Ranibizumab for the Management of Recurrent Epistaxis in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 主要终点
- Epistaxis as measured by the HHT Epistaxis Severity Score (ESS), hematocrit, and hemoglobin and serum feritin levels.
研究概览
简要总结
This study is for patients with recurrent epistaxis (nosebleeds) as a result of Hereditary Hemorrhagic Telangiectasia (HHT). The aim is to determine if ranibizumab, topically applied will diminish epistaxis in patients with HHT as measured by the HHT Epistaxis Severity Score (ESS), hematocrit, and hemoglobin and serum ferritin levels.
详细描述
This is an open-label, Phase I study of intranasal administered ranibizumab in subjects with HHT. Plans are to recruit patients with HHT from the UCSD Nasal Dysfunction Clinic.
Those who come for evaluation and are deemed appropriate for topical ranibizumab without laser will be recruited for this study. Ten (10) consented, enrolled subjects will receive a pulsatile nasal irrigator to clean their nose twice daily. They will complete the initial ESS and have a blood test for Hct, Hgb, serum ferritin, liver function tests, renal function tests and if of child-bearing age, a urine pregnancy test. They will return 1 week later and will then receive intranasal sprays of ranibizumab administered once per week during clinic. Patients will be treated with a total of 4 applications of ranibizumab in a fine mist spray. This will be done with 2 mgs in 0.2cc; 0.1cc sprayed into each nostril a day; therefore 8 mgs will require 4 days of application. If, following the first treatments, they experience recurrent bleeding (equal to or less than a 75% reduction in their ESS number as compared to pre treatment), they will be offered the opportunity to be prescribed a second administration, to be completed in the same way (4 applications of 4 mg in 0.4 cc with 0.2cc per nostril).
At the completion of the treatment, subjects will be observed monthly for examination and ESS until 6 months from the first treatment. At 3 and 6 months laboratory tests will be performed.
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Ranibizumab
Patients treated with topical ranibizumab.
干预措施: Ranibizumab (Drug)
结局指标
主要结局
Epistaxis as measured by the HHT Epistaxis Severity Score (ESS), hematocrit, and hemoglobin and serum feritin levels.
时间窗: Monthly until 6 months from the first treatment.
Epistaxis will be evaluated on a monthly basis by the HHT Epistaxis Severity Score (ESS), hematocrit, and hemoglobin and serum ferritin levels. This will continue through 6 months after the first treatment.
次要结局
未报告次要终点
研究者
Terence Davidson
Associate Dean/Professor
University of California, San Diego
