Phase II randomized study on efficacy of nintedanib for treatment of epistaxis in hereditary haemorrhagic telangiectasia (HHT) patients - EPISTOP
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 2
- 主要终点
- Proportion of patients with at least 30% reduction of monthly epistaxis duration after 16 weeks of study treatment (at V6, week 24) compared to baseline (V1, week 8) assessed in nintedanib arm and in placebo arm.
研究概览
简要总结
Whether the proportion of HHT patients with a reduction of at least 30% of epistaxis duration after 16 weeks of study treatment compared to baseline, is significantly higher in patients treated with nintedanib than in patients treated with placebo.
研究设计
- 分配方式
- Randomized
- 主要目的
- Nintedanib / Placebo
- 盲法
- Double (Subject, Investigator)
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
- •Signed informed consent
- •Definite HHT disease (defined as the presence of a pathogenic mutation in one of the HHT genes, or the presence of 3 out of 4 Curaçao clinical criteria2 )
- •Aged ≥18 years at the time of informed consent
- •Moderate to serious epistaxis (Epistaxis Severity Score ESS≥2.5)
- •Absence of cerebral arteriovenous malformation demonstrated by brain imaging
排除标准
- •Women who are pregnant or breastfeeding because of the potential dangerous effect of the treatment on the fetus or infant
- •Any other serious underlying medical condition that could interfere with study treatment and potential adverse events
- •Any mental or other impairment that may compromise compliance with the study requirements.
- •Pregnant woman or woman of child bearing potential not using two effective methods of birth control (one barrier and one highly effective non-barrier) for at least 1 month prior to trial and/or committing to using it until 3 months after the end of treatment.
- •Acute infection
- •AST or ALT or ALKP or GGT or total bilirubin >1.5x (or >2.5x in patients known for Gilbert’s syndrome) the upper limit of normal
- •Renal clearance by Cockcroft-Gault formula <30 ml/min
- •Untreated pulmonary arteriovenous malformation
- •Hemoptysis or hematuria within the last 12 months
- •Ulcus or active gastric bleeding within the last 12 months
- •Anticoagulant or antiplatelets treatment
- •Coronary heart disease
- •Participation in another interventional clinical trial which may interfere with the proposal trial (judgment of the investigator)
- •Thrombotic event within the last 12 months
- •Long QT syndrome (on ECG performed at screening)
- •Known allergy to Nintenanib, soya, peanuts
- •Bevacizumab, pazopanib or other anti-angiogenic treatments within the last 12 months
- •Concomitant treatment with ketoconazole, erythromycin, rifampicin, carbamazepine, phenytoin, St John’s Wort
- •Surgery within the last 3 months or planned within the next 9 months
- •Recent unhealed wound
结局指标
主要结局
Proportion of patients with at least 30% reduction of monthly epistaxis duration after 16 weeks of study treatment (at V6, week 24) compared to baseline (V1, week 8) assessed in nintedanib arm and in placebo arm.
Proportion of patients with at least 30% reduction of monthly epistaxis duration after 16 weeks of study treatment (at V6, week 24) compared to baseline (V1, week 8) assessed in nintedanib arm and in placebo arm.
The monthly epistaxis duration after 16 weeks of study treatment is defined as the average of epistaxis duration during the last 12 weeks of study treatment (minutes/4-weeks period averaged for weeks 12 to 24, i.e. V3 to V6)
The monthly epistaxis duration after 16 weeks of study treatment is defined as the average of epistaxis duration during the last 12 weeks of study treatment (minutes/4-weeks period averaged for weeks 12 to 24, i.e. V3 to V6)
The monthly epistaxis duration at baseline is defined as the average of epistaxis duration during the observation period (minutes/4-weeks period averaged for weeks 0 to 8, i.e. V0 to V1).
The monthly epistaxis duration at baseline is defined as the average of epistaxis duration during the observation period (minutes/4-weeks period averaged for weeks 0 to 8, i.e. V0 to V1).
次要结局
未报告次要终点
研究者
Dr DUPUIS GIROD
Scientific
Hospices Civils De Lyon