CAH - Congenital Adrenal HyperplasiaPhase 2NCT04045145

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia

A Phase 2 study testing Crinecerfont in people with CAH - Congenital Adrenal Hyperplasia, run by Neurocrine Biosciences at 6 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This is a Phase 2, open-label, multiple-dose, study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 (crinecerfont) in pediatric participants (14 to 17 years of age) with a documented medical diagnosis of classic 21-hydroxylase deficiency congenital adrenal hyperplasia… Read more about this study

Who is it for?: Ages 14 to 17 years
Conditions the study is for: CAH - Congenital Adrenal Hyperplasia
What is being tested?: Crinecerfont
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 8 participants
Where: 6 sitesUnited States
Study dates: 12 Dec 2019to 2 Jul 2021
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 14 to 17 years
All sexes
No healthy volunteers
Child

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.
Participants 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.
Participants 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.

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.

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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 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 ≥250 mL or donated blood within 56 days or donated plasma within 7 days before baseline.

Trial locations and contacts

6 sites in 1 country.

City, postcode or hospital

6 sites in 1 country

United States6 sites

Neurocrine Clinical Site
Ann Arbor, Michigan, 48109
Neurocrine Clinical Site
Aurora, Colorado, 80045
Neurocrine Clinical Site
Minneapolis, Minnesota, 55454
Neurocrine Clinical Site
Philadelphia, Pennsylvania, 19104
Neurocrine Clinical Site
San Diego, California, 92123
Neurocrine Clinical Site
Seattle, Washington, 98105

Other trials in United States

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 Pediatric Subjects With Congenital Adrenal Hyperplasia

Conditions: CAH - Congenital Adrenal Hyperplasia
Being tested: Crinecerfont
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.

CrinecerfontDrug
Also called NBI-74788
Crinecerfont administered orally for 14 consecutive days.

Study groups in NCT04045145
Study group	Role	What they receive
Crinecerfont 50 milligrams (mg) Twice Daily (BID)	Experimental	Crinecerfont administered orally for 14 consecutive days. Receives: Drug: Crinecerfont

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 Serum 17-OHP Concentrations (Morning Window Average)
Percent changes in 17-OHP were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The 2 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Measured: Baseline, Day 14

Secondary outcomes (4)

Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (24-hour Period)
Percent changes in 17-OHP were assessed through the collection of samples for a 24-hour period prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The average of each participant's serial sampling values at each visit where serial sampling was performed was calculated and used to determine the percent change from baseline.
Measured: Baseline, 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 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Measured: Baseline, Day 14
Percent Change From Baseline to Day 14 in Androstenedione (Morning Window Averages)
Percent changes in androstenedione were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Measured: Baseline, Day 14
Percent Change From Baseline to Day 14 in Testosterone (Morning Window Averages)
Percent change in testosterone were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.
Measured: Baseline, Day 14

Timeline

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

12 Dec 20192 Jul 2021

First posted5 Aug 2019
Study start12 Dec 2019
Primary completion2 Jul 2021
Study completion2 Jul 2021

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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 8 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04045145
Sponsor's protocol ID: NBI-74788-CAH2008
Status last verified: Jul 2024
First submitted: 31 Jul 2019
Last updated: 18 Jul 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Newfield RS, Sarafoglou K, Fechner PY, Nokoff NJ, Auchus RJ, Vogiatzi MG, Jeha GS, Giri N, Roberts E, Sturgeon J, Chan JL, Farber RH. Crinecerfont, a CRF1 Receptor Antagonist, Lowers Adrenal Androgens in Adolescents With Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2023 Oct 18;108(11):2871-2878. doi: 10.1210/clinem/dgad270.
PubMed 37216921

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

RecruitingObservational study
Natural History Study of Patients With Excess Androgen
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
Not yet recruitingPhase 1
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of OMS1620
Congenital Adrenal Hyperplasia (CAH)
RecruitingObservational study
Parenting and CAH - 21-hydroxylase Deficiency
CAH - 21-Hydroxylase Deficiency
Not yet recruitingObservational study
Hydrocortisone Modified-release in Adults: Real-world Monitoring of Longitudinal Outcomes in coNgenital Adrenal hYperplasia
CAH - Congenital Adrenal Hyperplasia

