Congenital Adrenal HyperplasiaPhase 2NCT05128942

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

A Phase 2 study testing Tildacerfont in people with Congenital Adrenal Hyperplasia and 21-OHD, run by Spruce Biosciences at 13 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

An investigation of the safety and efficacy of tildacerfont in participants with CAH. Read more about this study

Who is it for?: Ages 2 years and older
Conditions the study is for: Congenital Adrenal Hyperplasia, 21-OHD
What is being tested?: Tildacerfont
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 67 participants
Where: 13 sitesUnited States
Study dates: 10 Dec 2021to 31 Jan 2025
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 2 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

13 sites in 1 country.

City, postcode or hospital

13 sites in 1 country

United States13 sites

Spruce Study Site
Buffalo, New York, 14203
Spruce Study Site
Charlottesville, Virginia, 22903
Spruce Study Site
Chicago, Illinois, 60611
Spruce Study Site
Columbia, South Carolina, 29203
Spruce Study Site
Dallas, Texas, 75231
Spruce Study Site
Edinburg, Texas, 78539
Spruce Study Site
Fort Worth, Texas, 76104
Spruce Study Site
Minneapolis, Minnesota, 55454
Spruce Study Site
Providence, Rhode Island, 02903
Spruce Study Site
Richmond, Virginia, 23284
Spruce Study Site
Sacramento, California, 95821
Spruce Study Site
Salt Lake City, Utah, 84113
Spruce Study Site
San Diego, California, 92123

Other trials in United States

About this study

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)

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: Congenital Adrenal Hyperplasia, 21-OHD
Being tested: Tildacerfont
Keywords: Pediatric, CAH, Adrenal Disorder, Congenital Adrenal Hyperplasia
Medical subject headings: Adrenal Hyperplasia, Congenital, Adrenal Gland Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

TildacerfontDrug
Also called SPR001
Oral tablet formulation taken once daily in combination with glucocorticoid therapy.

Study groups in NCT05128942
Study group	Role	What they receive
Cohort 1: Age 11-17 Treatment with Tildacerfont	Experimental	50 mg daily for 12 consecutive weeks. Receives: Drug: Tildacerfont
Cohort 2: Age 11-17 Treatment with Tildacerfont	Experimental	200 mg daily for 12 consecutive weeks. Receives: Drug: Tildacerfont
Cohort 3: Age 2-10 Treatment with Tildacerfont	Experimental	50, 100, or 200 mg daily for 12 consecutive weeks. Receives: Drug: Tildacerfont
Cohort 4: Age >/= 18 Treatment with Tildacerfont	Experimental	200 mg twice daily for 4 consecutive weeks. Receives: Drug: Tildacerfont
Cohort 5: Age >/= 18 Treatment with Tildacerfont	Experimental	300 or 400 mg twice daily for 4 consecutive weeks. Receives: Drug: Tildacerfont
Cohort 6: Age 11-17 Treatment with Tildacerfont	Experimental	200 mg twice daily for 4 consecutive weeks. Receives: Drug: Tildacerfont
Cohort 7: Age 2-10 Treatment with Tildacerfont	Experimental	200 mg twice daily for 4 consecutive weeks. Receives: Drug: Tildacerfont
Cohort 8: Age 11-17 Treatment with Tildacerfont	Experimental	300 or 400 mg twice daily for 4 consecutive weeks. Receives: Drug: Tildacerfont
Cohort 9: Age 2-10 Treatment with Tildacerfont	Experimental	300 or 400 mg twice daily for 4 consecutive weeks. Receives: Drug: Tildacerfont

What is being measured

The primary outcome is the main question the study is designed to answer.

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
Measured: 12 weeks

Secondary outcomes (3)

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
Measured: 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
Measured: 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
Measured: 4 weeks or 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

10 Dec 202131 Jan 2025

First posted22 Nov 2021
Study start10 Dec 2021
Primary completion31 Jan 2025
Study completion31 Jan 2025

Why it 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.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Model description: Subjects will be assigned to treatment cohorts by age.
Masking: None (open label)
Enrolment: 67 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05128942
Sponsor's protocol ID: SPR001-205
Status last verified: Dec 2025
First submitted: 27 Oct 2021
Last updated: 24 Dec 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 4
Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)
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RecruitingPhase 2/3
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingPhase 3
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
Not yet recruitingObservational study
Development of Healthcare Transition for Patients With Congenital Adrenal Hyperplasia
Congenital Adrenal Hyperplasia
Not yet recruitingPhase 2
An Extension Trial of Asedebart in Participants With Congenital Adrenal Hyperplasia
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All Congenital Adrenal Hyperplasia trials

Study at a glance

From the registry record, last updated by the sponsor on 24 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

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)
ClinicalTrials.gov ID: NCT05128942
Acronym / sponsor ID: SPR001-205
Conditions: Congenital Adrenal Hyperplasia, 21-OHD
Keywords: Pediatric, CAH, Adrenal Disorder, Congenital Adrenal Hyperplasia
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason 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.
Enrolment: 67 participants (actual)
Lead sponsor: Spruce BiosciencesIndustry (pharma, biotech, devices)
Study start: 10 Dec 2021 (actual)
Primary completion: 31 Jan 2025 (actual)
Study completion: 31 Jan 2025 (actual)
First posted: 22 Nov 2021 (actual)
Results: Posted 24 Dec 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

Detailed description

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 9
Model description: Subjects will be assigned to treatment cohorts by age.

