A Phase 2 Study to Evaluate the Safety, Efficacy and PK o... | NCT05128942 | Trialant
NCT05128942
Sponsor
Spruce Biosciences
Status
Terminated
Last Update Posted
Dec 24, 2025Actual
Enrollment
67Actual
Phase
Phase 2
Conditions
Congenital Adrenal Hyperplasia
21-OHD
Interventions
Tildacerfont
Countries
United States
Protocol Section
Identification Module
NCT ID
NCT05128942
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
SPR001-205
Secondary IDs
Not provided
Brief Title
A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH
Official Title
A Phase 2 Study to Evaluate the Safety, Efficacy and Pharmacokinetics of SPR001 (Tildacerfont) in Children Aged 2 to 17 Years With Congenital Adrenal Hyperplasia (CAH)
Acronym
Not provided
Organization
Spruce BiosciencesINDUSTRY
Status Module
Record Verification Date
Dec 2025
Overall Recruitment Status or Expanded Access Status
Terminated
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
The SPR001-203 clinical trial did not achieve the primary efficacy endpoint of the absolute change in daily GC dose from baseline at week 24. It was decided to terminate the SPR001-205 in response to the SPR001-203 efficacy data.
Expanded Access Info
No
Start Date
Dec 10, 2021Actual
Primary Completion Date
Jan 31, 2025Actual
Completion Date
Jan 31, 2025Actual
First Submitted Date
Oct 27, 2021
First Submission Date that Met QC Criteria
Nov 9, 2021
First Posted Date
Nov 22, 2021Actual
Results Waived
Not provided
Results First Submitted Date
Sep 18, 2025
Results First Submitted that Met QC Criteria
Dec 7, 2025
Results First Posted Date
Dec 24, 2025Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Dec 7, 2025
Last Update Posted Date
Dec 24, 2025Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Spruce BiosciencesINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
An investigation of the safety and efficacy of tildacerfont in participants with CAH.
Detailed Description
This is a Phase 2 open-label study with up to 10 cohorts that will evaluate the safety, efficacy, and PK of different tildacerfont dosing regimens potentially up to 200mg QD for 12 weeks in children with classic CAH, and up to 400mg BID for 4 weeks in children and adults with classic CAH.
Conditions Module
Conditions
Congenital Adrenal Hyperplasia
21-OHD
Keywords
Pediatric
CAH
Adrenal Disorder
Congenital Adrenal Hyperplasia
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
67Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Cohort 1: Age 11-17 Treatment with Tildacerfont
Experimental
50 mg daily for 12 consecutive weeks.
Drug: Tildacerfont
Cohort 2: Age 11-17 Treatment with Tildacerfont
Experimental
200 mg daily for 12 consecutive weeks.
Drug: Tildacerfont
Cohort 3: Age 2-10 Treatment with Tildacerfont
Experimental
50, 100, or 200 mg daily for 12 consecutive weeks.
Drug: Tildacerfont
Cohort 4: Age >/= 18 Treatment with Tildacerfont
Experimental
200 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Cohort 5: Age >/= 18 Treatment with Tildacerfont
Experimental
300 or 400 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Cohort 6: Age 11-17 Treatment with Tildacerfont
Experimental
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Tildacerfont
Drug
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Cohort 1: Age 11-17 Treatment with Tildacerfont
Cohort 2: Age 11-17 Treatment with Tildacerfont
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0
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
12 weeks
Secondary Outcomes
Measure
Description
Time Frame
Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing
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
12 weeks
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Male and female subjects aged 2+
Diagnosis of CAH due to 21-hydroxylase deficiency (OHD) and/or elevated 17- hydroxyprogesterone (OHP) requiring ongoing GC replacement since diagnosis
Stable dose of GC replacement for at least 1 month prior to screening
Exclusion Criteria:
History of bilateral adrenalectomy or hypopituitarism
Clinically significant unstable medical conditions, illness, or chronic diseases
History of active bleeding disorders
Accepts Healthy Volunteers
No
Sex
All
Sex/Gender Based
Not provided
Sex/Gender Description
Not provided
Minimum Age
2 Years
Maximum Age
Not provided
Standard Ages
ChildAdultOlder Adult
Study Population
Not provided
Sampling Method
Not provided
Contacts/Locations Module
Central Contacts
Not provided
Overall Officials
Name
Affiliation
Role
Will Charlton, MD
Spruce Biosciences
Study Director
Locations
Facility
Status
City
State
ZIP
Country
Contacts
Spruce Study Site
Sacramento
California
95821
United States
Spruce Study Site
References Module
No data available
No data is available for this block.
IPD Sharing Statement Module
Plan to Share IPD
No
Description
Not provided
Types
Not provided
Time Frame
Not provided
Access Criteria
Not provided
URL
Not provided
Results Section
Participant Flow Module
Pre-assignment Details
Doses in Cohort 3 were determined to be weight-based dose equivalents of >/= 200 mg QD. Upon completion of the 4-week dosing on participants from Cohorts 4&6, DMC assessed safety data and provided recommendations on dosing for Cohort 5 (initiated either at 300mg or 400mg BID) and for Cohort 7. Following completion of Cohorts 5&7, DMC reviewed safety data made recommendations on whether to proceed to Cohort 8 and at what dose for each participant (either 300mg or 400mg).
Recruitment Details
Study was terminated before participants could be enrolled in Cohort 9
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Cohort 1: Age 11-17 Treatment With Tildacerfont
50 mg daily for 12 consecutive weeks.
FG001
Cohort 2: Age 11-17 Treatment With Tildacerfont
200 mg daily for 12 consecutive weeks.
Periods
Title
Milestones
Reasons Not Completed
Overall Study
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Not provided
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
0
More Info Module
Limitations and Caveats
Not provided
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Not provided
Uploaded Document Information
Type
Includes Protocol
Includes SAP
Includes ICF
Document Label
Document Date
Document Uploaded Date
Document File Name
Prot
Yes
No
No
Study Protocol
Nov 30, 2023
Sep 2, 2025
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Not provided
Submission Tracking
Estimated Results First Submitted Date
Not provided
Condition Browse Module
MeSH Terms
Intervention Browse Module
No data available
No data is available for this block.
Non-Randomized
Intervention Model
Sequential Assignment
Intervention Model Description
Subjects will be assigned to treatment cohorts by age.
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
None (Open Label)
Masking Description
Not provided
Who Masked
Not provided
200 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Cohort 7: Age 2-10 Treatment with Tildacerfont
Experimental
200 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Cohort 8: Age 11-17 Treatment with Tildacerfont
Experimental
300 or 400 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Cohort 9: Age 2-10 Treatment with Tildacerfont
Experimental
300 or 400 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Cohort 3: Age 2-10 Treatment with Tildacerfont
Cohort 4: Age >/= 18 Treatment with Tildacerfont
Cohort 5: Age >/= 18 Treatment with Tildacerfont
Cohort 6: Age 11-17 Treatment with Tildacerfont
Cohort 7: Age 2-10 Treatment with Tildacerfont
Cohort 8: Age 11-17 Treatment with Tildacerfont
Cohort 9: Age 2-10 Treatment with Tildacerfont
SPR001
Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4
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
4 weeks
Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 Who Achieve A4 Normalization
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
4 weeks or 12 weeks
San Diego
California
92123
United States
Spruce Study Site
Chicago
Illinois
60611
United States
Spruce Study Site
Minneapolis
Minnesota
55454
United States
Spruce Study Site
Buffalo
New York
14203
United States
Spruce Study Site
Providence
Rhode Island
02903
United States
Spruce Study Site
Columbia
South Carolina
29203
United States
Spruce Study Site
Dallas
Texas
75231
United States
Spruce Study Site
Edinburg
Texas
78539
United States
Spruce Study Site
Fort Worth
Texas
76104
United States
Spruce Study Site
Salt Lake City
Utah
84113
United States
Spruce Study Site
Charlottesville
Virginia
22903
United States
Spruce Study Site
Richmond
Virginia
23284
United States
FG002
Cohort 3: Age 2-10 Treatment With Tildacerfont
50, 100, or 200 mg daily for 12 consecutive weeks.
FG003
Cohort 4: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
FG004
Cohort 5: Treatment With Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
FG005
Cohort 6: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
FG006
Cohort 7: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
FG007
Cohort 8: Treatment With Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
FG008
Cohort 9: Treatment With Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
FG0007 subjects
FG0017 subjects
FG00218 subjects
FG0032 subjects
FG0046 subjects
FG0058 subjects
FG00612 subjects
FG0077 subjects
FG0080 subjects
COMPLETED
FG0002 subjects
FG0011 subjects
FG0021 subjects
FG0030 subjects
FG0041 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
NOT COMPLETED
FG0005 subjects
FG0016 subjects
FG00217 subjects
FG0032 subjects
FG0045 subjects
FG0058 subjects
FG00612 subjects
FG0077 subjects
FG0080 subjects
Type
Comment
Reasons
Withdrawal by Subject
FG0001 subjects
FG0010 subjects
FG0021 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
Other
FG0002 subjects
FG0010 subjects
FG0022 subjects
FG0030 subjects
FG004
Termination by Sponsor
FG0002 subjects
FG0016 subjects
FG00214 subjects
FG0032 subjects
FG004
Adverse Event
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Lost to Follow-up
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Cohort 1: Age 11-17 Treatment With Tildacerfont
50 mg daily for 12 consecutive weeks.
BG001
Cohort 2: Age 11-17 Treatment With Tildacerfont
200 mg daily for 12 consecutive weeks.
BG002
Cohort 3: Age 2-10 Treatment With Tildacerfont
50, 100, or 200 mg daily for 12 consecutive weeks.
BG003
Cohort 4: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
BG004
Cohort 5: Treatment With Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
BG005
Cohort 6: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
BG006
Cohort 7: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
BG007
Cohort 8: Treatment With Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
BG008
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG0007
BG0017
BG00218
BG0032
BG0046
BG0058
BG00612
BG0077
BG00867
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
Title
Measurements
BG00013.238± 1.688
BG00114.381± 2.539
BG0027.722± 2.3279
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0007
BG0013
BG002
Ethnicity (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Hispanic or Latino
BG0001
BG0012
BG002
Race
Count of Participants
Participants
Title
Denominators
Categories
Asian
Title
Measurements
BG0000
BG0011
BG002
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
Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0
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
For Cohorts 5 and 8, the Statistical Analysis Plan pre-specified to analyze the data separately "per Cohort," without regard to dose.
Posted
Count of Participants
Participants
12 weeks
ID
Title
Description
OG000
Cohort 1: Age 11-17 Treatment With Tildacerfont
50 mg daily for 12 consecutive weeks.
OG001
Cohort 2: Age 11-17 Treatment With Tildacerfont
200 mg daily for 12 consecutive weeks.
OG002
Cohort 3: Age 2-10 Treatment With Tildacerfont
50, 100, or 200 mg daily for 12 consecutive weeks.
OG003
Cohort 4: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
OG004
Cohort 5: Treatment With Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
OG005
Cohort 6: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
OG006
Cohort 7: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
OG007
Cohort 8: Treatment With Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
Units
Counts
Participants
OG0007
OG0017
OG00218
OG003
Title
Denominators
Categories
Title
Measurements
OG0004
OG0016
OG00218
OG003
Secondary
Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing
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
For Cohorts 5 and 8, the Statistical Analysis Plan pre-specified to analyze the data separately "per Cohort," without regard to dose.
Posted
Count of Participants
Participants
12 weeks
ID
Title
Description
OG000
Cohort 1: Age 11-17 Treatment With Tildacerfont
50 mg daily for 12 consecutive weeks.
OG001
Cohort 2: Age 11-17 Treatment With Tildacerfont
200 mg daily for 12 consecutive weeks.
OG002
Cohort 3: Age 2-10 Treatment With Tildacerfont
50, 100, or 200 mg daily for 12 consecutive weeks.
OG003
Cohort 4: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
Secondary
Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4
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
For Cohorts 5 and 8, the Statistical Analysis Plan pre-specified to analyze the data separately "per Cohort," without regard to dose.
Posted
Count of Participants
Participants
4 weeks
ID
Title
Description
OG000
Cohort 1: Age 11-17 Treatment With Tildacerfont
50 mg daily for 12 consecutive weeks.
OG001
Cohort 2: Age 11-17 Treatment With Tildacerfont
200 mg daily for 12 consecutive weeks.
OG002
Cohort 3: Age 2-10 Treatment With Tildacerfont
50, 100, or 200 mg daily for 12 consecutive weeks.
OG003
Cohort 4: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
Secondary
Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 Who Achieve A4 Normalization
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
For Cohorts 5 and 8, the Statistical Analysis Plan pre-specified to analyze the data separately "per Cohort," without regard to dose.
Posted
Count of Participants
Participants
4 weeks or 12 weeks
ID
Title
Description
OG000
Cohort 1: Age 11-17 Treatment With Tildacerfont
50 mg daily for 12 consecutive weeks.
OG001
Cohort 2: Age 11-17 Treatment With Tildacerfont
200 mg daily for 12 consecutive weeks.
OG002
Cohort 3: Age 2-10 Treatment With Tildacerfont
50, 100, or 200 mg daily for 12 consecutive weeks.
OG003
Cohort 4: Treatment With Tildacerfont
200 mg twice daily for 4 consecutive weeks.
Time Frame
24 weeks
Description
Not provided
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Cohort 1: Age 11-17 Treatment With Tildacerfont
50 mg daily for 12 consecutive weeks.
0
7
0
7
4
7
EG001
Cohort 2: Age 11-17 Treatment With Tildacerfont
200 mg daily for 12 consecutive weeks.
0
7
1
7
6
7
EG002
Cohort 3: Age 2-10 Treatment With Tildacerfont
50, 100, or 200 mg daily for 12 consecutive weeks.