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.
Trial 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). Read more about this study
- 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
- Are there results?
- None posted in the registry
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
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 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.
- 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)
Sponsor and investigators
- Lead sponsor
- Neurocrine Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
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
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02349503
- Acronym / sponsor ID
- NBI-77860-1401
- Conditions
- Congenital Adrenal Hyperplasia
- Keywords
- Adrenocortical function, Adrenal hyperplasia, Congenital, 21-hydroxylase deficiency, Adrenogenital Syndrome, Adolescents, Female, Hyperplasia, Adrenal Gland Diseases
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Neurocrine BiosciencesIndustry (pharma, biotech, devices)
- Study start
- Feb 2015
- Primary completion
- Oct 2015 (estimated)
- Study completion
- Oct 2015 (estimated)
- First posted
- 29 Jan 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| NBI-77860 Dose Group1 | Experimental | Drug: NBI-77860NBI-77860 administered orally on Night 1 at bedtime (at approximately 2200 hours). |
| NBI-77860 Dose Group 2 | Experimental | Drug: NBI-77860NBI-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. |
| NBI-77860 Dose Group 3 | Experimental | Drug: NBI-77860NBI-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. |
Who may qualify
- Ages
- 12 to 18 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NBI-77860 | Drug | NBI-77860 Dose Group1 |
| NBI-77860 | Drug | NBI-77860 Dose Group 2 |
| NBI-77860 | Drug | NBI-77860 Dose Group 3 |
Primary outcome
Number of participants with adverse events following one oral dose of NBI-77860
Time frame: Up to 8 weeks
3 secondary outcomes
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)
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing NBI-77860 in people with Congenital Adrenal Hyperplasia, run by Neurocrine Biosciences at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 11 Jun 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with adverse events following one oral dose of NBI-77860 (time frame: Up to 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2015.