A Phase 3 interventional study of Crinecerfont and Placebo in Congenital Adrenal Hyperplasia, sponsored by Neurocrine Biosciences. Active, not recruiting at 46 sites in 10 countries. Open to participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2025-02-05.
Sponsored by Neurocrine Biosciences · Phase 3, Interventional, and Treatment
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Among 16 studies in Adrenal Hyperplasia, Congenital by Neurocrine Biosciences — 2 open to participants now, 5 under way, 2 of them phase 3. Adrenal Hyperplasia, Congenital · Neurocrine Biosciences.
Among the 16 studies in Adrenal Hyperplasia, Congenital by Neurocrine Biosciences, this one is the 3rd largest by enrollment, is the only one that started in 2021, is one of 6 in phase 3, and was the 12th registered.
101 studies on the registry are indexed under Adrenal Hyperplasia, Congenital; 28 are open to participants now.
Browse Adrenal Hyperplasia, Congenital studies →2 of the 16 studies in Adrenal Hyperplasia, Congenital by Neurocrine Biosciences are open now, in 1 country. Recruiting first, the newest first:
Neurocrine Switzerland GmbH · Sites in United States
Ages 3 mo–47 mo
Neurocrine Biosciences
Ages 18–65
By country, each count listing its studies:
Of the 20 Adrenal Hyperplasia, Congenital studies recruiting now, 10 (50%) set no upper age limit and 9 (45%) stop at 75 or younger; 7 take children under 18; none take healthy volunteers.
This study: 2 Years to 17 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
16 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 4 · Phase 2 7 · Phase 3 6
First in 2007; this study’s year is marked. 1 started in 2021.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 103 is above the median of 37 across 50 interventional studies indexed under Adrenal Hyperplasia, Congenital.
Median enrollment 24 across the 16 interventional studies. Of the 9 that completed or were terminated, 7 (78%) have results posted.
This tab as a report to print or save as PDF → With an account.
Neurocrine Biosciences is the lead sponsor of 87 studies on the registry; 17 are open to participants now.
Of its 35 completed or terminated interventional studies of FDA-regulated products, 23 (66%) have results posted.
None of the studies under way reaches primary completion within a year.
Among the 16 studies in Adrenal Hyperplasia, Congenital by Neurocrine Biosciences, from their registry updates:
This tab as a report to print or save as PDF → With an account.
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks.
Drug: Crinecerfont
Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.
Drug: Crinecerfont · Drug: Placebo
CRF type 1 receptor antagonist
Also known as: NBI-74788, Crenessity
Non-active dosage form
Change From Baseline in Serum Androstenedione at Week 4
Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
Time frame: Baseline, Week 4
Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4
Time frame: Baseline, Week 4
Percent Change From Baseline in Glucocorticoid Daily Dose at Week 28
Time frame: Baseline, Week 28
Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 28
Time frame: Week 28
Change From Baseline in Body Mass Index (BMI) Standard Deviation Score (SDS) at Week 28
Time frame: Baseline, Week 28
Change From Baseline in Mean 24-hour Salivary 17-OHP at Week 28
Time frame: Baseline, Week 28
Change From Baseline in the Ratio of Bone Age to Chronological Age (BA:CA) at Week 28
Time frame: Baseline, Week 28
| Milestone | Placebo | Crinecerfont |
|---|---|---|
| Started | 34 | 69 |
| Received at least 1 dose of study drug | 33 | 69 |
| Completed | 31 | 66 |
| Not completed | 3 | 3 |
| Withdrew: Withdrawal by subject | 3 | 1 |
| Withdrew: Adverse event | 0 | 2 |
| Milestone | Placebo | Crinecerfont |
|---|---|---|
| Started | 31 | 66 |
| Received at least 1 dose of study drug | 31 | 66 |
| Completed | 15 | 29 |
| Not completed | 16 | 37 |
| Withdrew: Ongoing in the study | 16 | 37 |
Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
| ng/dL | Placebo | Crinecerfont |
|---|---|---|
| Change From Baseline in Serum Androstenedione at Week 4 | 70.986 ± 56.198 | -196.789 ± 39.369 |
Results for this outcome have not been posted.
Results for this outcome have not been posted.
Results for this outcome have not been posted.
Results for this outcome have not been posted.
Results for this outcome have not been posted.
Results for this outcome have not been posted.
Collected over Baseline up to Week 28. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| DB Period: Placebo | 0/34 (0%) | 4/33 (12.1%) | 22/33 (66.7%) |
| DB Period: Crinecerfont | 0/69 (0%) | 1/69 (1.4%) | 51/69 (73.9%) |
| Event | DB Period: Placebo | DB Period: Crinecerfont |
|---|---|---|
| VomitingGastrointestinal disorders | 2/33 | 0/69 |
| PharyngitisInfections and infestations | 1/33 | 0/69 |
| Chest painGeneral disorders | 1/33 | 0/69 |
| Gastroenteritis norovirusInfections and infestations | 1/33 | 0/69 |
| GastroenteritisInfections and infestations | 1/33 | 0/69 |
| Gastroenteritis viralInfections and infestations | 1/33 | 0/69 |
| PyrexiaGeneral disorders | 0/33 | 1/69 |
| Event | DB Period: Placebo | DB Period: Crinecerfont |
|---|---|---|
| HeadacheNervous system disorders | 2/33 | 17/69 |
| PyrexiaGeneral disorders | 8/33 | 15/69 |
| VomitingGastrointestinal disorders | 8/33 | 10/69 |
| NasopharyngitisInfections and infestations | 6/33 | 7/69 |
| Upper respiratory tract infectionInfections and infestations | 0/33 | 8/69 |
| Coronavirus infectionInfections and infestations | 3/33 | 5/69 |
| DizzinessNervous system disorders | 3/33 | 4/69 |
| Blood androstenedione increasedInvestigations | 3/33 | 3/69 |
| Blood corticotrophin increasedInvestigations | 3/33 | 2/69 |
| DiarrhoeaGastrointestinal disorders | 3/33 | 2/69 |
Full Analysis Set included all randomized participants.
| Age, Continuous(years) | Placebo | Crinecerfont | Total |
|---|---|---|---|
| Mean | 12.142 ± 3.690 | 12.022 ± 3.405 | 12.061 ± 3.484 |
| Sex: Female, Male(Participants) | Placebo | Crinecerfont | Total |
|---|---|---|---|
| Female | 16 | 34 | 50 |
| Male | 18 | 35 | 53 |
| Ethnicity (NIH/OMB)(Participants) | Placebo | Crinecerfont | Total |
|---|---|---|---|
| Hispanic or Latino | 3 | 8 | 11 |
| Not Hispanic or Latino | 25 | 52 | 77 |
| Unknown or Not Reported | 6 | 9 | 15 |
| Race/Ethnicity, Customized(Participants) | Placebo | Crinecerfont | Total |
|---|---|---|---|
| Race — American Indian or Alaska Native | 0 | 1 | 1 |
| Race — Asian | 2 | 7 | 9 |
| Race — Black or African American | 0 | 3 | 3 |
| Race — White | 23 | 42 | 65 |
| Race — Native Hawaiian or Other Pacific Islander | 0 | 1 | 1 |
| Race — Other | 2 | 3 | 5 |
| Race — Multiple | 1 | 3 | 4 |
| Race — Not Collected | 6 | 9 | 15 |
| Serum Androstenedione Concentration(nanograms (ng)/deciliter (dL)) | Placebo | Crinecerfont | Total |
|---|---|---|---|
| Mean | 483.32 ± 455.64 | 404.96 ± 464.10 | 430.83 ± 460.58 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Jun 2021 to primary completion in Mar 2023, 20 months.
Results were first posted in Feb 2025, 1 year 10 months after primary completion. A results delay was requested in Mar 2024.
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This study is active, not recruiting, as verified in Jan 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Crinecerfont
Adrenal Hyperplasia, Congenital →