All CAH - Congenital Adrenal Hyperplasia trials

Study at a glance

From the registry record, last updated by the sponsor on 18 Jul 2024. 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 Pediatric Subjects With Congenital Adrenal Hyperplasia
ClinicalTrials.gov ID: NCT04045145
Acronym / sponsor ID: NBI-74788-CAH2008
Conditions: CAH - Congenital Adrenal Hyperplasia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 8 participants (actual)
Lead sponsor: Neurocrine BiosciencesIndustry (pharma, biotech, devices)
Study start: 12 Dec 2019 (actual)
Primary completion: 2 Jul 2021 (actual)
Study completion: 2 Jul 2021 (actual)
First posted: 5 Aug 2019 (actual)
Results: Posted 18 Jul 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Crinecerfont 50 milligrams (mg) Twice Daily (BID)	Experimental	Drug: CrinecerfontCrinecerfont administered orally for 14 consecutive days.

Who may qualify

Ages: 14 to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (7 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.
Participants 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.
Participants 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
CrinecerfontAlso: NBI-74788Crinecerfont administered orally for 14 consecutive days.	Drug	Crinecerfont 50 milligrams (mg) Twice Daily (BID)

Primary outcome

Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average)
Time frame: Baseline, Day 14
Percent changes in 17-OHP were assessed through the collection of samples from 0700 hours to 1000 hours (morning window) both prior to study drug administration (i.e., baseline) and after 14 days of study drug dosing. The 2 samples collected during this morning window at each visit were averaged and used to determine the percent change from baseline.

4 secondary outcomes

Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (24-hour Period)
Time frame: Baseline, Day 14
Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) (Morning Window Averages)
Time frame: Baseline, Day 14
Percent Change From Baseline to Day 14 in Androstenedione (Morning Window Averages)
Time frame: Baseline, Day 14
Percent Change From Baseline to Day 14 in Testosterone (Morning Window Averages)
Time frame: Baseline, Day 14

Results and safety

Results posted to the registry on 18 Jul 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Crinecerfont 50 mg BID	8	8

Primary outcome: Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average)

Time frame: Baseline, Day 14. Median (Inter-Quartile Range). Unit: percent change

Measure	Crinecerfont 50 mg n = 8
Value	-69.48 (-89.20 to -23.05)

Population analysed: Pharmacodynamic (PD) analysis set included all enrolled participants who received at least one dose of study drug and had at least one PD parameter measurement at baseline and at least one PD parameter measurement at the Day 14 visit.

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: Day 1 (after dosing) up to Day 35. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Crinecerfont 50 mg	0 of 8 (0%)	6 of 8 (75%)	0 of 8

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 37216921Newfield RS, Sarafoglou K, Fechner PY, Nokoff NJ, Auchus RJ, Vogiatzi MG, Jeha GS, Giri N, Roberts E, Sturgeon J, Chan JL, Farber RH. Crinecerfont, a CRF1 Receptor Antagonist, Lowers Adrenal Androgens in Adolescents With Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2023 Oct 18;108(11):2871-2878. doi: 10.1210/clinem/dgad270.
Individual participant dataSharing planned: No

Related trials for CAH - Congenital Adrenal Hyperplasia

RecruitingObservational study
Natural History Study of Patients With Excess Androgen
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
Not yet recruitingPhase 1
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of OMS1620
Congenital Adrenal Hyperplasia (CAH)
RecruitingObservational study
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CAH - 21-Hydroxylase Deficiency
Not yet recruitingObservational study
Hydrocortisone Modified-release in Adults: Real-world Monitoring of Longitudinal Outcomes in coNgenital Adrenal hYperplasia
CAH - Congenital Adrenal Hyperplasia

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.

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 Crinecerfont 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 7 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 18 Jul 2024. 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 18 Jul 2024). 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 Serum 17-OHP Concentrations (Morning Window Average) (time frame: Baseline, 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 NCT04045145, 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 18 Jul 2024.