Trial · NCT05128942
A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH
NCT05128942, Phase 2, Terminated, Spruce Biosciences; results first posted 2025-12-24
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH. Trials, record NCT05128942. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05128942
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH
- NCT number
- NCT05128942
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 67 participants, actual
- Lead sponsor
- Spruce Biosciences
- Conditions
- Congenital Adrenal Hyperplasia, 21-OHD
- Interventions
- Tildacerfont
- First posted
- 2021-11-22
- Last updated
- 2025-12-24
- Results first posted
- 2025-12-24
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 | 12 weeks | To evaluate safety of tildacerfont in participants with CAH as measured by number of subjects with adverse events following dosing by CTCAE version 5.0 |
| Secondary | Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing | 12 weeks | To determine the efficacy of tildacerfont on disease control or reduction of GC use in participants with classic CAH as measured by number of subjects who achieve a reduction in A4 or reduction in GC dosing during treatment period |
| Secondary | Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 | 4 weeks | To determine the efficacy of tildacerfont on disease control in participants with classic CAH measured by the number of participants with elevated baseline A4 who achieve reduction in A4 at week 4 |
| Secondary | Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 Who Achieve A4 Normalization | 4 weeks or 12 weeks | To determine the efficacy of tildacerfont on disease control in participants with classic CAH measured by the number of participants with elevated baseline A4 who achieve reduction in A4 at week 4 or week 12 |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 | 12 weeks | For Cohorts 5 and 8, the Statistical Analysis Plan pre-specified to analyze the data separately per Cohort, without regard to dose. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing | 12 weeks | For Cohorts 5 and 8, the Statistical Analysis Plan pre-specified to analyze the data separately per Cohort, without regard to dose. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 | 4 weeks | For Cohorts 5 and 8, the Statistical Analysis Plan pre-specified to analyze the data separately per Cohort, without regard to dose. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 Who Achieve A4 Normalization | 4 weeks or 12 weeks | For Cohorts 5 and 8, the Statistical Analysis Plan pre-specified to analyze the data separately per Cohort, without regard to dose. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group40
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 | Cohort 1: Age 11-17 Treatment With Tildacerfont | 4 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 | Cohort 2: Age 11-17 Treatment With Tildacerfont | 6 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 | Cohort 3: Age 2-10 Treatment With Tildacerfont | 18 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 | Cohort 4: Treatment With Tildacerfont | 2 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 | Cohort 5: Treatment With Tildacerfont | 3 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 | Cohort 6: Treatment With Tildacerfont | 8 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 | Cohort 7: Treatment With Tildacerfont | 12 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 | Cohort 8: Treatment With Tildacerfont | 5 | Participants | ||
| Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing | Cohort 1: Age 11-17 Treatment With Tildacerfont | 5 | Participants | ||
| Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing | Cohort 2: Age 11-17 Treatment With Tildacerfont | 4 | Participants |
1 to 10 of 40Source ClinicalTrials.gov
See all 40 results by groupParticipant flow27
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: Age 11-17 Treatment With Tildacerfont | 7 | |
| Overall Study | STARTED | Cohort 2: Age 11-17 Treatment With Tildacerfont | 7 | |
| Overall Study | STARTED | Cohort 3: Age 2-10 Treatment With Tildacerfont | 18 | |
| Overall Study | STARTED | Cohort 4: Treatment With Tildacerfont | 2 | |
| Overall Study | STARTED | Cohort 5: Treatment With Tildacerfont | 6 | |
| Overall Study | STARTED | Cohort 6: Treatment With Tildacerfont | 8 | |
| Overall Study | STARTED | Cohort 7: Treatment With Tildacerfont | 12 | |
| Overall Study | STARTED | Cohort 8: Treatment With Tildacerfont | 7 | |
| Overall Study | STARTED | Cohort 9: Treatment With Tildacerfont | 0 | |
| Overall Study | COMPLETED | Cohort 1: Age 11-17 Treatment With Tildacerfont | 2 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 participant flowAdverse events1,170
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 | Neutropenia | Blood and lymphatic system disorders | Cohort 1: Age 11-17 Treatment With Tildacerfont | 0 | 7 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 2: Age 11-17 Treatment With Tildacerfont | 0 | 7 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 3: Age 2-10 Treatment With Tildacerfont | 1 | 18 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 4: Treatment With Tildacerfont | 0 | 2 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 5: 300 mg Tildacerfont | 0 | 1 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 5: 400 mg Tildacerfont | 0 | 5 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 6: Treatment With Tildacerfont | 0 | 8 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 7: Treatment With Tildacerfont | 0 | 12 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 8: 300 mg Tildacerfont | 0 | 3 | |
| other | Neutropenia | Blood and lymphatic system disorders | Cohort 8: 400 mg Tildacerfont | 0 | 4 |
1 to 10 of 1,170Source ClinicalTrials.gov
See all 1,170 adverse eventsAdverse event totals30
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: Age 11-17 Treatment With Tildacerfont | 0 | 7 |
| Total, all-cause mortality | Cohort 2: Age 11-17 Treatment With Tildacerfont | 0 | 7 |
| Total, all-cause mortality | Cohort 3: Age 2-10 Treatment With Tildacerfont | 0 | 18 |
| Total, all-cause mortality | Cohort 4: Treatment With Tildacerfont | 0 | 2 |
| Total, all-cause mortality | Cohort 5: 300 mg Tildacerfont | 0 | 1 |
| Total, all-cause mortality | Cohort 5: 400 mg Tildacerfont | 0 | 5 |
| Total, all-cause mortality | Cohort 6: Treatment With Tildacerfont | 0 | 8 |
| Total, all-cause mortality | Cohort 7: Treatment With Tildacerfont | 0 | 12 |
| Total, all-cause mortality | Cohort 8: 300 mg Tildacerfont | 0 | 3 |
| Total, all-cause mortality | Cohort 8: 400 mg Tildacerfont | 0 | 4 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license 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/NCT05128942
GET https://conductscience.org/api/trials/v1/NCT05128942/results?page=2