Congenital Adrenal HyperplasiaPhase 2NCT03687242
Study to Evaluate the Safety and Efficacy of SPR001 in Subjects 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 8 sites in United States.
Trial summary
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects. 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, CAH - 21-Hydroxylase Deficiency
- What is being tested?
- SPR001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 8 sitesUnited States
- Study dates
- 6 Sep 2018to 9 Aug 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
- Is approved by the Sponsor's Medical Monitor
- Is on a stable regimen of glucocorticoid replacement for ≥30 days before baseline that is expected to remain stable throughout the study
- If screening for this study occurs >3 months after the subject's final follow-up visit in Study SPR001-201, the subject will have serum 17-OHP measured at screening.
- Agrees to follow contraception guidelines
- Is able to understand all study procedures and risks involved and provides written informed consent indicating willingness to comply with all aspects of the protocol
Who cannot join
- Experienced a clinically significant AE considered at least possibly related to SPR001 in Study SPR001-201
- If screening for this study occurs >3 months after the subject's final follow-up visit in Study SPR001-201, the subject will be screened for any clinically significant unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening
- Is at increased risk of suicide
- Clinically significant depression or anxiety at screening or baseline
- Clinically significant abnormal clinical or laboratory assessments must be discussed with the Medical Monitor to determine eligibility for this study.
- Subjects who routinely work overnight shifts require Medical Monitor approval for enrollment
Show all 9Show fewer
- Females who are pregnant or lactating
- Use of any other investigational drug within 30 days or 5 half-lives before screening
- Use of prohibited concomitant medications (including rosiglitazone, testosterone, and strong inhibitors and/or inducers of CYP3A4) within 30 days or 5 half-lives of baseline. Medications metabolized by CYP3A4, 2C8, 2C9, or 2C19, especially those that are sensitive substrates or substrates with narrow therapeutic ranges should be discussed on a case-by-case basis with the Medical Monitor.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 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
Minneapolis, Minnesota, 55414
Spruce Biosciences Clinical Site
Orange, California, 92868
Spruce Biosciences Clinical Site
Philadelphia, Pennsylvania, 19104
Spruce Biosciences Clinical Site
San Diego, California, 92123
About this study
Official title: A 3-Month Phase 2 Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects.
Read the detailed description
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects. To be eligible for this study, an individual must either have completed Study SPR001-201 or meet eligibility criteria for SPR001-naïve subjects. The expected duration of study participation for each subject is up to approximately 5 months. This includes a screening period of ≤30 days, a treatment period of 12 weeks, and a safety follow-up period of 30 days.
- Conditions
- Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia, CAH - 21-Hydroxylase Deficiency
- Being tested
- SPR001
- 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
Open label SPR001
| Study group | Role | What they receive |
|---|---|---|
| SPR001 | Experimental | SPR001 at Dose A Receives: Drug: SPR001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH
Incidence of treatment-emergent adverse events including any serious adverse events, dose-limiting toxicities, and adverse events leading to discontinuation of study drug.
Measured: Over 12 weeks
Secondary outcomes (3)
Change From Baseline in 17-hydroxyprogesterone (17-OHP)
Change from Baseline to Week 12 in 17-OHP following dosing of SPR001 in subjects with CAH. 17-OHP is measured in patient serum sample. Results are expressed as mean percent change from baseline. Reductions in 17-OHP are indicators of better disease control.
Measured: Week 12
Change From Baseline in Androstenedione (A4)
Change from Baseline to Week 12 in androstenedione (A4) following dosing of SPR001 in subjects with CAH. A4 is measured in patient serum. Results are expressed as mean percent change from baseline. Reductions in A4 are indicators of better disease control.
Measured: Week 12
Change From Baseline in Adrenocorticotropic Hormone (ACTH)
Change from Baseline to Week 12 in adrenocorticotropic hormone (ACTH) following dosing of SPR001 in subjects with CAH. ACTH is measured in patient serum. Results are expressed as mean percent change from baseline. Reductions in ACTH are indicators of better disease control.
Measured: Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Sep 2018
- Study start6 Sep 2018
- Primary completion8 Jul 2019
- Study completion9 Aug 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Spruce Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chief Medical Officer, Study director, Spruce Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03687242
- Sponsor's protocol ID
- SPR001-202
- Status last verified
- Mar 2025
- First submitted
- 11 Sep 2018
- Last updated
- 1 Apr 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
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 3-Month Phase 2 Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
- ClinicalTrials.gov ID
- NCT03687242
- Acronym / sponsor ID
- SPR001-202
- Conditions
- Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia, CAH - 21-Hydroxylase Deficiency
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Spruce BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 6 Sep 2018 (actual)
- Primary completion
- 8 Jul 2019 (actual)
- Study completion
- 9 Aug 2019 (actual)
- First posted
- 27 Sep 2018 (actual)
- Results
- Posted 1 Apr 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects.
Detailed description
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects. To be eligible for this study, an individual must either have completed Study SPR001-201 or meet eligibility criteria for SPR001-naïve subjects. The expected duration of study participation for each subject is up to approximately 5 months. This includes a screening period of ≤30 days, a treatment period of 12 weeks, and a safety follow-up period of 30 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SPR001 | Experimental | Drug: SPR001SPR001 at Dose A |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Is approved by the Sponsor's Medical Monitor
- Is on a stable regimen of glucocorticoid replacement for ≥30 days before baseline that is expected to remain stable throughout the study
- If screening for this study occurs >3 months after the subject's final follow-up visit in Study SPR001-201, the subject will have serum 17-OHP measured at screening.
- Agrees to follow contraception guidelines
- Is able to understand all study procedures and risks involved and provides written informed consent indicating willingness to comply with all aspects of the protocol
Key exclusion criteria (9 listed)
- Experienced a clinically significant AE considered at least possibly related to SPR001 in Study SPR001-201
- If screening for this study occurs >3 months after the subject's final follow-up visit in Study SPR001-201, the subject will be screened for any clinically significant unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening
- Is at increased risk of suicide
- Clinically significant depression or anxiety at screening or baseline
- Clinically significant abnormal clinical or laboratory assessments must be discussed with the Medical Monitor to determine eligibility for this study.
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 |
|---|---|---|
| SPR001Open label SPR001 | Drug | SPR001 |
Primary outcome
The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH
Time frame: Over 12 weeks
Incidence of treatment-emergent adverse events including any serious adverse events, dose-limiting toxicities, and adverse events leading to discontinuation of study drug.
3 secondary outcomes
Change From Baseline in 17-hydroxyprogesterone (17-OHP)
Time frame: Week 12
Change From Baseline in Androstenedione (A4)
Time frame: Week 12
Change From Baseline in Adrenocorticotropic Hormone (ACTH)
Time frame: Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 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.
- Individual participant dataSharing planned: No
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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.
Chief Medical Officer study director
Spruce Biosciences
8 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 8 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 5 inclusion and 9 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 1 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 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 1 Apr 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?
The Incidence of Treatment-emergent Adverse Events (Safety and Tolerability) in Subjects With CAH (time frame: Over 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 NCT03687242, 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 1 Apr 2025.