Polycystic Ovary SyndromePhase 2NCT05370521
A Study of Safety and Efficacy of Tildacerfont in Females With Polycystic Ovary Syndrome and Elevated Adrenal Androgens
A Phase 2 study testing Tildacerfont in people with Polycystic Ovary Syndrome, run by Spruce Biosciences at 21 sites in United States.
Trial summary
An investigation of the safety and efficacy of tildacerfont in women with PCOS and elevated adrenal androgens Read more about this study
- Who is it for?
- Ages 18 to 40 years, women only
- Conditions the study is for
- Polycystic Ovary Syndrome
- What is being tested?
- Tildacerfont
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 21 sitesUnited States
- Study dates
- 15 May 2022to 31 Aug 2023
- 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 to 40 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- Female subjects aged 18 to 40 years old at Screening visit
- Diagnosis of PCOS (either historical or during Screening) according to NIH (1990) criteria
- DHEAS level > age-matched upper limit of normal (ULN) at Screening visit
- Agree to follow industry standard contraception guidelines within protocol
Who cannot join
- Evidence of:
- History of congenital adrenal hyperplasia (CAH), Cushing's syndrome, pituitary or adrenal disease
- Clinically significant hyperprolactinemia
- Thyroid stimulating hormone (TSH) <0.1 mU/mL or >4.5mU/mL at Screening
- Cortisol levels concerning for adrenal insufficiency
- Other findings suggestive of secondary cause for anovulation and/or hyperandrogenemia
Show all 11Show fewer
- Total testosterone levels >140 ng/dL, DHEAS >650 mcg/dL, virilization or other signs or symptoms concerning for ovarian hyperthecosis or androgen-secreting tumors
- Medical conditions that require glucocorticoid treatment within 30 days of screening and throughout the duration of the study
- Clinically significant unstable medical conditions, illness, or chronic diseases
- Prior hysterectomy or bilateral oophorectomy
- Females who are pregnant or nursing
Trial locations and contacts
21 sites in 1 country.
21 sites in 1 country
United States21 sites
Spruce Study Site
Bedford, Texas, 76022
Spruce Study Site
Boston, Massachusetts, 02115
Spruce Study Site
Cincinnati, Ohio, 45219
Spruce Study Site
Clearwater, Florida, 33759
Spruce Study Site
Cleveland, Ohio, 44106
Spruce Study Site
Fall River, Massachusetts, 02720
Spruce Study Site
Houston, Texas, 77024
Spruce Study Site
Houston, Texas, 77030
Spruce Study Site
Idaho Falls, Idaho, 83404
Spruce Study Site
Lake Worth, Florida, 33461
Spruce Study Site
Miami, Florida, 33173
Spruce Study Site
Morgantown, West Virginia, 26506
Spruce Study Site
New Haven, Connecticut, 06519
Spruce Study Site
Philadelphia, Pennsylvania, 19104
Spruce Study Site
Raleigh, North Carolina, 27612
Spruce Study Site
Sacramento, California, 95817
Spruce Study Site
San Francisco, California, 94158
Spruce Study Site
Southfield, Michigan, 48034
Spruce Study Site
Wichita, Kansas, 67226
Spruce Study Site
Williamsville, New York, 14221
Spruce Study Site
Winter Park, Florida, 32792
About this study
Official title: A Randomized, Placebo-Controlled, Dose Escalation Study to Evaluate the Safety and Efficacy of Tildacerfont in Adult Subjects With Polycystic Ovary Syndrome (PCOS) and Elevated Adrenal Androgens
An investigation of the safety and efficacy of tildacerfont in women with PCOS and elevated adrenal androgens
Read the detailed description
This was a phase 2 study to evaluate the efficacy, safety and tolerability of 3 doses of tildacerfont in approximately 39 women with PCOS and elevated adrenal androgens assessed by elevated DHEAS levels. Subjects were randomized 2:1 (tildacerfont:placebo) at baseline. The study consisted of 3 consecutive 4-week treatment periods at each dose level. Duration of participation was approximately 25 weeks.
- Conditions
- Polycystic Ovary Syndrome
- Being tested
- Tildacerfont, Placebo
- Keywords
- PCOS, Elevated Adrenal Androgen, Adrenal Disorder, Polycystic Ovary Syndrome, Womens health
- Medical subject headings
- Polycystic Ovary Syndrome, 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.
PlaceboDrug
Non-active dosage form taken once daily.
| Study group | Role | What they receive |
|---|---|---|
| Treatment with Tildacerfont | Experimental | Subjects randomized in this arm received 4 weeks of 50 mg of oral tildacerfont tablet, followed by 4 weeks of 100 mg oral tildacerfont tablet, and then 4 weeks of 200 mg oral tildacerfont tablet for a total of 12 weeks of treatment. Receives: Drug: Tildacerfont |
| Placebo Control Arm | Placebo comparator | Subjects randomized in this arm received 12 weeks of oral matched-placebo tablet Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in DHEAS
To evaluate the effect of tildacerfont in changing (reducing) DHEAS in subjects with PCOS and elevated adrenal androgens
Measured: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Secondary outcomes (3)
Reduction in DHEAS
Number of subjects with >/=30% change from baseline in DHEAS
Measured: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Normalization of DHEAS
Number of subjects with DHEAS =/< upper limit of normal for DHEAS
Measured: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Number of Subjects With TEAE as Assessed by CTCAE Version 5
Evaluation of safety of tildacerfont with PCOS as measured by number of subjects with adverse events following dosing by CTCAE Version 5
Measured: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 May 2022
- Study start15 May 2022
- Primary completion3 Aug 2023
- Study completion31 Aug 2023
Why it stopped: The study was discontinued early by the sponsor
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 Polycystic Ovary SyndromeStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Spruce Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Will Charlton, MD, Study director, Spruce Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05370521
- Sponsor's protocol ID
- SPR001-210
- Status last verified
- Sep 2025
- First submitted
- 6 May 2022
- Last updated
- 9 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Placebo-Controlled, Dose Escalation Study to Evaluate the Safety and Efficacy of Tildacerfont in Adult Subjects With Polycystic Ovary Syndrome (PCOS) and Elevated Adrenal Androgens
- ClinicalTrials.gov ID
- NCT05370521
- Acronym / sponsor ID
- SPR001-210
- Conditions
- Polycystic Ovary Syndrome
- Keywords
- PCOS, Elevated Adrenal Androgen, Adrenal Disorder, Polycystic Ovary Syndrome, Womens health
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was discontinued early by the sponsor
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Spruce BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 15 May 2022 (actual)
- Primary completion
- 3 Aug 2023 (actual)
- Study completion
- 31 Aug 2023 (actual)
- First posted
- 11 May 2022 (actual)
- Results
- Posted 9 Oct 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 women with PCOS and elevated adrenal androgens
Detailed description
This was a phase 2 study to evaluate the efficacy, safety and tolerability of 3 doses of tildacerfont in approximately 39 women with PCOS and elevated adrenal androgens assessed by elevated DHEAS levels. Subjects were randomized 2:1 (tildacerfont:placebo) at baseline. The study consisted of 3 consecutive 4-week treatment periods at each dose level. Duration of participation was approximately 25 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment with Tildacerfont | Experimental | Drug: TildacerfontSubjects randomized in this arm received 4 weeks of 50 mg of oral tildacerfont tablet, followed by 4 weeks of 100 mg oral tildacerfont tablet, and then 4 weeks of 200 mg oral tildacerfont tablet for a total of 12 weeks of treatment. |
| Placebo Control Arm | Placebo comparator | Drug: PlaceboSubjects randomized in this arm received 12 weeks of oral matched-placebo tablet |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- Female subjects aged 18 to 40 years old at Screening visit
- Diagnosis of PCOS (either historical or during Screening) according to NIH (1990) criteria
- DHEAS level > age-matched upper limit of normal (ULN) at Screening visit
- Agree to follow industry standard contraception guidelines within protocol
Key exclusion criteria (11 listed)
- Evidence of:
- Total testosterone levels >140 ng/dL, DHEAS >650 mcg/dL, virilization or other signs or symptoms concerning for ovarian hyperthecosis or androgen-secreting tumors
- Medical conditions that require glucocorticoid treatment within 30 days of screening and throughout the duration of the study
- Clinically significant unstable medical conditions, illness, or chronic diseases
- Prior hysterectomy or bilateral oophorectomy
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. | Drug | Treatment with Tildacerfont |
| PlaceboNon-active dosage form taken once daily. | Drug | Placebo Control Arm |
Primary outcome
Change in DHEAS
Time frame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
To evaluate the effect of tildacerfont in changing (reducing) DHEAS in subjects with PCOS and elevated adrenal androgens
3 secondary outcomes
Reduction in DHEAS
Time frame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Normalization of DHEAS
Time frame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Number of Subjects With TEAE as Assessed by CTCAE Version 5
Time frame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Will Charlton, MD study director
Spruce Biosciences
21 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 Polycystic Ovary Syndrome, run by Spruce Biosciences at 21 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 11 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 9 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 21 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. Some participants in this study may receive a placebo. 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 9 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?
Change in DHEAS (time frame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Oct 2025; AzTrials last refreshed its copy on 8 Nov 2026.