug: NBI-77860
Study Arms  ICMJE
  • Experimental: NBI-77860 Dose Group1
    NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours).
    Intervention: 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.
    Intervention: 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.
    Intervention: Drug: NBI-77860
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: June 9, 2015)
0
Original Estimated Enrollment  ICMJE
 (submitted: January 23, 2015)
15
Estimated Study Completion Date  ICMJE October 2015
Estimated Primary Completion Date October 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Female
Ages  ICMJE 12 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02349503
Other Study ID Numbers  ICMJE NBI-77860-1401
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Neurocrine Biosciences
Study Sponsor  ICMJE Neurocrine Biosciences
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Neurocrine Biosciences
Verification Date June 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP