Clinical trial NCT05370521: A Study of Safety and Efficacy of Tildacerfont in Females With Polycystic Ovary Syndrome and Elevated Adrenal Androgens
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Polycystic Ovary Syndrome
Keywords: PCOS
Elevated Adrenal Androgen
Adrenal Disorder
Polycystic Ovary Syndrome
Womens health
Lead sponsor: Spruce Biosciences
Sponsor class: INDUSTRY
First posted: 2022-05-11
Enrollment: 27
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 40
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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

Detailed description: This is 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 will be randomized 2:1 (tildacerfont:placebo) at baseline. The study will consist of 3 consecutive 4-week treatment periods at each dose level. Duration of participation is approximately 25 weeks.

Eligibility criteria: 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

Primary outcomes:
measure: DHEAS level
timeFrame: 12 Weeks (assessed at Baseline, Weeks 4, 8, and 12)
Secondary outcomes:
measure: Reduction in DHEAS
timeFrame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
measure: Normalization of DHEAS
timeFrame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
measure: Number of subjects with TEAE as assessed by CTCAE Version 5
timeFrame: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: Spruce Study Site
state: California
city: San Francisco
country: United States
facility: Spruce Study Site
state: California
city: New Haven
country: United States
facility: Spruce Study Site
state: Connecticut
city: Clearwater
country: United States
facility: Spruce Study Site
state: Florida
city: Lake Worth
country: United States
facility: Spruce Study Site
state: Florida
city: Miami
country: United States
facility: Spruce Study Site
state: Florida
city: Winter Park
country: United States
facility: Spruce Study Site
state: Florida
city: Idaho Falls
country: United States
facility: Spruce Study Site
state: Idaho
city: Wichita
country: United States
facility: Spruce Study Site
state: Kansas
city: Boston
country: United States
facility: Spruce Study Site
state: Massachusetts
city: Fall River
country: United States
facility: Spruce Study Site
state: Massachusetts
city: Southfield
country: United States
facility: Spruce Study Site
state: Michigan
city: Williamsville
country: United States
facility: Spruce Study Site
state: New York
city: Raleigh
country: United States
facility: Spruce Study Site
state: North Carolina
city: Cincinnati
country: United States
facility: Spruce Study Site
state: Ohio
city: Cleveland
country: United States
facility: Spruce Study Site
state: Ohio
city: Philadelphia
country: United States
facility: Spruce Study Site
state: Pennsylvania
city: Bedford
country: United States
facility: Spruce Study Site
state: Texas
city: Houston
country: United States
facility: Spruce Study Site
state: Texas
city: Houston
country: United States
facility: Spruce Study Site
state: Texas
city: Morgantown
country: United States
facility: Spruce Study Site
state: West Virginia
Study officials:
affiliation: Spruce Biosciences
name: Will Charlton, MD
role: STUDY_DIRECTOR
References: