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

September 19, 2025 updated by: Spruce Biosciences

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

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

27

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 40 years (Adult)

Accepts Healthy Volunteers

No

Description

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 upper limit of normal (ULN) at Screening visit
  • Agree to follow industry standard contraception guidelines within protocol

Exclusion Criteria:

  • Evidence of:

    1. History of congenital adrenal hyperplasia (CAH), Cushing's syndrome, pituitary or adrenal disease
    2. Clinically significant 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 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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
Oral tablet formulation taken once daily.
Other Names:
  • SPR001
Placebo Comparator: Placebo Control Arm
Subjects randomized in this arm received 12 weeks of oral matched-placebo tablet
Drug: Placebo
Non-active dosage form taken once daily.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
12 weeks (assessed at Baseline, Weeks 4, 8 and 12)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Reduction in DHEAS
Time Frame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Number of subjects with >/=30% change from baseline in DHEAS
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 DHEAS =/< upper limit of normal for DHEAS
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)
Evaluation of safety of tildacerfont with PCOS as measured by number of subjects with adverse events following dosing by CTCAE Version 5
12 weeks (assessed at Baseline, Weeks 4, 8 and 12)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Spruce Biosciences

Investigators

  • Study Director: Will Charlton, MD, Spruce Biosciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 15, 2022

Primary Completion (Actual)

August 3, 2023

Study Completion (Actual)

August 31, 2023

Study Registration Dates

First Submitted

May 6, 2022

First Submitted That Met QC Criteria

May 6, 2022

First Posted (Actual)

May 11, 2022

Study Record Updates

Last Update Posted (Estimated)

October 9, 2025

Last Update Submitted That Met QC Criteria

September 19, 2025

Last Verified

September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SPR001-210

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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