Arm	Type	Receives
Cohort 1: Age 11-17 Treatment with Tildacerfont	Experimental	Drug: Tildacerfont50 mg daily for 12 consecutive weeks.
Cohort 2: Age 11-17 Treatment with Tildacerfont	Experimental	Drug: Tildacerfont200 mg daily for 12 consecutive weeks.
Cohort 3: Age 2-10 Treatment with Tildacerfont	Experimental	Drug: Tildacerfont50, 100, or 200 mg daily for 12 consecutive weeks.
Cohort 4: Age >/= 18 Treatment with Tildacerfont	Experimental	Drug: Tildacerfont200 mg twice daily for 4 consecutive weeks.
Cohort 5: Age >/= 18 Treatment with Tildacerfont	Experimental	Drug: Tildacerfont300 or 400 mg twice daily for 4 consecutive weeks.
Cohort 6: Age 11-17 Treatment with Tildacerfont	Experimental	Drug: Tildacerfont200 mg twice daily for 4 consecutive weeks.
Cohort 7: Age 2-10 Treatment with Tildacerfont	Experimental	Drug: Tildacerfont200 mg twice daily for 4 consecutive weeks.
Cohort 8: Age 11-17 Treatment with Tildacerfont	Experimental	Drug: Tildacerfont300 or 400 mg twice daily for 4 consecutive weeks.
Cohort 9: Age 2-10 Treatment with Tildacerfont	Experimental	Drug: Tildacerfont300 or 400 mg twice daily for 4 consecutive weeks.

Who may qualify

Ages: 2 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

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

Key exclusion criteria (3 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
TildacerfontAlso: SPR001Oral tablet formulation taken once daily in combination with glucocorticoid therapy.	Drug	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: 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

Primary outcome

Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0
Time frame: 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

3 secondary outcomes

Proportion of Participants Who Achieve a Reduction in Androstenedione (A4) or Reduction in Glucocorticoid (GC) Dosing
Time frame: 12 weeks
Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4
Time frame: 4 weeks
Proportion of Participants With Elevated Baseline A4 Who Achieve a Reduction in A4 Who Achieve A4 Normalization
Time frame: 4 weeks or 12 weeks

Results and safety

Results posted to the registry on 24 Dec 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Cohort 1: Age 11-17 Treatment With Tildacerfont	7	2
Cohort 2: Age 11-17 Treatment With Tildacerfont	7	1
Cohort 3: Age 2-10 Treatment With Tildacerfont	18	1
Cohort 4: Treatment With Tildacerfont	2	0
Cohort 5: Treatment With Tildacerfont	6	1
Cohort 6: Treatment With Tildacerfont	8	0
Cohort 7: Treatment With Tildacerfont	12	0
Cohort 8: Treatment With Tildacerfont	7	0
Cohort 9: Treatment With Tildacerfont	0	0

Primary outcome: Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0

Time frame: 12 weeks. Count of participants. Unit: Participants

Measure	Cohort 1: Age 11-17 Treatment With Tildacerfont n = 7	Cohort 2: Age 11-17 Treatment With Tildacerfont n = 7	Cohort 3: Age 2-10 Treatment With Tildacerfont n = 18	Cohort 4: Treatment With Tildacerfont n = 2	Cohort 5: Treatment With Tildacerfont n = 6	Cohort 6: Treatment With Tildacerfont n = 8	Cohort 7: Treatment With Tildacerfont n = 12	Cohort 8: Treatment With Tildacerfont n = 7
Value	4	6	18	2	3	8	12	5

Population analysed: For Cohorts 5 and 8, the Statistical Analysis Plan pre-specified to analyze the data separately "per Cohort," without regard to dose.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: 24 weeks. Other adverse events reported at 0% frequency or more

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

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Dec 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Congenital Adrenal Hyperplasia

RecruitingPhase 4
Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)
Mild Autonomous Cortisol Secretion (MACS)
RecruitingPhase 2/3
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
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RecruitingPhase 3
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Will Charlton, MD study director

Spruce Biosciences

13 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Tildacerfont in people with Congenital Adrenal Hyperplasia and 21-OHD, run by Spruce Biosciences at 13 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 24 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 24 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Treatment-emergent Adverse Event (TEAE) as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 (time frame: 12 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05128942.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Dec 2025.