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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Crinecerfont 50 milligrams (mg) Twice Daily (BID) | Experimental | Crinecerfont administered orally for 14 consecutive days. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Crinecerfont | Drug | Crinecerfont administered orally for 14 consecutive days. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline, Day 14 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neurocrine Clinical Site | San Diego | California | 92123 | United States | ||
| Neurocrine Clinical Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37216921 | Result | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Crinecerfont 50 mg BID | Participants received crinecerfont 50 milligrams (mg) twice daily (BID) orally for 14 consecutive days. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 10, 2019 | Jun 14, 2024 |
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| 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. | 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. | 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. | Baseline, Day 14 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Neurocrine Clinical Site | Ann Arbor | Michigan | 48109 | United States |
| Neurocrine Clinical Site | Minneapolis | Minnesota | 55454 | United States |
| Neurocrine Clinical Site | Philadelphia | Pennsylvania | 19104 | United States |
| Neurocrine Clinical Site | Seattle | Washington | 98105 | United States |
| Received at Least 1 Dose of Study Drug |
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| COMPLETED |
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| NOT COMPLETED |
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All participants who received at least 1 dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Crinecerfont 50 BID | Participants received crinecerfont 50 mg BID orally for 14 consecutive days. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| 17-hydroxyprogesterone (OHP) (Morning Window Average) | Median | Inter-Quartile Range | nanograms/deciliter (ng/dL) |
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| 17-OHP (24-Hour Average) | Median | Inter-Quartile Range | ng/dL |
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| Androstenedione Morning Window Average) | Median | Inter-Quartile Range | ng/dL |
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| Adrenocorticotropic hormone (ACTH) (Morning Window Average) | Median | Inter-Quartile Range | picograms/milliliter (pg/mL) |
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| Testosterone (Morning Window Average) | Testosterone (morning window average) is presented by female and male participants separately. | Median | Inter-Quartile Range | ng/dL |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | 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. | Posted | Median | Inter-Quartile Range | percent change | Baseline, Day 14 |
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| Secondary | 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. | 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. | Posted | Median | Inter-Quartile Range | percent change | Baseline, Day 14 |
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| Secondary | 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. | 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. | Posted | Median | Inter-Quartile Range | percent change | Baseline, Day 14 |
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| Secondary | 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. | 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. | Posted | Median | Inter-Quartile Range | percent change | Baseline, Day 14 |
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| Secondary | 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. | 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. The overall number of participants analyzed is based on male and female participants. | Posted | Median | Inter-Quartile Range | percent change | Baseline, Day 14 |
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Day 1 (after dosing) up to Day 35
Safety analysis set included all participants who took at least one dose of study drug and had any postbaseline safety data.
Treatment-emergent (after dosing) or post-treatment emergent (up to 21 days after the last dose of study drug) are reported.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Crinecerfont 50 mg | Participants received crinecerfont 50 mg BID orally for 14 consecutive days. | 0 | 8 | 0 | 8 | 6 | 8 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
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| Arthropod sting | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
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| Blepharospasm | Eye disorders | MedDRA 24.0 | Systematic Assessment |
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| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
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| Frequent bowel movements | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Gastritis | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 24.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Neurocrine Medical Information Call Center | Neurocrine Biosciences | 877-641-3461 | medinfo@neurocrine.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 19, 2021 | Jun 14, 2024 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000312 | Adrenal Hyperplasia, Congenital |
| ID | Term |
|---|---|
| D047808 | Adrenogenital Syndrome |
| D012734 | Disorders of Sex Development |
| D014564 | Urogenital Abnormalities |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D043202 | Steroid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000307 | Adrenal Gland Diseases |
| D004700 | Endocrine System Diseases |
| D006058 | Gonadal Disorders |
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| ID | Term |
|---|---|
| C000723083 | crinecerfont |
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| Unknown or Not Reported |
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| Male |
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