A Randomized Double Blind Placebo Controlled Trial of Intranasal Submucosal Bevacizumab in Hereditary Hemorrhagic Telangiectasia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- relative change in average daily Epistaxis VAS scores compared to baseline
研究概览
简要总结
In a case series intranasal submucosal bevacizumab has been shown to reduce epistaxis in patients suffering from Hereditary Haemorrhagic Telangiectasia together with KTP Laser therapy. The aim of this study is to evaluate the effectiveness of submucosal intranasal bevacizumab compared to placebo in a randomized double blind trial setting.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed and staged HHT (Shovlin et al 2000)
- •Age 18-80
- •Minimum of 2 episodes of epistaxis/ week
- •Ability and willingness to complete diary and comply with study requirements.
排除标准
- •Uncontrolled hypertension (systolic blood pressure > 150mmHg, diastolic blood pressure > 90mmHg)
- •History of a thromboembolic event, including myocardial infarction or cerebral vascular accident
- •Malignancy of the upper respiratory tract within the last year
- •Recent (<3 months) or planned surgery
- •Proteinuria
- •Nasal intervention (Laser or Cautery) in pretreatment phase
- •Allergy to local anesthetic
研究组 & 干预措施
bevacizumab
submucosal intranasal bevacizumab on day 0
干预措施: Bevacizumab (Drug)
placebo
0.9% NaCl intranasal submucosal on day 0
干预措施: NaCl (Drug)
结局指标
主要结局
relative change in average daily Epistaxis VAS scores compared to baseline
时间窗: day 10 - 84 posttreatment
Daily epistaxis VAS scores are recorded in a diary. The baseline score is the average daily epistaxis VAS score 4 weeks before treatment (day -28 to 0). This score is compared to the average daily VAS score day 10-84 posttreatment. The relative change of this average score compared to baseline is the primary outcome.
次要结局
- Epistaxis Severity Score HHT-ESS compared to baseline(3 months post treatment)
- Epistaxis frequency, duration and severity compared to baseline(day 10 - 84 posttreatment)
- Number of emergency department visits due to epistaxis compared to baseline(day 10 - 84 posttreatment)
- lab results (ferritin values, Hb, Hct) compared to baseline(day 84 posttreatment)
- Number of transfusions needed compared to baseline(day 10-84 posttreatment)
- Average daily epistaxis VAS scores compared to baseline among age groups and among groups with different epistaxis severity(day 10-84 posttreatment)
研究者
Martin Burian
Prof. Dr. Martin Burian
Medical University of Vienna