Trial · NCT04045145
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia
NCT04045145, Phase 2, Completed, Neurocrine Biosciences; results first posted 2024-07-18
3 sourcestrials-2026-11-12-0.4.0
Cite this record
ConductScience Library. Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia. Trials, record NCT04045145. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04045145
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia
- NCT number
- NCT04045145
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 8 participants, actual
- Lead sponsor
- Neurocrine Biosciences
- Conditions
- CAH - Congenital Adrenal Hyperplasia
- Interventions
- Crinecerfont
- First posted
- 2019-08-05
- Last updated
- 2024-07-18
- Results first posted
- 2024-07-18
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average) | 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. |
| Secondary | Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (24-hour Period) | Baseline, Day 14 | 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. |
| Secondary | Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) (Morning Window Averages) | Baseline, Day 14 | 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. |
| Secondary | Percent Change From Baseline to Day 14 in Androstenedione (Morning Window Averages) | Baseline, Day 14 | 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. |
| Secondary | Percent Change From Baseline to Day 14 in Testosterone (Morning Window Averages) | Baseline, Day 14 | 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. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average) | Baseline, Day 14 | 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. | percent change | MEDIAN |
| SECONDARY | Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (24-hour Period) | Baseline, Day 14 | 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. | percent change | MEDIAN |
| SECONDARY | Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) (Morning Window Averages) | Baseline, Day 14 | 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. | percent change | MEDIAN |
| SECONDARY | Percent Change From Baseline to Day 14 in Androstenedione (Morning Window Averages) | Baseline, Day 14 | 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. | percent change | MEDIAN |
| SECONDARY | Percent Change From Baseline to Day 14 in Testosterone (Morning Window Averages) | Baseline, Day 14 | 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. | percent change | MEDIAN |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (Morning Window Average) | Crinecerfont 50 mg | -69.48 | Inter-Quartile Range -89.20 to -23.05 | percent change | |
| Percent Change From Baseline to Day 14 in Serum 17-OHP Concentrations (24-hour Period) | Crinecerfont 50 mg | -76.0 | Inter-Quartile Range -82.34 to -29.74 | percent change | |
| Percent Change From Baseline to Day 14 in Adrenocorticotropic Hormone (ACTH) (Morning Window Averages) | Crinecerfont 50 mg | -57.12 | Inter-Quartile Range -70.08 to -28.45 | percent change | |
| Percent Change From Baseline to Day 14 in Androstenedione (Morning Window Averages) | Crinecerfont 50 mg | -58.27 | Inter-Quartile Range -82.22 to -11.99 | percent change | |
| Percent Change From Baseline to Day 14 in Testosterone (Morning Window Averages) | Crinecerfont 50 mg | Female | -76.18 | Inter-Quartile Range -83.53 to -28.57 | percent change |
| Percent Change From Baseline to Day 14 in Testosterone (Morning Window Averages) | Crinecerfont 50 mg | Male | 1.39 | Inter-Quartile Range -2.67 to 49.55 | percent change |
Source ClinicalTrials.gov
Participant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Crinecerfont 50 mg BID | 8 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Crinecerfont 50 mg BID | 8 | |
| Overall Study | COMPLETED | Crinecerfont 50 mg BID | 8 | |
| Overall Study | NOT COMPLETED | Crinecerfont 50 mg BID | 0 |
Source ClinicalTrials.gov
Adverse events11
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Headache | Nervous system disorders | Crinecerfont 50 mg | 2 | 8 | |
| other | Arthropod sting | Injury, poisoning and procedural complications | Crinecerfont 50 mg | 1 | 8 | |
| other | Blepharospasm | Eye disorders | Crinecerfont 50 mg | 1 | 8 | |
| other | Dermatitis contact | Skin and subcutaneous tissue disorders | Crinecerfont 50 mg | 1 | 8 | |
| other | Dizziness | Nervous system disorders | Crinecerfont 50 mg | 1 | 8 | |
| other | Frequent bowel movements | Gastrointestinal disorders | Crinecerfont 50 mg | 1 | 8 | |
| other | Gastritis | Gastrointestinal disorders | Crinecerfont 50 mg | 1 | 8 | |
| other | Myalgia | Musculoskeletal and connective tissue disorders | Crinecerfont 50 mg | 1 | 8 | |
| other | Nasopharyngitis | Infections and infestations | Crinecerfont 50 mg | 1 | 8 | |
| other | Pyrexia | General disorders | Crinecerfont 50 mg | 1 | 8 |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Crinecerfont 50 mg | 0 | 8 |
| Total, other adverse events | Crinecerfont 50 mg | 6 | 8 |
| Total, serious adverse events | Crinecerfont 50 mg | 0 | 8 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37216921 | RESULT |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04045145
GET https://conductscience.org/api/trials/v1/NCT04045145/registered