Congenital Adrenal HyperplasiaPhase 1NCT02349503

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

A Phase 1 study testing NBI-77860 in people with Congenital Adrenal Hyperplasia, run by Neurocrine Biosciences at 2 sites in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record

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Trial summary

Who is it for?: Ages 12 to 18 years, women only
Conditions the study is for: Congenital Adrenal Hyperplasia
What is being tested?: NBI-77860
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 0 participants
Where: 2 sitesUnited States
Study dates: Feb 2015to Oct 2015
Phase: Phase 1

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 12 to 18 years
Women only
No healthy volunteers
Child, Adult

Who may be able to join

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.
Be in good general health.
Have a medically confirmed diagnosis of classic 21-hydroxylase deficiency CAH.
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.
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.
Subjects of childbearing potential must have a negative pregnancy test at screening and negative urine pregnancy test at baseline (Night 1).

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Have a negative urine drug (for illegal drugs) and alcohol breath test at screening and baseline (Night 1).
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.
Be willing to provide authorization for access to personal health information in conjunction with US Health Insurance Portability and Accountability Act (HIPAA).

Who cannot join

Have a clinically significant unstable medical condition or chronic disease, or malignancy.
Had a medically significant illness within 30 days of screening.
Have a known or suspected differential diagnosis of any of the other known forms of classic CAH.
Have a history that includes bilateral adrenalectomy, hypopituitarism, or other condition requiring daily therapy with orally administered glucocorticoids.
Pregnant or lactating females.
Have a history of epilepsy or serious head injury.

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Test positive at screening for hepatitis B, hepatitis C, or human immunodeficiency virus (HIV), or have a history of a positive result.
Have a recent history (≤1 year) of alcohol or drug abuse, or current evidence of substance dependence or abuse criteria.
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.
Have a blood loss ≥250 mL or donated blood within 56 days or donated plasma within 7 days before baseline.
Self-report consumption of more than 6 caffeine-containing beverages a day within the last month before baseline.

Trial locations and contacts

2 sites in 1 country.

United States2 sites

Study site
Ann Arbor, Michigan, 48109
Study site
Seattle, Washington, 98105

Other trials in United States

About this study

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

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.

Conditions: Congenital Adrenal Hyperplasia
Being tested: NBI-77860
Keywords: 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
Medical subject headings: Adrenal Hyperplasia, Congenital, Congenital adrenal hyperplasia due to 21 hydroxylase deficiency, Adrenogenital Syndrome, Hyperplasia, Adrenal Gland Diseases, Congenital Abnormalities, Adrenocortical Hyperfunction, Disorders of Sex Development

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

NBI-77860Drug
NBI-77860Drug
NBI-77860Drug

Study groups in NCT02349503
Study group	Role	What they receive
NBI-77860 Dose Group1	Experimental	NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours). Receives: Drug: NBI-77860
NBI-77860 Dose Group 2	Experimental	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. Receives: Drug: NBI-77860
NBI-77860 Dose Group 3	Experimental	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. Receives: Drug: NBI-77860

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of participants with adverse events following one oral dose of NBI-77860
Measured: Up to 8 weeks

Secondary outcomes (3)

Area Under Concentration Curve (AUC) of NBI-77860 and its metabolites following one oral dose of NBI-77860
Measured: 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
Measured: 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
Measured: Screening, Night 1, Day 2 (10, 12 and 24 hours postdose) and 35 (or early termination)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Feb 2015Oct 2015

First posted29 Jan 2015 (estimated)
Study startFeb 2015
Primary completionOct 2015 (estimated)
Study completionOct 2015 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
More trials run by Neurocrine BiosciencesThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02349503
Sponsor's protocol ID: NBI-77860-1401
Status last verified: Jun 2015
First submitted: 20 Jan 2015
Last updated: 11 Jun 2015
Results posted: No
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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