ClinicalTrials.gg
TerminatedNCT05370521Updated Oct 9, 2025Results posted

A Study of Safety and Efficacy of Tildacerfont in Females With Polycystic Ovary Syndrome and Elevated Adrenal Androgens

A Phase 2 interventional study of Tildacerfont and Placebo in Polycystic Ovary Syndrome, sponsored by Spruce Biosciences. Terminated at 21 sites in United States. Open to female participants aged 18 Years to 40 Years. Per ClinicalTrials.gov, last updated 2025-10-09.

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

Why this study was terminated
The study was discontinued early by the sponsor
Phase
Phase 2
Study type
Interventional
Enrollment
27
Allocation
Randomized
Ages
18 Years to 40 Years
Sex
Female
01

Study summary

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.

02

Conditions studied

  • Polycystic Ovary Syndrome

Keywords

  • PCOS
  • Elevated Adrenal Androgen
  • Adrenal Disorder
  • Polycystic Ovary Syndrome
  • Womens health
03

In context

Polycystic Ovary Syndrome

956 studies on the registry are indexed under Polycystic Ovary Syndrome; 176 are open to participants now.

This study's enrollment of 27 is below the median of 70 across 697 interventional studies indexed under Polycystic Ovary Syndrome.

Browse Polycystic Ovary Syndrome 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.

04

Who can participate

Ages eligible
18 Years to 40 Years
Sexes eligible
Female
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

  • 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 (ULN) at Screening visit
  • Agree to follow industry standard guidelines within protocol

Exclusion criteria

Exclusion Criteria:

  • Evidence of:

    1. History of congenital adrenal hyperplasia (CAH), Cushing's syndrome, pituitary or adrenal disease
    2. hyperprolactinemia
    3. Thyroid stimulating hormone (TSH) \<0.1 mU/mL or >4.5mU/mL at Screening
    4. Cortisol levels concerning for adrenal insufficiency
    5. Other findings suggestive of secondary cause for anovulation and/or hyperandrogenemia
  • 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 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
Terms in these criteria, in plain language(4)

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.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

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.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
27 participants (actual)

Study arms

  • Experimental
    Treatment with Tildacerfont

    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.

    Drug: Tildacerfont

  • Placebo comparator
    Placebo Control Arm

    Subjects randomized in this arm received 12 weeks of oral matched-placebo tablet

    Drug: Placebo

Interventions

  • Oral tablet formulation taken once daily.

    Also known as: SPR001

  • DrugPlacebo

    Non-active dosage form taken once daily.

06

What researchers measure

Primary outcomes

  1. Change in DHEAS

    To evaluate the effect of tildacerfont in changing (reducing) DHEAS in subjects with PCOS and elevated adrenal androgens

    Time frame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)

Secondary outcomes

  1. Reduction in DHEAS

    Number of subjects with \>/=30% change from baseline in DHEAS

    Time frame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)

  2. Normalization of DHEAS

    Number of subjects with DHEAS =/\< upper limit of normal for DHEAS

    Time frame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)

  3. 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

    Time frame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)

07

Results

Posted Oct 9, 2025
Limitations and caveats
The study was terminated by the sponsor; therefore, there were a small number of subjects for analysis.

Participant flow

Of the 82 subjects that were screened, a total of 27 subjects enrolled in the study (10 subjects in the placebo arm and 17 subjects in the tildacerfont arm).

Participant flow — Overall Study
MilestoneTreatment With TildacerfontPlacebo Control Arm
Started1710
Completed169
Not completed11

Outcome measures

SecondaryReduction in DHEAS

Number of subjects with \>/=30% change from baseline in DHEAS

Time frame:
12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Reported as:
Count of participants · Participants
Reduction in DHEAS
ParticipantsTreatment With TildacerfontPlacebo Control Arm
Reduction in DHEAS11
SecondaryNormalization of DHEAS

Number of subjects with DHEAS =/\< upper limit of normal for DHEAS

Time frame:
12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Reported as:
Count of participants · Participants
Normalization of DHEAS
ParticipantsTreatment With TildacerfontPlacebo Control Arm
Normalization of DHEAS86
SecondaryNumber 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

Time frame:
12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Reported as:
Count of participants · Participants
Number of Subjects With TEAE as Assessed by CTCAE Version 5
ParticipantsTreatment With TildacerfontPlacebo Control Arm
Number of Subjects With TEAE as Assessed by CTCAE Version 5139
PrimaryChange in DHEAS

To evaluate the effect of tildacerfont in changing (reducing) DHEAS in subjects with PCOS and elevated adrenal androgens

Time frame:
12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Reported as:
Mean · ug/dL
Change in DHEAS
ug/dLTreatment With TildacerfontPlacebo Control Arm
Change in DHEAS-22.93 ± 67.600-7.56 ± 16.00

