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

Spruce Biosciences

Status and phase: Terminated; Phase 2

Conditions: Polycystic Ovary Syndrome

Treatments: Drug: Placebo; Drug: Tildacerfont

Study type: Interventional

Funder types: Industry

Identifiers

NCT05370521

SPR001-210

Details and patient eligibility

About

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

Full 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.

Enrollment: 27 patients

Sex: Female

Ages: 18 to 40 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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27 participants in 2 patient groups, including a placebo group

Treatment with Tildacerfont

Experimental group

Description:

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.

Treatment:

Drug: Tildacerfont

Placebo Control Arm

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo