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

NCT ID: NCT05370521

Last Updated: 2025-10-09

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

27 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-05-15

Study Completion Date

2023-08-31

Brief Summary

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An investigation of the safety and efficacy of tildacerfont in women with PCOS and elevated adrenal androgens

Detailed Description

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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

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Polycystic Ovary Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators
Double-blind randomization performed by IRT

Study Groups

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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.

Group Type EXPERIMENTAL

Tildacerfont

Intervention Type DRUG

Oral tablet formulation taken once daily.

Placebo Control Arm

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Non-active dosage form taken once daily.

Interventions

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Tildacerfont

Oral tablet formulation taken once daily.

Intervention Type DRUG

Placebo

Non-active dosage form taken once daily.

Intervention Type DRUG

Other Intervention Names

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SPR001

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

40 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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Spruce Biosciences

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Will Charlton, MD

Role: STUDY_DIRECTOR

Spruce Biosciences

Locations

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Spruce Study Site

Sacramento, California, United States

Site Status

Spruce Study Site

San Francisco, California, United States

Site Status

Spruce Study Site

New Haven, Connecticut, United States

Site Status

Spruce Study Site

Clearwater, Florida, United States

Site Status

Spruce Study Site

Lake Worth, Florida, United States

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Spruce Study Site

Miami, Florida, United States

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Spruce Study Site

Winter Park, Florida, United States

Site Status

Spruce Study Site

Idaho Falls, Idaho, United States

Site Status

Spruce Study Site

Wichita, Kansas, United States

Site Status

Spruce Study Site

Boston, Massachusetts, United States

Site Status

Spruce Study Site

Fall River, Massachusetts, United States

Site Status

Spruce Study Site

Southfield, Michigan, United States

Site Status

Spruce Study Site

Williamsville, New York, United States

Site Status

Spruce Study Site

Raleigh, North Carolina, United States

Site Status

Spruce Study Site

Cincinnati, Ohio, United States

Site Status

Spruce Study Site

Cleveland, Ohio, United States

Site Status

Spruce Study Site

Philadelphia, Pennsylvania, United States

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Spruce Study Site

Bedford, Texas, United States

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Spruce Study Site

Houston, Texas, United States

Site Status

Spruce Study Site

Houston, Texas, United States

Site Status

Spruce Study Site

Morgantown, West Virginia, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

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SPR001-210

Identifier Type: -

Identifier Source: org_study_id

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