Adverse events

Collected over 12 weeks. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Treatment With Tildacerfont0/17 (0%)1/17 (5.9%)13/17 (76.5%)
Placebo Control Arm0/10 (0%)0/10 (0%)9/10 (90%)
Most frequent serious events
Most frequent serious events
EventTreatment With TildacerfontPlacebo Control Arm
Obstructive pancreatitisGastrointestinal disorders1/170/10
Most frequent other events
Showing 10 of 34
Most frequent other events
EventTreatment With TildacerfontPlacebo Control Arm
DiarrheaGastrointestinal disorders8/171/10
FatigueGeneral disorders0/172/10
AcneSkin and subcutaneous tissue disorders0/172/10
Abdominal PainGastrointestinal disorders2/171/10
Abdominal pain lowerGastrointestinal disorders2/170/10
NasopharyngitisInfections and infestations2/171/10
Vertigo positionalEar and labyrinth disorders0/171/10
NauseaGastrointestinal disorders1/171/10
COVID-19Infections and infestations1/171/10
Vulvovaginal mycotic infectionInfections and infestations1/171/10

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Treatment With TildacerfontPlacebo Control ArmTotal
<=18 years000
Between 18 and 65 years171027
>=65 years000
Sex: Female, Male
Sex: Female, Male(Participants)Treatment With TildacerfontPlacebo Control ArmTotal
Female171027
Male000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Treatment With TildacerfontPlacebo Control ArmTotal
American Indian or Alaska Native000
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American213
White15924
More than one race000
Unknown or Not Reported000
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Treatment With TildacerfontPlacebo Control ArmTotal
Hispanic or Latino7613
Not Hispanic or Latino10414
Unknown or Not Reported000
Dehydroepiandrosterone sulfate (DHEAS)
Dehydroepiandrosterone sulfate (DHEAS)(ug/dL)Treatment With TildacerfontPlacebo Control ArmTotal
Mean351.3 ± 90.53387.8 ± 107.16364.8 ± 96.65
Adrenocorticotropic hormone (ACTH)
Adrenocorticotropic hormone (ACTH)(pg/dL)Treatment With TildacerfontPlacebo Control ArmTotal
Mean23.88 ± 11.92722.3 ± 12.01323.27 ± 11.744
17-hydroxyprogesterone (17-OHP)
17-hydroxyprogesterone (17-OHP)(ng/dL)Treatment With TildacerfontPlacebo Control ArmTotal
Mean83.2 ± 86.2862.1 ± 54.175.4 ± 75.51
Androstenedione (A4)
Androstenedione (A4)(ng/dL)Treatment With TildacerfontPlacebo Control ArmTotal
Mean185.2 ± 75.19130.0 ± 65.96166.8 ± 75.61

2 further baseline measures are reported on the registry.

08

Study locations

21 sites
  • Spruce Study Site
    Sacramento, California 95817, United States
  • Spruce Study Site
    San Francisco, California 94158, United States
  • Spruce Study Site
    New Haven, Connecticut 06519, United States
  • Spruce Study Site
    Clearwater, Florida 33759, United States
  • Spruce Study Site
    Lake Worth, Florida 33461, United States
  • Spruce Study Site
    Miami, Florida 33173, United States
  • Spruce Study Site
    Winter Park, Florida 32792, United States
  • Spruce Study Site
    Idaho Falls, Idaho 83404, United States
  • Spruce Study Site
    Wichita, Kansas 67226, United States
  • Spruce Study Site
    Boston, Massachusetts 02115, United States
  • Spruce Study Site
    Fall River, Massachusetts 02720, United States
  • Spruce Study Site
    Southfield, Michigan 48034, United States
  • Spruce Study Site
    Williamsville, New York 14221, United States
  • Spruce Study Site
    Raleigh, North Carolina 27612, United States
  • Spruce Study Site
    Cincinnati, Ohio 45219, United States
  • Spruce Study Site
    Cleveland, Ohio 44106, United States
  • Spruce Study Site
    Philadelphia, Pennsylvania 19104, United States
  • Spruce Study Site
    Bedford, Texas 76022, United States
  • Spruce Study Site
    Houston, Texas 77024, United States
  • Spruce Study Site
    Houston, Texas 77030, United States
  • Spruce Study Site
    Morgantown, West Virginia 26506, United States
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 9, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT05370521
Lead sponsor
Spruce Biosciences
Responsible party
Sponsor
First posted
May 11, 2022
Start date
May 15, 2022
Primary completion
Aug 3, 2023
Completion
Aug 31, 2023
Results posted
Oct 9, 2025
Last update
Oct 9, 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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