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The study was discontinued early by the sponsor
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An investigation of the safety and efficacy of tildacerfont in women with PCOS and elevated adrenal androgens
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Placebo Control Arm | Placebo Comparator | Subjects randomized in this arm received 12 weeks of oral matched-placebo tablet |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tildacerfont | Drug | Oral tablet formulation taken once daily. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in DHEAS | 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) |
| Measure | Description | Time Frame |
|---|---|---|
| Reduction in DHEAS | Number of subjects with >/=30% change from baseline in DHEAS | 12 weeks (assessed at Baseline, Weeks 4, 8 and 12) |
| Normalization of DHEAS | Number of subjects with DHEAS =/< upper limit of normal for DHEAS |
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Inclusion Criteria:
Exclusion Criteria:
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
Females who are pregnant or nursing
Eligibility for females is based on gender assignment at birth.
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| Name | Affiliation | Role |
|---|---|---|
| Will Charlton, MD | Spruce Biosciences | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Spruce Study Site | Sacramento | California | 95817 | United States | ||
| Spruce Study Site |
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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).
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment With Tildacerfont | Subjects randomized in this arm received 4 weeks of 50mg 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. |
| FG001 | Placebo Control Arm |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 28, 2022 | Apr 24, 2025 |
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Double-blind randomization performed by IRT
| Placebo | Drug | Non-active dosage form taken once daily. |
|
| 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 | 12 weeks (assessed at Baseline, Weeks 4, 8 and 12) |
| 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 |
Subjects randomized in this arm received 12 weeks of oral matched-placebo tablet |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Treatment With Tildacerfont | Subjects randomized in this arm will receive 4 weeks of 50mg 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. |
| BG001 | Placebo Control Arm | Subjects randomized in this arm will receive 12 weeks of oral matched-placebo tablet |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Female subjects aged 18 to 40 years old due to the nature of the disease | Count of Participants | Participants |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Dehydroepiandrosterone sulfate (DHEAS) | Mean | Standard Deviation | ug/dL |
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| Adrenocorticotropic hormone (ACTH) | Mean | Standard Deviation | pg/dL |
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| 17-hydroxyprogesterone (17-OHP) | Mean | Standard Deviation | ng/dL |
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| Androstenedione (A4) | Mean | Standard Deviation | ng/dL |
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| Testosterone | Mean | Standard Deviation | ng/dL |
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| Cortisol | Mean | Standard Deviation | ug/dL |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Secondary | Reduction in DHEAS | Number of subjects with >/=30% change from baseline in DHEAS | Number of subjects with a >/=30% change from Baseline in DHEAS (μg/dL) at Week 12, Modified Intent-to-Treat Analysis Set. | Posted | Count of Participants | Participants | 12 weeks (assessed at Baseline, Weeks 4, 8 and 12) |
|
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| |||||||||||||||||||||||||||||
| Secondary | Normalization of DHEAS | Number of subjects with DHEAS =/< upper limit of normal for DHEAS | Number of subjects with DHEAS =/< upper limit of normal for DHEAS at Week 12, Modified Intent-to-treat Analysis Set. | Posted | Count of Participants | Participants | 12 weeks (assessed at Baseline, Weeks 4, 8 and 12) |
|
| ||||||||||||||||||||||||||||||
| Secondary | 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 | All subjects who received at least 1 dose of placebo or tildacerfont; Safety Analysis Set. | Posted | Count of Participants | Participants | 12 weeks (assessed at Baseline, Weeks 4, 8 and 12) |
|
| ||||||||||||||||||||||||||||||
| Primary | Change in DHEAS | To evaluate the effect of tildacerfont in changing (reducing) DHEAS in subjects with PCOS and elevated adrenal androgens | Analysis of Change from Baseline in DHEAS (μg/dL) at Week 12, Modified Intent-to-Treat Analysis Set. | Posted | Mean | Standard Deviation | ug/dL | 12 weeks (assessed at Baseline, Weeks 4, 8 and 12) |
|
|
12 weeks
Adverse events are presented as totals for subjects receiving tildacerfont and are not separated by dose level.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Treatment With Tildacerfont | Subjects randomized in this arm will receive 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. | 0 | 17 | 1 | 17 | 13 | 17 |
| EG001 | Placebo Control Arm | Subjects randomized in this arm will receive 12 weeks of oral matched-placebo tablet | 0 | 10 | 0 | 10 | 9 | 10 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Obstructive pancreatitis | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vertigo positional | Ear and labyrinth disorders | MedDRA 24.1 | Systematic Assessment |
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| Diarrhea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Abdominal Pain | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Abdominal pain lower | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Haematochezia | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 24.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Vulvovaginal mycotic infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Gastroenteritis viral | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Oral herpes | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Otitis media | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Pharyngitis streptococcal | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Viral rhinitis | Infections and infestations | MedDRA 24.1 | Systematic Assessment |
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| Blood potassium increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Total bile acids increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Weight increased | Investigations | MedDRA 24.1 | Systematic Assessment |
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| Fibrous histiocytoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Tension headache | Nervous system disorders | MedDRA 24.1 | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA 24.1 | Systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA 24.1 | Systematic Assessment |
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| Mood swings | Psychiatric disorders | MedDRA 24.1 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1 | Systematic Assessment |
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| Acne | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Pruitis | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.1 | Systematic Assessment |
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| Gastrointestinal Pain | Gastrointestinal disorders | MedDRA 24.1 | Systematic Assessment |
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The study was terminated by the sponsor; therefore, there were a small number of subjects for analysis.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | Spruce Biosciences | 415-655-4169 | clinicaltrials@sprucebio.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 17, 2023 | Apr 24, 2025 | SAP_001.pdf |
| ICF | No | No | Yes | Informed Consent Form | Oct 18, 2021 | May 19, 2025 | ICF_002.pdf |
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| ID | Term |
|---|---|
| D011085 | Polycystic Ovary Syndrome |
| D000307 | Adrenal Gland Diseases |
| ID | Term |
|---|---|
| D010048 | Ovarian Cysts |
| D003560 | Cysts |
| D009369 | Neoplasms |
| D010049 | Ovarian Diseases |
| D000291 | Adnexal Diseases |
| D005831 | Genital Diseases, Female |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D000091662 | Genital Diseases |
| D006058 | Gonadal Disorders |
| D004700 | Endocrine System Diseases |
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| >=65 years |
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| Male |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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