Inhaled Mometasone to Promote Reduction in Vasoocclusive Events
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 54
- 试验地点
- 1
- 主要终点
- Number of Participants Who Completed Follow up
研究概览
简要总结
The proposed research is designed to test the global hypothesis that inhaled corticosteroids (ICS), a therapy developed to treat asthma, will prevent vasoocclusive painful episodes in adults with Sickle Cell Disease (SCD) who wheeze, but do not meet criteria for a diagnosis of asthma. The specific aims of this proposal are 1) Conduct a feasibility study - a randomized controlled trial of ICS for adults with SCD who do not meet criteria for a diagnosis of asthma but report recurrent cough or wheezing, 2) Measure the effects of ICS on biological correlates of pulmonary inflammation (as determined by exhaled nitric oxide) and vascular injury (as determined by sVCAM) in SCD, and 3) Compare properties of traditional and Bayesian adaptive clinical trial design for therapeutic trials in SCD in preparation for designing a definitive trial of ICS. These aims have the potential to 1) change the standard of care for individuals with SCD and recurrent cough or wheeze, 2) provide insight into the pathogenesis of non-asthmatic wheezing in SCD and its response to treatment, 3) explore the suitability of innovative clinical trial designs to overcome the challenges that have hindered therapeutic innovation for SCD.
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
- 15 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 15 or older
- •Sever SCD phenotypes (Hb SS and Sβthalassemia0)
- •A positive response to cough/wheeze questions
排除标准
- •Patient carries a physician diagnosis of asthma
- •Patient is prescribed asthma medications
- •Patient is currently having a painful crisis (as defined by validated pain diary questions)
- •Patient has acute respiratory symptoms
- •Known hypersensitivity to milk proteins
- •Meets criteria for our operational diagnosis of asthma
- •More than 15 ED visits for pain over the preceding 12 months
- •Admitted or discharged from the hospital for SCD pain within the last 7 days
研究组 & 干预措施
Mometasone Furoate
1 puff daily (220mcg) for 16 weeks
干预措施: Mometasone Furoate (Drug)
Placebo
1 puff daily for 16 weeks. Training inhaler that does not contain any medication (placebo).
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants Who Completed Follow up
时间窗: at 2 years
Feasibility is determined by calculating the proportion of randomized participants who complete follow up and a minimum of 30 pain diaries with good adherence to the study medication vs. the number enrolled.
次要结局
- Change in Exhaled Nitric Oxide (eNO)(Before ICS therapy begins and at 8 weeks post enrollment)
- Change in Soluble Vascular Cell Adhesion Molecule (sVCAM) Level(Before ICS therapy begins and at 8 weeks post enrollment)
- Change in Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me)(baseline and week 20)
- The Medication Adherence Report Scale(20 weeks)
- Change in the Numerical Rating Scale (NRS) for Pain(baseline and 20 weeks)
- Asthma Control Test(8 weeks)
- Admissions or Visits to the Hospital(baseline through 8 weeks)
- Change in Reticulocytes Count(baseline and 8 weeks)
- Change in FEV1/FVC(baseline and 8 weeks)
研究者
Jeffrey Glassberg
Assistant Professor
Icahn School of Medicine at Mount Sinai