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
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 27
- 试验地点
- 1
- 主要终点
- Change in DHEAS
研究概览
简要总结
An investigation of the safety and efficacy of tildacerfont in women with PCOS and elevated adrenal androgens
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
盲法说明
Double-blind randomization performed by IRT
入排标准
- 年龄范围
- 18 Years 至 40 Years（Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •Evidence of:
- •History of congenital adrenal hyperplasia (CAH), Cushing's syndrome, pituitary or adrenal disease
- •Clinically significant hyperprolactinemia
- •Thyroid stimulating hormone (TSH) <0.1 mU/mL or >4.5mU/mL at Screening
- •Cortisol levels concerning for adrenal insufficiency
- •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
研究组 & 干预措施
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.
干预措施: Tildacerfont (Drug)
Placebo Control Arm
Subjects randomized in this arm received 12 weeks of oral matched-placebo tablet
干预措施: Placebo (Drug)
结局指标
主要结局
Change in DHEAS
时间窗: 12 weeks (assessed at Baseline, Weeks 4, 8 and 12)
To evaluate the effect of tildacerfont in changing (reducing) DHEAS in subjects with PCOS and elevated adrenal androgens
次要结局
- Reduction in DHEAS(12 weeks (assessed at Baseline, Weeks 4, 8 and 12))
- Normalization of DHEAS(12 weeks (assessed at Baseline, Weeks 4, 8 and 12))
- Number of Subjects With TEAE as Assessed by CTCAE Version 5(12 weeks (assessed at Baseline, Weeks 4, 8 and 12))