TerminatedNCT05128942Updated Dec 24, 2025Results posted

A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH

A Phase 2 interventional study of Tildacerfont in Congenital Adrenal Hyperplasia and 21-OHD, sponsored by Spruce Biosciences. Terminated at 13 sites in United States. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2025-12-24.

Sponsored by Spruce Biosciences · Phase 2, Interventional, and Treatment

Why this study was terminated

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.

Phase: Phase 2
Study type: Interventional
Enrollment: 67
Allocation: Non-randomized
Ages: 2 Years and older
Sex: All

Study summary

An investigation of the safety and efficacy of tildacerfont in participants with CAH.

Read the 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 studied

Congenital Adrenal Hyperplasia
21-OHD

Browse trials for

Keywords

Pediatric
CAH
Adrenal Disorder
Congenital Adrenal Hyperplasia

In context

Adrenal Hyperplasia, Congenital

101 studies on the registry are indexed under Adrenal Hyperplasia, Congenital; 28 are open to participants now.

This study's enrollment of 67 is above the median of 37 across 50 interventional studies indexed under Adrenal Hyperplasia, Congenital.

Browse Adrenal Hyperplasia, Congenital studies →

Lead sponsor

Spruce Biosciences is the lead sponsor of 8 studies on the registry; 2 are open to participants now.

Of its 6 completed or terminated interventional studies of FDA-regulated products, 6 (100%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 2 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

Exclusion Criteria:

History of bilateral adrenalectomy or hypopituitarism
unstable medical conditions, illness, or chronic diseases
History of active bleeding disorders

Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Clinically significant: Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Study design

Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomized
Intervention model: Sequential assignment
Masking: None (open label)
Enrollment: 67 participants (actual)

Study arms

Experimental
Cohort 1: Age 11-17 Treatment with Tildacerfont
50 mg daily for 12 consecutive weeks.
Drug: Tildacerfont
Experimental
Cohort 2: Age 11-17 Treatment with Tildacerfont
200 mg daily for 12 consecutive weeks.
Drug: Tildacerfont
Experimental
Cohort 3: Age 2-10 Treatment with Tildacerfont
50, 100, or 200 mg daily for 12 consecutive weeks.
Drug: Tildacerfont
Experimental
Cohort 4: Age >/= 18 Treatment with Tildacerfont
200 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Experimental
Cohort 5: Age >/= 18 Treatment with Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Experimental
Cohort 6: Age 11-17 Treatment with Tildacerfont
200 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Experimental
Cohort 7: Age 2-10 Treatment with Tildacerfont
200 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Experimental
Cohort 8: Age 11-17 Treatment with Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont
Experimental
Cohort 9: Age 2-10 Treatment with Tildacerfont
300 or 400 mg twice daily for 4 consecutive weeks.
Drug: Tildacerfont

Interventions

DrugTildacerfont
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.
Also known as: SPR001

What researchers measure

Primary outcomes

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
Time frame: 12 weeks

Secondary outcomes

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
Time frame: 12 weeks
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
Time frame: 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
Time frame: 4 weeks or 12 weeks

Results

Posted Dec 24, 2025

Participant flow

Study was terminated before participants could be enrolled in Cohort 9

Participant flow — Overall Study
Milestone	Cohort 1: Age 11-17 Treatment With Tildacerfont	Cohort 2: Age 11-17 Treatment With Tildacerfont	Cohort 3: Age 2-10 Treatment With Tildacerfont	Cohort 4: Treatment With Tildacerfont	Cohort 5: Treatment With Tildacerfont	Cohort 6: Treatment With Tildacerfont	Cohort 7: Treatment With Tildacerfont	Cohort 8: Treatment With Tildacerfont
Started	7	7	18	2	6	8	12	7
Completed	2	1	1	0	1	0	0	0
Not completed	5	6	17	2	5	8	12	7
Withdrew: Withdrawal by subject	1	0	1	0	0	0	0	0
Withdrew: Other	2	0	2	0	0	0	0	0
Withdrew: Termination by sponsor	2	6	14	2	4	8	11	6
Withdrew: Adverse event	0	0	0	0	0	0	1	1
Withdrew: Lost to follow-up	0	0	0	0	1	0	0	0

Outcome measures

PrimaryNumber 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

Time frame:: 12 weeks
Reported as:: Count of participants · Participants

Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0
Participants	Cohort 1: Age 11-17 Treatment With Tildacerfont	Cohort 2: Age 11-17 Treatment With Tildacerfont	Cohort 3: Age 2-10 Treatment With Tildacerfont	Cohort 4: Treatment With Tildacerfont	Cohort 5: Treatment With Tildacerfont	Cohort 6: Treatment With Tildacerfont	Cohort 7: Treatment With Tildacerfont	Cohort 8: Treatment With Tildacerfont
Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0	4	6	18	2	3	8	12	5

SecondaryProportion 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

Time frame:: 12 weeks
Reported as:: Count of participants · Participants

Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing
Participants	Cohort 1: Age 11-17 Treatment With Tildacerfont	Cohort 2: Age 11-17 Treatment With Tildacerfont	Cohort 3: Age 2-10 Treatment With Tildacerfont	Cohort 4: Treatment With Tildacerfont	Cohort 5: Treatment With Tildacerfont	Cohort 6: Treatment With Tildacerfont	Cohort 7: Treatment With Tildacerfont	Cohort 8: Treatment With Tildacerfont
Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing	5	4	14	2	0	4	1	0

SecondaryProportion 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

Time frame:: 4 weeks
Reported as:: Count of participants · Participants

Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4
Participants	Cohort 1: Age 11-17 Treatment With Tildacerfont	Cohort 2: Age 11-17 Treatment With Tildacerfont	Cohort 3: Age 2-10 Treatment With Tildacerfont	Cohort 4: Treatment With Tildacerfont	Cohort 5: Treatment With Tildacerfont	Cohort 6: Treatment With Tildacerfont	Cohort 7: Treatment With Tildacerfont	Cohort 8: Treatment With Tildacerfont
Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4	4	3	9	0	1	6	5	0

SecondaryProportion 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

Time frame:: 4 weeks or 12 weeks
Reported as:: Count of participants · Participants

Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 Who Achieve A4 Normalization
Participants	Cohort 1: Age 11-17 Treatment With Tildacerfont	Cohort 2: Age 11-17 Treatment With Tildacerfont	Cohort 3: Age 2-10 Treatment With Tildacerfont	Cohort 4: Treatment With Tildacerfont	Cohort 5: Treatment With Tildacerfont	Cohort 6: Treatment With Tildacerfont	Cohort 7: Treatment With Tildacerfont	Cohort 8: Treatment With Tildacerfont
Week 4	1	0	1	0	0	1	1	0
Week 12	0	0	2	0	0	1	0	0

Adverse events

Collected over 24 weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Cohort 1: Age 11-17 Treatment With Tildacerfont	0/7 (0%)	0/7 (0%)	4/7 (57.1%)
Cohort 2: Age 11-17 Treatment With Tildacerfont	0/7 (0%)	1/7 (14.3%)	6/7 (85.7%)
Cohort 3: Age 2-10 Treatment With Tildacerfont	0/18 (0%)	1/18 (5.6%)	18/18 (100%)
Cohort 4: Treatment With Tildacerfont	0/2 (0%)	0/2 (0%)	2/2 (100%)
Cohort 5: 300 mg Tildacerfont	0/1 (0%)	0/1 (0%)	0/1 (0%)
Cohort 5: 400 mg Tildacerfont	0/5 (0%)	0/5 (0%)	3/5 (60%)
Cohort 6: Treatment With Tildacerfont	0/8 (0%)	0/8 (0%)	8/8 (100%)
Cohort 7: Treatment With Tildacerfont	0/12 (0%)	1/12 (8.3%)	12/12 (100%)
Cohort 8: 300 mg Tildacerfont	0/3 (0%)	0/3 (0%)	2/3 (66.7%)
Cohort 8: 400 mg Tildacerfont	0/4 (0%)	0/4 (0%)	3/4 (75%)

Most frequent serious events

Most frequent serious events
Event	Cohort 1: Age 11-17 Treatment With Tildacerfont	Cohort 2: Age 11-17 Treatment With Tildacerfont	Cohort 3: Age 2-10 Treatment With Tildacerfont	Cohort 4: Treatment With Tildacerfont	Cohort 5: 300 mg Tildacerfont	Cohort 5: 400 mg Tildacerfont	Cohort 6: Treatment With Tildacerfont	Cohort 7: Treatment With Tildacerfont	Cohort 8: 300 mg Tildacerfont	Cohort 8: 400 mg Tildacerfont
Adrenal insufficiencyEndocrine disorders	0/7	1/7	0/18	0/2	0/1	—	0/8	1/12	0/3	—
GastroenteritisGastrointestinal disorders	0/7	0/7	1/18	0/2	0/1	—	0/8	0/12	0/3	—

