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

Phase 2

Completed

• Conditions: Polycystic Ovary Syndrome
• Interventions: Drug: Placebo; Drug: Tildacerfont

• Registration Number: NCT05370521
• Lead Sponsor: Spruce Biosciences

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.

Study Timeline

• Study First Submitted: 2022-05-06
• Study First Submitted QC: 2022-05-06
• Study First Posted: 2022-05-11
• Study Start: 2022-05-15
• Primary Completion: 2023-08-03
• Study Completion: 2023-08-31
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-25
• Last Update Posted: 2023-09-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Female
• Target Recruitment: 27

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo Control Arm	Placebo	Subjects randomized in this arm will receive 12 weeks of oral matched-placebo tablet
Treatment with Tildacerfont	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.

Primary Outcome Measures

Name	Time	Method
DHEAS level	12 Weeks (assessed at Baseline, Weeks 4, 8, and 12)	Change from baseline in DHEAS level

Secondary Outcome Measures

Name	Time	Method
Reduction in DHEAS	12 weeks (assessed at Baseline, Weeks 4, 8 and 12)	Proportion of subjects with \>/=30% change from baseline in DHEAS
Normalization of DHEAS	12 weeks (assessed at Baseline, Weeks 4, 8 and 12)	Proportion of subjects with DHEAS =/\< upper limit of normal for DHEAS
Number of subjects with TEAE as assessed by CTCAE Version 5	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

Trial Locations

Locations (1)

Spruce Study Site; 🇺🇸 Morgantown, West Virginia, United States