An Uncontrolled, Pilot-study Assessing the Efficacy of Octreotide Long-acting Release to Decrease Transfusion Requirements and Endoscopy Frequency in Patients With Rendu-Osler-Weber and Gastrointestinal Bleeding
试验速览
- 阶段
- 2 期
- 入组人数
- 15
- 试验地点
- 2
- 主要终点
- The percentage of patients who are full responder, partial responder and non-responder at the end of the treatment period
研究概览
简要总结
The purpose of this study is to determine whether long-acting octreotide is safe and effective in the treatment of patients with Rendu-Osler-Weber (e.g. HHT).
The study hypothesis is that octreotide is safe and will reduce transfusion requirements and endoscopy frequency in ROW patients with refractory anaemia due to bleeding gastrointestinal telangiectasias.
详细描述
Rationale: Rendu-Osler-Weber (ROW) is an autosomal dominant hereditary disease which affects 1 / 5-8000 individuals. It is characterized by arteriovenous malformations (AVMs) and telangiectasias in multiple organs, including the gastrointestinal tract. Patients can be transfusion dependent due to severe gastrointestinal bleeding from those telangiectasias. Endoscopy is not as effective due to the recurrent character of the telangiectasias. Based on literature in patients with non-ROW AVMs and telangiectasias, octreotide might be beneficial for these patients to decrease their transfusion needs.
Objective: To assess the efficacy of octreotide in decreasing the need for transfusions and endoscopic intervention in patients ROW with refractory anaemia due to gastrointestinal bleeding telangiectasias.
Study design: Multicenter, open-label uncontrolled pilot study.
Study population: Patients with ROW and symptomatic gastrointestinal bleeding telangiectasias, who are transfusion and/or endoscopy dependent:
- Transfusion dependent: at least 2 blood and/or iron infusions in the 6 months before inclusion.
- Endoscopy dependent: at least one endoscopic intervention with argon plasma coagulation (APC) after the initial/first endoscopic treatment after diagnosis in the half year before inclusion or unsuitable for endoscopy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Patients with Rendu-Osler-Weber
- •Symptomatic gastrointestinal bleeds out of telangiectasias
- •Transfusion and / or endoscopy dependent:
- •Transfusion: at least 2 blood and/or iron infusions in the 6 months before inclusion.
- •Endoscopy: at least one endoscopy with APC after the initial endoscopic treatment after diagnosis in the half year before inclusion or unsuitable for endoscopic therapy.
排除标准
- •liver cirrhosis Child-Pugh C or acute liver failure
- •previous unsuccessful treatment with somatostatin analogues (SST) for the same indication (refractory anaemia due to telangiectasias) or current effective treatment with a somatostatin analogue
- •severe diseases with life expectancy < 1 year
- •patients with left ventricular assist devices (LVAD's)
- •Symptomatic cholecystolithiasis (without cholecystectomy)
- •pregnancy or nursing women or women who have a pregnancy wish in the study period or who use anticonception inadequate
- •current chemotherapy
- •patients with a known hypersensitivity to SST analogues or any component of the octreotide LAR formulations
- •no understanding of Dutch or English
研究组 & 干预措施
Active comparator: Octreotide LAR
Sandostatin LAR Sandostatin LAR 20 mg will be administered once every 4 weeks as a intramuscular injection
干预措施: Octreotide LAR (Drug)
结局指标
主要结局
The percentage of patients who are full responder, partial responder and non-responder at the end of the treatment period
时间窗: Comparing the 6 months before inclusion and the study period (26 weeks)
Full responder: no endoscopy and no blood/iron transfusions during treatment period. * Partial responder: a decrease in number of blood/iron transfusions and/or endoscopy during the treatment period compared with the 6 months prior to inclusion. * Non-responder: no decrease in number of blood/iron transfusions and endoscopy during the treatment period compared with the 6 months prior to inclusion.
次要结局
- The percentual decrease in blood and iron requirements(Comparing the 6 months prior to inclusion and the treatment period of 6 months.)
- The percentual decrease in the number of endoscopic interventions(Comparing the 6 months prior to inclusion and the treatment period of 6 months.)
- The mean/median decrease on the epistaxis severity score (ESS)(Comparing the 6 months prior to inclusion and the treatment period of 6 months.)
- Change in quality of life using the Short Form (SF)-36 questionnaire(Comparing baseline and end of treatment visit)
- The number, type and severity of adverse events(Study period)