Study 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.
A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH
1 other identifier
interventional
67
1 country
13
Brief Summary
An investigation of the safety and efficacy of tildacerfont in participants with CAH.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Dec 2021
Typical duration for phase_2
13 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 27, 2021
CompletedFirst Posted
Study publicly available on registry
November 22, 2021
CompletedStudy Start
First participant enrolled
December 10, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 31, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
January 31, 2025
CompletedResults Posted
Study results publicly available
December 24, 2025
CompletedDecember 24, 2025
December 1, 2025
3.1 years
October 27, 2021
September 18, 2025
December 7, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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 (3)
Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing
12 weeks
Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4
4 weeks
Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 Who Achieve A4 Normalization
4 weeks or 12 weeks
Study Arms (9)
Cohort 1: Age 11-17 Treatment with Tildacerfont
EXPERIMENTAL50 mg daily for 12 consecutive weeks.
Cohort 2: Age 11-17 Treatment with Tildacerfont
EXPERIMENTAL200 mg daily for 12 consecutive weeks.
Cohort 3: Age 2-10 Treatment with Tildacerfont
EXPERIMENTAL50, 100, or 200 mg daily for 12 consecutive weeks.
Cohort 4: Age >/= 18 Treatment with Tildacerfont
EXPERIMENTAL200 mg twice daily for 4 consecutive weeks.
Cohort 5: Age >/= 18 Treatment with Tildacerfont
EXPERIMENTAL300 or 400 mg twice daily for 4 consecutive weeks.
Cohort 6: Age 11-17 Treatment with Tildacerfont
EXPERIMENTAL200 mg twice daily for 4 consecutive weeks.
Cohort 7: Age 2-10 Treatment with Tildacerfont
EXPERIMENTAL200 mg twice daily for 4 consecutive weeks.
Cohort 8: Age 11-17 Treatment with Tildacerfont
EXPERIMENTAL300 or 400 mg twice daily for 4 consecutive weeks.
Cohort 9: Age 2-10 Treatment with Tildacerfont
EXPERIMENTAL300 or 400 mg twice daily for 4 consecutive weeks.
Interventions
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- History of bilateral adrenalectomy or hypopituitarism
- Clinically significant unstable medical conditions, illness, or chronic diseases
- History of active bleeding disorders
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (13)
Spruce Study Site
Sacramento, California, 95821, United States
Spruce Study Site
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
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Spruce CMO, MD
- Organization
- Spruce BioSciences Inc.
Study Officials
- STUDY DIRECTOR
Will Charlton, MD
Spruce Biosciences
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 27, 2021
First Posted
November 22, 2021
Study Start
December 10, 2021
Primary Completion
January 31, 2025
Study Completion
January 31, 2025
Last Updated
December 24, 2025
Results First Posted
December 24, 2025
Record last verified: 2025-12
Data Sharing
- IPD Sharing
- Will not share