2025-524490-17-00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
- 12
- 试验地点
- 19
- 主要终点
- Total number of epistaxis events through Week 28 as assessed by the HHT eDiary
研究概览
简要总结
To assess the efficacy of engasertib 40 mg QD in reducing the frequency of epistaxis compared to placebo QD during 28 weeks of double-blind treatment in subjects with moderate to severe HHT
研究设计
- 分配方式
- Randomized
- 主要目的
- Treatment
- 盲法
- Double (Investigator, Carer, Subject, Monitor)
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Subjects are ≥18 years of age at the Screening Visit.
- •Subjects 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: o Multiple telangiectases at characteristic sites: lips, oral cavity, fingers, or nose; o Visceral lesions: gastrointestinal telangiectasia and/or pulmonary, hepatic, cerebral, or spinal arteriovenous malformations; or o A first degree relative with HHT according to these criteria.
- •Subjects must have an ESS >4 at screening, and, in the judgement of the Investigator, subjects 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
- •Subjects have anemia (hemoglobin levels <13 g/dL in men and <12 g/dL in women) 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.
- •Subjects with prediabetes should be clinically stable, and those with known diabetes must have their disease adequately controlled, as defined by a glycosylated hemoglobin (HbA1c) ≤8.0%.
排除标准
- •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.
- •Uncontrolled hypertension as per Investigator’s judgement. If blood pressure is uncontrolled at the Screening Visit, initiation or adjustment of antihypertensive medication(s) is permitted during the Qualifying Period. The dose should be stable prior to randomization.
研究组 & 干预措施
Engasertib Placebo
Placebo
干预措施: Engasertib Placebo (Drug)
Engasertib, Engasertib
Test
干预措施: Engasertib (Drug)
结局指标
主要结局
Total number of epistaxis events through Week 28 as assessed by the HHT eDiary
Total number of epistaxis events through Week 28 as assessed by the HHT eDiary
次要结局
- Absolute change from baseline in total duration of epistaxis at Week 28 as assessed by the HHTeDiary
- Absolute change from baseline in ESS at Week 28, based on symptoms as they have occurred over the last 4 weeks as assessed by the ESS questionnaire
- Absolute change from baseline in the NOSE HHT score at Week 28, based on symptoms as they have occurred over the past 2 weeks as assessed by the NOSE HHT questionnaire
- Absolute change in red blood cell (RBC) unit equivalents (RUEs) received at Week 28
研究者
Damien Picard
Scientific
Vaderis Therapeutics AG
研究点 (19)
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