CAH - Congenital Adrenal HyperplasiaPhase 2NCT03525886
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 in Adults With Congenital Adrenal Hyperplasia
A Phase 2 study testing NBI-74788 in people with CAH - Congenital Adrenal Hyperplasia, run by Neurocrine Biosciences at 6 sites in United States.
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 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… Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- CAH - Congenital Adrenal Hyperplasia
- What is being tested?
- NBI-74788
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 6 sitesUnited States
- Study dates
- 10 Apr 2018to 7 Apr 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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 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 a prespecified window 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.
- Have a negative urine drug (for illegal drugs) and alcohol breath test at screening and baseline.
- 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.
- Are pregnant or lactating females.
- Have a history of epilepsy or serious head injury.
Show all 14Show fewer
- Have a known history of long QT syndrome or cardiac tachy-arrhythmia.
- Have hypersensitivity to any corticotropin releasing hormone antagonists.
- 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 anticoagulants or antiplatelet therapies within 30 days before screening.
- Have an active bleeding disorder.
- 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 ≥550 mL or donated blood within 56 days or donated plasma within 7 days before baseline.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Neurocrine Clinical Site
Ann Arbor, Michigan, 48109
Neurocrine Clinical Site
Aurora, Colorado, 80045
Neurocrine Clinical Site
Indianapolis, Indiana, 46202
Neurocrine Clinical Site
Minneapolis, Minnesota, 55454
Neurocrine Clinical Site
Philadelphia, Pennsylvania, 19104
Neurocrine Clinical Site
Seattle, Washington, 98105
About this study
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
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 four NBI-74788 dosing regimens, with each regimen administered for 14 days.
- Conditions
- CAH - Congenital Adrenal Hyperplasia
- Being tested
- NBI-74788
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NBI-74788Drug
Capsule, administered daily.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (50 mg QHS) | Experimental | NBI-74788 50 mg once daily at bedtime (QHS) administered orally for 14 consecutive days. Receives: Drug: NBI-74788 |
| Cohort 2 (100 mg QHS) | Experimental | NBI-74788 100 mg once daily at bedtime (QHS) administered orally for 14 consecutive days. Receives: Drug: NBI-74788 |
| Cohort 3 (100 mg QPM) | Experimental | NBI-74788 100 mg once daily in the evening (QPM) administered orally for 14 consecutive days. Receives: Drug: NBI-74788 |
| Cohort 4 (100 mg BID) | Experimental | NBI-74788 100 mg twice daily (BID) administered orally for 14 consecutive days. Receives: Drug: NBI-74788 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change From Baseline to Day 14 in 17-hydroxyprogesterone (17-OHP) Morning Window Averages
Percent changes in 17-OHP were assessed through the collection of samples from 0600 hours to 1000 hours both prior to study drug administration (i.e., at baseline) and after 14 days of study drug dosing. The 3 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Measured: Baseline and Day 14
Secondary outcomes (2)
Percent Change From Baseline to Day 14 in Androstenedione Morning Window Averages
Percent changes in androstenedione were assessed through the collection of samples from 0600 hours to 1000 hours prior to study drug administration (baseline) and after 14 days of study drug dosing. The 3 samples collected at each visit during this morning window were averaged and used to determine the change and percent change from baseline.
Measured: Baseline and Day 14
Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) Morning Window Averages
Percent changes in ACTH were assessed through the collection of samples from 0600 hours to 1000 hours prior to study drug administration (baseline) and after 14 days of study drug dosing. The 3 samples collected at each visit during this morning window were averaged and used to determine the change and percent change from baseline.
Measured: Baseline and Day 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2018
- Study start10 Apr 2018
- Primary completion7 Apr 2020
- Study completion7 Apr 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for CAH - 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurocrine Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Development Lead, Study director, Neurocrine Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03525886
- Sponsor's protocol ID
- NBI-74788-CAH2001
- Status last verified
- Apr 2022
- First submitted
- 2 May 2018
- Last updated
- 3 May 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Auchus RJ, Sarafoglou K, Fechner PY, Vogiatzi MG, Imel EA, Davis SM, Giri N, Sturgeon J, Roberts E, Chan JL, Farber RH. Crinecerfont Lowers Elevated Hormone Markers in Adults With 21-Hydroxylase Deficiency Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2022 Feb 17;107(3):801-812. doi: 10.1210/clinem/dgab749.
PubMed 34653252
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03525886
- Acronym / sponsor ID
- NBI-74788-CAH2001
- Conditions
- CAH - Congenital Adrenal Hyperplasia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Neurocrine BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 10 Apr 2018 (actual)
- Primary completion
- 7 Apr 2020 (actual)
- Study completion
- 7 Apr 2020 (actual)
- First posted
- 16 May 2018 (actual)
- Results
- Posted 3 May 2022
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 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 four NBI-74788 dosing regimens, with each regimen administered for 14 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (50 mg QHS) | Experimental | Drug: NBI-74788NBI-74788 50 mg once daily at bedtime (QHS) administered orally for 14 consecutive days. |
| Cohort 2 (100 mg QHS) | Experimental | Drug: NBI-74788NBI-74788 100 mg once daily at bedtime (QHS) administered orally for 14 consecutive days. |
| Cohort 3 (100 mg QPM) | Experimental | Drug: NBI-74788NBI-74788 100 mg once daily in the evening (QPM) administered orally for 14 consecutive days. |
| Cohort 4 (100 mg BID) | Experimental | Drug: NBI-74788NBI-74788 100 mg twice daily (BID) administered orally for 14 consecutive days. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- 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 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 a prespecified window 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.
Key exclusion criteria (14 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.
- Are 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-74788Capsule, administered daily. | Drug | Cohort 1 (50 mg QHS), Cohort 2 (100 mg QHS), Cohort 3 (100 mg QPM), Cohort 4 (100 mg BID) |
Primary outcome
Percent Change From Baseline to Day 14 in 17-hydroxyprogesterone (17-OHP) Morning Window Averages
Time frame: Baseline and Day 14
Percent changes in 17-OHP were assessed through the collection of samples from 0600 hours to 1000 hours both prior to study drug administration (i.e., at baseline) and after 14 days of study drug dosing. The 3 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
2 secondary outcomes
Percent Change From Baseline to Day 14 in Androstenedione Morning Window Averages
Time frame: Baseline and Day 14
Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) Morning Window Averages
Time frame: Baseline and Day 14
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 34653252Auchus RJ, Sarafoglou K, Fechner PY, Vogiatzi MG, Imel EA, Davis SM, Giri N, Sturgeon J, Roberts E, Chan JL, Farber RH. Crinecerfont Lowers Elevated Hormone Markers in Adults With 21-Hydroxylase Deficiency Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2022 Feb 17;107(3):801-812. doi: 10.1210/clinem/dgab749.
- Individual participant dataSharing planned: No
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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.
Clinical Development Lead study director
Neurocrine Biosciences
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NBI-74788 in people with CAH - Congenital Adrenal Hyperplasia, run by Neurocrine Biosciences at 6 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 8 inclusion and 14 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: finished, as of the sponsor's last update on 3 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 May 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percent Change From Baseline to Day 14 in 17-hydroxyprogesterone (17-OHP) Morning Window Averages (time frame: Baseline and Day 14). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03525886, which also lists 1 linked publication.
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 3 May 2022.