Congenital Adrenal HyperplasiaPhase 2NCT03257462

Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

A Phase 2 study testing SPR001 in people with Congenital Adrenal Hyperplasia and CAH - Congenital Adrenal Hyperplasia, run by Spruce Biosciences at 9 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

This is a multicenter Phase 2, multiple dose, dose escalation study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of SPR001 in adult patients with classic congenital adrenal hyperplasia (CAH). Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
What is being tested?: SPR001
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 24 participants
Where: 9 sitesUnited States
Study dates: 12 Jul 2017to 29 Mar 2019
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 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Male and female patients age 18 or older.
Documented diagnosis of classic CAH due to 21-hydroxylase deficiency
Elevated 17-OHP at screening
On a stable glucocorticoid replacement regimen for a minimum of 30 days

Who cannot join

Clinically significant unstable medical condition, illness, or chronic disease
Clinically significant psychiatric disorder.
Clinically significant abnormal laboratory finding or assessment
History of bilateral adrenalectomy or hypopituitarism
Pregnant or nursing females
Use of any other investigational drug within 30 days
Unable to understand and comply with the study procedures, understand the risks, and/or unwilling to provide written informed consent.

Trial locations and contacts

9 sites in 1 country.

City, postcode or hospital

9 sites in 1 country

United States9 sites

Spruce Biosciences Clinical Site
Ann Arbor, Michigan, 48109
Spruce Biosciences Clinical Site
Atlanta, Georgia, 30046
Spruce Biosciences Clinical Site
Indianapolis, Indiana, 46202
Spruce Biosciences Clinical Site
Las Vegas, Nevada, 89148
Spruce Biosciences Clinical Site
Melbourne, Florida, 32935
Spruce Biosciences Clinical Site
Minneapolis, Minnesota, 55414
Spruce Biosciences Clinical Site
Orange, California, 92123
Spruce Biosciences Clinical Site
Philadelphia, Pennsylvania, 19104
Spruce Biosciences Clinical Site
San Diego, California, 92123

Other trials in United States

About this study

Official title: A Phase 2, Multiple-Dose, Dose-Escalation Study to Evaluate the Safety and Efficacy of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia (CAH)

Read the detailed description

This is a 6-week, multiple-dose, dose escalation study of SPR001 for the treatment of adults with classic CAH. After screening, eligible patients will be enrolled into a 6-week treatment period followed by a 4-week washout/safety follow-up period.

It is initially planned that up to approximately 18 patients in 2 dose cohorts will be enrolled. Additional patients or dose groups may be considered based upon specific safety, PK/PD, and/or efficacy findings, or if an active dose has not yet been reached.

SPR001 will be administered as an oral daily dose. Patients will undergo titration of SPR001 through three escalating dosage strengths at 2-week intervals. Patients will have overnight PK/PD assessments performed at baseline, which include an pre-dose overnight assessment and a post-dose overnight assessment for PK/PD following administration of the first dose. At the end of each 2-week dosing period, patients will return for single overnight visits for steady-state PK/PD assessments.

A follow-up outpatient visit will occur 30 days after their last dose.

Conditions: Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Being tested: SPR001
Keywords: 17-hydroxyprogesterone
Medical subject headings: Adrenal Hyperplasia, Congenital

What participants receive

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

SPR001Drug
SPR001 Capsules

Study groups in NCT03257462
Study group	Role	What they receive
Cohort A	Experimental	The first cohort of 9 patients will be administered SPR001 at dose strength of Dose A daily for 2 weeks, and escalating through Dose B per day for 2 weeks and Dose C per day for 2 weeks. Receives: Drug: SPR001
Cohort B	Experimental	Cohort B will begin enrollment after Cohort A has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort B will be determined by an interim review of safety and PK/PD data from from Cohort A. Receives: Drug: SPR001
Cohort C	Experimental	Cohort C will begin enrollment after Cohort B has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort C will be determined by an interim review of safety and PK/PD data from from Cohort A and B. Receives: Drug: SPR001

What is being measured

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

Safety of SPR001 in Patients With CAH
Incidence of treatment-emergent adverse events, changes from Baseline to End-of-study in clinical laboratory parameters, physical examination findings, vital signs, ECG parameters
Measured: 6 weeks
Change in 17-hydroxyprogesterone
Change in 17-hydroxyprogesterone from Baseline to End-of-study. Results are expressed as mean percent change from baseline. Reductions in 17-OHP are indicators of better disease control.
Measured: Cohort A: Baseline/2a (Day -1-0), First dose/2b (Day 0-1), Visit 3 (Day 13-14), Visit 4 (Day 27-28), Visit 5 (Day 41-42). Cohort B and Cohort C: Visit 2 (Day 0-1), Visit 3 (Day 8), Visit 4 (Day 14-15), Visit 5 (Last dose +30d)

Secondary outcomes (3)

Changes in Pharmacodynamic (PD) Markers
Changes in adrenocorticotropic hormone (ACTH) and androstenedione (A4) from Baseline to End-of-study are measured in patient serum. Results are expressed as a mean percentage change from baseline. A negative change indicates improvement.
Measured: Cohort A: Visit 2a (Day -1 to 0), Visit 2b (Days 0-1), Visit 3 (Day 13-14), Visit 4 (Day 27-28), Visit 5 (Day 41 to 42). Cohorts B+C: Visit 2 (Day 0-1), Visit 3 (Day 8), Visit 4 (Day 27-28), Visit 5 (+30 days after last dose)
Pharmacokinetic Parameter - Maximum Plasma Concentration (Cmax)
To evaluate the pharmacokinetic (PK) parameter of maximum plasma concentration (Cmax) of SPR001 in patients with CAH.
Measured: Serial PK sampling was performed at the end of the 2 weeks for all cohorts and dose levels
Pharmacokinetic Parameter - Area Under the Concentration-time Curve (AUC)
To evaluate the PK parameter of area under the concentration-time curve (AUC) of SPR001 in patients with CAH
Measured: For Cohort A, serial blood collections were made for PK measurements on Day 1 for 200 mg SD and at Week 2 for all QD dose levels. In Cohorts B and C, serial blood samples were drawn for PK measurements at the end of the 2-week treatment period.

Timeline

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

12 Jul 201729 Mar 2019

First posted22 Aug 2017
Study start12 Jul 2017
Primary completion2 Mar 2019
Study completion29 Mar 2019

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.
Publications (1)Papers the sponsor has linked to this study.
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
Masking: None (open label)
Enrolment: 24 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03257462
Sponsor's protocol ID: SPR001-201
Status last verified: Oct 2025
First submitted: 15 Aug 2017
Last updated: 22 Oct 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Sarafoglou K, Barnes CN, Huang M, Imel EA, Madu IJ, Merke DP, Moriarty D, Nakhle S, Newfield RS, Vogiatzi MG, Auchus RJ. Tildacerfont in Adults With Classic Congenital Adrenal Hyperplasia: Results from Two Phase 2 Studies. J Clin Endocrinol Metab. 2021 Oct 21;106(11):e4666-e4679. doi: 10.1210/clinem/dgab438.
PubMed 34146101

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 2/3
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Natural History Study of Patients With Excess Androgen
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
RecruitingPhase 3
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
RecruitingPhase 4
Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)
Mild Autonomous Cortisol Secretion (MACS)

All Congenital Adrenal Hyperplasia trials

Study at a glance

From the registry record, last updated by the sponsor on 22 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Multiple-Dose, Dose-Escalation Study to Evaluate the Safety and Efficacy of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia (CAH)
ClinicalTrials.gov ID: NCT03257462
Acronym / sponsor ID: SPR001-201
Conditions: Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Keywords: 17-hydroxyprogesterone
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 24 participants (actual)
Lead sponsor: Spruce BiosciencesIndustry (pharma, biotech, devices)
Study start: 12 Jul 2017 (actual)
Primary completion: 2 Mar 2019 (actual)
Study completion: 29 Mar 2019 (actual)
First posted: 22 Aug 2017 (actual)
Results: Posted 22 Oct 2025

Clinical summary

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

Detailed description

A follow-up outpatient visit will occur 30 days after their last dose.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 3

Arm	Type	Receives
Cohort A	Experimental	Drug: SPR001The first cohort of 9 patients will be administered SPR001 at dose strength of Dose A daily for 2 weeks, and escalating through Dose B per day for 2 weeks and Dose C per day for 2 weeks.
Cohort B	Experimental	Drug: SPR001Cohort B will begin enrollment after Cohort A has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort B will be determined by an interim review of safety and PK/PD data from from Cohort A.
Cohort C	Experimental	Drug: SPR001Cohort C will begin enrollment after Cohort B has been fully enrolled. Starting dose selection and the stepwise dosing paradigm for Cohort C will be determined by an interim review of safety and PK/PD data from from Cohort A and B.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Male and female patients age 18 or older.
Documented diagnosis of classic CAH due to 21-hydroxylase deficiency
Elevated 17-OHP at screening
On a stable glucocorticoid replacement regimen for a minimum of 30 days

Key exclusion criteria (7 listed)

Clinically significant unstable medical condition, illness, or chronic disease
Clinically significant psychiatric disorder.
Clinically significant abnormal laboratory finding or assessment
History of bilateral adrenalectomy or hypopituitarism
Pregnant or nursing females

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
SPR001SPR001 Capsules	Drug	Cohort A, Cohort B, Cohort C

Primary outcome

Safety of SPR001 in Patients With CAH
Time frame: 6 weeks
Incidence of treatment-emergent adverse events, changes from Baseline to End-of-study in clinical laboratory parameters, physical examination findings, vital signs, ECG parameters
Change in 17-hydroxyprogesterone
Time frame: Cohort A: Baseline/2a (Day -1-0), First dose/2b (Day 0-1), Visit 3 (Day 13-14), Visit 4 (Day 27-28), Visit 5 (Day 41-42). Cohort B and Cohort C: Visit 2 (Day 0-1), Visit 3 (Day 8), Visit 4 (Day 14-15), Visit 5 (Last dose +30d)
Change in 17-hydroxyprogesterone from Baseline to End-of-study. Results are expressed as mean percent change from baseline. Reductions in 17-OHP are indicators of better disease control.

3 secondary outcomes

Changes in Pharmacodynamic (PD) Markers
Time frame: Cohort A: Visit 2a (Day -1 to 0), Visit 2b (Days 0-1), Visit 3 (Day 13-14), Visit 4 (Day 27-28), Visit 5 (Day 41 to 42). Cohorts B+C: Visit 2 (Day 0-1), Visit 3 (Day 8), Visit 4 (Day 27-28), Visit 5 (+30 days after last dose)
Pharmacokinetic Parameter - Maximum Plasma Concentration (Cmax)
Time frame: Serial PK sampling was performed at the end of the 2 weeks for all cohorts and dose levels
Pharmacokinetic Parameter - Area Under the Concentration-time Curve (AUC)
Time frame: For Cohort A, serial blood collections were made for PK measurements on Day 1 for 200 mg SD and at Week 2 for all QD dose levels. In Cohorts B and C, serial blood samples were drawn for PK measurements at the end of the 2-week treatment period.

Results and safety

Results posted to the registry on 22 Oct 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
Period 1: Cohort A	10	9
Period 2: Cohort B	0	0
Period 3: Cohort C	0	0
Period 4: Cohort D	0	0

Primary outcome: Safety of SPR001 in Patients With CAH

Time frame: 6 weeks. Count of participants. Unit: Participants

Measure	Cohort A n = 10	Cohort B n = 9	Cohort C n = 7
Value	6	5	4

Population analysed: This full analysis set includes all subjects who received at least 1 dose of study drug. The FAS was used to analyze all safety data

Primary outcome: Change in 17-hydroxyprogesterone

Time frame: Cohort A: Baseline/2a (Day -1-0), First dose/2b (Day 0-1), Visit 3 (Day 13-14), Visit 4 (Day 27-28), Visit 5 (Day 41-42). Cohort B and Cohort C: Visit 2 (Day 0-1), Visit 3 (Day 8), Visit 4 (Day 14-15), Visit 5 (Last dose +30d). Mean (Standard Deviation). Unit: percentage of mean change from baseline

Measure	Cohort B n = 7	Cohort C n = 7	Cohort A - Dose A n = 9	Cohort A - Dose B n = 7	Cohort A - Dose C n = 8
Value	-47.79 (63.300)	-33.19 (41.604)	22.16 (172.298)	-23.71 (64.146)	-13.79 (74.742)

Population analysed: Only subjects with both baseline and post baseline (week 2) assessments were summarized. Baseline values were collected at 8am on Visit 2a (Day -1) and post-baseline Week 2 values were collected at 8a on Visit 3, Visit 4, or Visit 5. A negative change indicates improvement.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: 6-week treatment period followed by a 4-week washout/safety follow-up period. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cohort A - Dose A	0 of 10 (0%)	6 of 10 (60%)	0 of 10
Cohort A - Dose B	0 of 10 (0%)	3 of 10 (30%)	0 of 10
Cohort A - Dose C	0 of 10 (0%)	3 of 9 (33.3%)	0 of 9
Cohort B	0 of 9 (0%)	5 of 9 (55.6%)	0 of 9
Cohort C	0 of 7 (0%)	4 of 7 (57.1%)	0 of 7

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 34146101Sarafoglou K, Barnes CN, Huang M, Imel EA, Madu IJ, Merke DP, Moriarty D, Nakhle S, Newfield RS, Vogiatzi MG, Auchus RJ. Tildacerfont in Adults With Classic Congenital Adrenal Hyperplasia: Results from Two Phase 2 Studies. J Clin Endocrinol Metab. 2021 Oct 21;106(11):e4666-e4679. doi: 10.1210/clinem/dgab438.
On-line clinical study-related information for patients, including pre-screening questionnaire.Link provided by the sponsor.
Individual participant dataSharing planned: No

Related trials for Congenital Adrenal Hyperplasia

RecruitingPhase 2/3
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Natural History Study of Patients With Excess Androgen
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
RecruitingPhase 3
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
RecruitingPhase 4
Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)
Mild Autonomous Cortisol Secretion (MACS)

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.

Spruce Chief Medical Officer, MD study director

Spruce Biosciences

Richard Auchus, MD, PhD principal investigator

University of Michigan

9 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 SPR001 in people with Congenital Adrenal Hyperplasia and CAH - Congenital Adrenal Hyperplasia, run by Spruce Biosciences at 9 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 4 inclusion and 7 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: finished, as of the sponsor's last update on 22 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 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 22 Oct 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?

Safety of SPR001 in Patients With CAH (time frame: 6 weeks). The study lists 2 primary outcomes in all. 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 NCT03257462, which also lists 1 linked publication.

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 22 Oct 2025.