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

CAH - Congenital Adrenal Hyperplasia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04045145
• Other study ID #: NBI-74788-CAH2008
• Status: Completed
• Phase: Phase 2
• Start date: September 11, 2019
• Est. completion date: July 2, 2021

Study information

• Verified date: June 2022
• Source: Neurocrine Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: July 2, 2021
• Est. primary completion date: July 2, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Adrenal Hyperplasia, Congenital
• Adrenocortical Hyperfunction
• Adrenogenital Syndrome
• CAH - Congenital Adrenal Hyperplasia
• Hyperplasia

Intervention

Drug:
• NBI-74788
NBI-74788 administered orally for 14 consecutive days.

Locations

Country	Name	City	State
United States	Neurocrine Clinical Site	Ann Arbor	Michigan
United States	Neurocrine Clinical Site	Aurora	Colorado
United States	Neurocrine Clinical Site	Minneapolis	Minnesota
United States	Neurocrine Clinical Site	Philadelphia	Pennsylvania
United States	Neurocrine Clinical Site	San Diego	California
United States	Neurocrine Clinical Site	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Neurocrine Biosciences

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with adverse events following dosing of NBI-74788		Up to 7 Weeks
Secondary	Area under the plasma concentration versus time curve (AUC) of NBI-74788 and its metabolites following dosing of NBI-74788		From baseline up to 7 weeks
Secondary	Concentrations of 17-hydroxyprogesterone (17-OHP) following dosing of NBI-74788		From baseline up to 7 weeks
Secondary	Concentrations of biomarkers following dosing of NBI-74788		From baseline up to 7 weeks

External Links

•   Study website