A Phase 2a/2b Randomized Double-Blind Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in Adult Women With Intrahepatic Cholestasis of Pregnancy and Elevated Serum Bile Acid Concentrations (OHANA).
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 4
- 试验地点
- 20
- 主要终点
- Assess the Safety and Tolerability of Volixibat in Participants With ICP
研究概览
简要总结
Part 1 is an open-label randomized study of volixibat in patients with Intrahepatic Cholestasis of Pregnancy (ICP) and elevated serum bile acid concentrations (sBA) to evaluate safety and tolerability of two doses of volixibat. Part 2 is a double-blind, placebo controlled, study designed to evaluate the safety and efficacy of a selected volixibat dose.
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
- Double (Participant, Investigator)
盲法说明
Masking in Part 2 Only
入排标准
- 年龄范围
- 18 Years 至 45 Years（Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Female aged ≥18 and ≤45 years with a viable pregnancy.
- •Provide signed informed consent as described in the protocol and willing to comply with all study visits and requirements.
- •Diagnosis of ICP.
- •(Part 2 only) Qualified level of pruritus associated with ICP, during screening.
排除标准
- •At the time of either the screening or baseline visit, decision has already been made to deliver within the next 7 days, for any indication.
- •Known non-reassuring fetal status based upon antepartum testing (e.g., NST/CTG or BPP) at or within 7 days before the baseline visit.
- •Known fetal anomaly likely to result in intrauterine fetal demise or neonatal death within the first 30 days of life.
- •Participating in another ongoing interventional clinical study at screening or planning to participate in another contemporaneous interventional clinical study while participating in this study.
研究组 & 干预措施
Part 1 Arm 1 - Volixibat 20mg
Participants randomized to this arm will receive volixibat 20mg twice daily.
干预措施: Volixibat (Drug)
Part 1 Arm 2 - Volixibat 80mg
Participants randomized to this arm will receive volixibat 80mg twice daily.
干预措施: Volixibat (Drug)
Part 2 Arm 1 - Volixibat Selected Dose mg
Participants randomized to this arm will receive volixibat selected dose (mg) twice daily.
干预措施: Volixibat (Drug)
Part 2 Arm 2 - Placebo (Placebo Comparator)
Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
干预措施: Placebo (Drug)
结局指标
主要结局
Assess the Safety and Tolerability of Volixibat in Participants With ICP
时间窗: Through to end of treatment, up to 21 weeks
To assess the safety and tolerability of volixibat in participants with ICP on the basis of the following endpoints: Proportion of participants experiencing one or more of the following: Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs), events of clinical interest (ECIs), and adverse events (AEs) that lead to discontinuation of study drugs. Clinically significant laboratory abnormalities
次要结局
- Mean Change in the Weekly Average Worst Daily Itch Score as Measured by the Adult Itch Reported Outcome (ItchRO)(Through to end of treatment, up to 21 weeks)
- Proportion of Participants Experiencing One or More of Adverse Perinatal Outcomes(At least one month after delivery.)