A Phase 2 interventional study of Crinecerfont in CAH - Congenital Adrenal Hyperplasia, sponsored by Neurocrine Biosciences. Completed at 6 sites in United States. Open to participants aged 14 Years to 17 Years. Per ClinicalTrials.gov, last updated 2024-07-18.
Sponsored by Neurocrine Biosciences · Phase 2, 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 13th largest by enrollment, is the only one that started in 2019, is one of 6 in phase 2, and was the 10th 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: 14 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 2019.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 8 is below 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 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 (CAH).
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.
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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
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.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
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.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
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.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
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.
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.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
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.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
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.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
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 administered orally for 14 consecutive days.
Drug: Crinecerfont
Crinecerfont administered orally for 14 consecutive days.
Also known as: NBI-74788
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.
Time frame: Baseline, Day 14
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline, Day 14
| Milestone | Crinecerfont 50 mg BID |
|---|---|
| Started | 8 |
| Received at least 1 dose of study drug | 8 |
| Completed | 8 |
| Not completed | 0 |
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.
| percent change | Crinecerfont 50 mg |
|---|---|
| Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average) | -69.48 (-89.20 to -23.05) |
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.
| percent change | Crinecerfont 50 mg |
|---|---|
| Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (24-hour Period) | -76.0 (-82.34 to -29.74) |
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.
| percent change | Crinecerfont 50 mg |
|---|---|
| Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) (Morning Window Averages) | -57.12 (-70.08 to -28.45) |
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.
| percent change | Crinecerfont 50 mg |
|---|---|
| Percent Change From Baseline to Day 14 in Androstenedione (Morning Window Averages) | -58.27 (-82.22 to -11.99) |
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.
| percent change | Crinecerfont 50 mg |
|---|---|
| Female | -76.18 (-83.53 to -28.57) |
| Male | 1.39 (-2.67 to 49.55) |
Collected over Day 1 (after dosing) up to Day 35. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Crinecerfont 50 mg | 0/8 (0%) | 0/8 (0%) | 6/8 (75%) |
| Event | Crinecerfont 50 mg |
|---|---|
| HeadacheNervous system disorders | 2/8 |
| Arthropod stingInjury, poisoning and procedural complications | 1/8 |
| BlepharospasmEye disorders | 1/8 |
| Dermatitis contactSkin and subcutaneous tissue disorders | 1/8 |
| DizzinessNervous system disorders | 1/8 |
| Frequent bowel movementsGastrointestinal disorders | 1/8 |
| GastritisGastrointestinal disorders | 1/8 |
| MyalgiaMusculoskeletal and connective tissue disorders | 1/8 |
| NasopharyngitisInfections and infestations | 1/8 |
| PyrexiaGeneral disorders | 1/8 |
All participants who received at least 1 dose of study drug.
| Age, Continuous(years) | Crinecerfont 50 BID |
|---|---|
| Mean | 15.0 ± 0.9 |
| Sex: Female, Male(Participants) | Crinecerfont 50 BID |
|---|---|
| Female | 5 |
| Male | 3 |
| Ethnicity (NIH/OMB)(Participants) | Crinecerfont 50 BID |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 7 |
| Unknown or Not Reported | 0 |
| Race/Ethnicity, Customized(Participants) | Crinecerfont 50 BID |
|---|---|
| Asian | 1 |
| White | 7 |
| 17-hydroxyprogesterone (OHP) (Morning Window Average)(nanograms/deciliter (ng/dL)) | Crinecerfont 50 BID |
|---|---|
| Median | 7703.74 (3344.01 to 10467.50) |
| 17-OHP (24-Hour Average)(ng/dL) | Crinecerfont 50 BID |
|---|---|
| Median | 2739.74 (1264.84 to 5114.45) |
| Androstenedione Morning Window Average)(ng/dL) | Crinecerfont 50 BID |
|---|---|
| Median | 367.88 (207.45 to 600.73) |
| Adrenocorticotropic hormone (ACTH) (Morning Window Average)(picograms/milliliter (pg/mL)) | Crinecerfont 50 BID |
|---|---|
| Median | 226.23 (37.48 to 414.73) |
1 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Dec 2019 to primary completion in Jul 2021, 18 months.
Results were first posted in Jul 2024, 3 years after primary completion. A results delay was requested in Jun 2022.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: Crinecerfont
Adrenal Hyperplasia, Congenital →