NCT07743671招募中3 期
A Phase 3, Randomized, Double-blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Engasertib in Subjects With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 240
- 试验地点
- 4
- 主要终点
- Total Number of Epistaxis Events Through Week 28
研究概览
简要总结
The primary objective of this trial is to assess the efficacy of engasertib 40 mg once daily (QD) in reducing the frequency of epistaxis compared to placebo QD during 28 weeks of double-blind treatment in participants with moderate to severe HHT.
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)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants are ≥18 years of age at the Screening Visit.
- •Participants have a definite diagnosis of HHT by the Curaçao criteria, defined as spontaneous and recurrent epistaxis and having at least 2 of the following criteria:
- •Multiple telangiectases at characteristic sites: lips, oral cavity, fingers, or nose;
- •Visceral lesions: gastrointestinal telangiectasia and/or pulmonary, hepatic, cerebral, or spinal arteriovenous malformations (AVMs); or
- •A first degree relative with HHT according to these criteria.
- •Participants must have an ESS >4 at screening, and, in the judgement of the Investigator, participants are expected to have regular epistaxis that typically lasts for several minutes. This criterion is assessed at screening only and does not require reconfirmation prior to randomization on Day
- •Participants have anemia OR in the prior 6 months have received a parenteral infusion of at least 250 mg of iron OR in the prior 6 months have received a red cell or whole blood transfusion.
排除标准
- •History or current diagnosis of clinically significant electrocardiogram (ECG) abnormalities.
- •History of significant or uncontrolled skin disorders per Investigator's judgement.
- •Local ablative (eg, cauterization) or surgical procedures on nasal telangiectases <6 weeks before the Screening Visit.
- •Use of drugs with anti-angiogenic properties, including, but not limited to, bevacizumab, pazopanib, thalidomide, lenalidomide, pomalidomide, tacrolimus, sirolimus, or selective estrogen response modulators (tamoxifen, raloxifene, or bazedoxifene) < 6 weeks before the Screening Visit.
- •Use of oral tranexamic or epsilon-aminocaproic acid unless they are on a stable dose for at least 4 weeks before the Screening Visit, which will need to be continued during the entire duration of the double-blind Treatment Period.
研究组 & 干预措施
Placebo
Placebo Comparator
Participants will receive matching placebo as an oral capsule QD for 28 weeks.
干预措施: Placebo (Drug)
Engasertib 40 mg
Experimental
Participants will receive engasertib as an oral capsule QD for 28 weeks.
干预措施: Engasertib (Drug)
结局指标
主要结局
Total Number of Epistaxis Events Through Week 28
时间窗: Day 1 through Week 28
次要结局
- Absolute Change from Baseline in the Nasal Outcome Score for Epistaxis in HHT (NOSE HHT) Score at Week 28(Baseline and Week 28)
- Absolute Change in Red Blood Cell (RBC) Unit Equivalents (RUEs) Received at Week 28(Baseline and Week 28)
- Patient Global Impression of Change (PGIC) Nosebleeds Sub-score at Week 28(Week 28)
- HHT-specific Quality of Life (HHT-QoL) Score at Week 28(Week 28)
- Absolute Change from Baseline in Total Duration of Epistaxis at Week 28(Baseline and Week 28)
- Absolute Change from Baseline in Epistaxis Severity Score (ESS) at Week 28(Baseline and Week 28)
- Absolute Change from Baseline in the Intensity-adjusted Duration of Epistaxis per Month at Week 28(Baseline and Week 28)
研究者
研究点 (4)
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