Most frequent other events

Showing 10 of 115

Most frequent other events
Event	Cohort 1: Age 11-17 Treatment With Tildacerfont	Cohort 2: Age 11-17 Treatment With Tildacerfont	Cohort 3: Age 2-10 Treatment With Tildacerfont	Cohort 4: Treatment With Tildacerfont	Cohort 5: 300 mg Tildacerfont	Cohort 5: 400 mg Tildacerfont	Cohort 6: Treatment With Tildacerfont	Cohort 7: Treatment With Tildacerfont	Cohort 8: 300 mg Tildacerfont	Cohort 8: 400 mg Tildacerfont
Eye irritationEye disorders	0/7	0/7	0/18	1/2	0/1	0/5	0/8	0/12	0/3	0/4
VomitingGastrointestinal disorders	0/7	0/7	7/18	0/2	0/1	1/5	1/8	5/12	0/3	2/4
DiarrheaGastrointestinal disorders	0/7	0/7	0/18	1/2	0/1	0/5	0/8	0/12	0/3	0/4
PyrexiaGeneral disorders	1/7	0/7	9/18	1/2	0/1	0/5	1/8	4/12	0/3	0/4
Viral upper respiratory tract infectionInfections and infestations	0/7	0/7	0/18	1/2	0/1	0/5	1/8	1/12	0/3	0/4
Nasal congestionRespiratory, thoracic and mediastinal disorders	1/7	0/7	0/18	1/2	0/1	0/5	1/8	0/12	0/3	0/4
Dermatitis contactSkin and subcutaneous tissue disorders	0/7	0/7	0/18	1/2	0/1	0/5	0/8	0/12	0/3	0/4
Rash papularSkin and subcutaneous tissue disorders	0/7	0/7	0/18	1/2	0/1	0/5	0/8	0/12	0/3	0/4
HeadacheNervous system disorders	2/7	1/7	7/18	0/2	0/1	0/5	1/8	0/12	0/3	1/4
NasopharyngitisInfections and infestations	1/7	0/7	6/18	0/2	0/1	0/5	0/8	3/12	0/3	1/4

Baseline characteristics

Age, Continuous
Age, Continuous(years)	Cohort 1: Age 11-17 Treatment With Tildacerfont	Cohort 2: Age 11-17 Treatment With Tildacerfont	Cohort 3: Age 2-10 Treatment With Tildacerfont	Cohort 4: Treatment With Tildacerfont	Cohort 5: Treatment With Tildacerfont	Cohort 6: Treatment With Tildacerfont	Cohort 7: Treatment With Tildacerfont	Cohort 8: Treatment With Tildacerfont	Total
Mean	13.238 ± 1.688	14.381 ± 2.539	7.722 ± 2.3279	25.125 ± 8.8978	26.333 ± 8.2635	14.354 ± 2.0792	6.882 ± 2.5185	15.214 ± 1.7192	12.604 ± 6.7977

Sex: Female, Male
Sex: Female, Male(Participants)	Cohort 1: Age 11-17 Treatment With Tildacerfont	Cohort 2: Age 11-17 Treatment With Tildacerfont	Cohort 3: Age 2-10 Treatment With Tildacerfont	Cohort 4: Treatment With Tildacerfont	Cohort 5: Treatment With Tildacerfont	Cohort 6: Treatment With Tildacerfont	Cohort 7: Treatment With Tildacerfont	Cohort 8: Treatment With Tildacerfont	Total
Female	7	3	9	1	4	6	7	4	41
Male	0	4	9	1	2	2	5	3	26

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	Cohort 1: Age 11-17 Treatment With Tildacerfont	Cohort 2: Age 11-17 Treatment With Tildacerfont	Cohort 3: Age 2-10 Treatment With Tildacerfont	Cohort 4: Treatment With Tildacerfont	Cohort 5: Treatment With Tildacerfont	Cohort 6: Treatment With Tildacerfont	Cohort 7: Treatment With Tildacerfont	Cohort 8: Treatment With Tildacerfont	Total
Hispanic or Latino	1	2	7	1	3	2	3	2	21
Not Hispanic or Latino	6	5	11	1	3	6	9	5	46
Unknown or Not Reported	0	0	0	0	0	0	0	0	0

Race
Race(Participants)	Cohort 1: Age 11-17 Treatment With Tildacerfont	Cohort 2: Age 11-17 Treatment With Tildacerfont	Cohort 3: Age 2-10 Treatment With Tildacerfont	Cohort 4: Treatment With Tildacerfont	Cohort 5: Treatment With Tildacerfont	Cohort 6: Treatment With Tildacerfont	Cohort 7: Treatment With Tildacerfont	Cohort 8: Treatment With Tildacerfont	Total
Asian	0	1	1	1	0	0	0	0	3
Black or African American	0	1	1	0	0	1	0	0	3
White	7	5	14	0	6	7	12	7	58
Other	0	0	2	1	0	0	0	0	3

Study locations

13 sites

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

References and documents

Study documents

Study protocol ↗ · Nov 30, 2023
Statistical analysis plan ↗ · Jul 24, 2024
Informed consent form ↗ · Apr 26, 2024

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Dec 24, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT05128942
Lead sponsor: Spruce Biosciences
Responsible party: Sponsor
First posted: Nov 22, 2021
Start date: Dec 10, 2021
Primary completion: Jan 31, 2025
Completion: Jan 31, 2025
Results posted: Dec 24, 2025
Last update: Dec 24, 2025

Study contacts

Will Charlton, MD

study director · Spruce Biosciences

Oversight

Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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This study is terminated, as verified in Dec 2025. You cannot join it, but the record below documents what was studied.

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