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临床试验/NCT02187445
NCT02187445已完成1 期

Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial

Vanderbilt University1 个研究点 分布在 1 个国家目标入组 36 人开始时间: 2014年6月最近更新:
适应症
干预措施
相关药物

试验速览

阶段
1 期
状态
已完成
入组人数
36
试验地点
1
主要终点
The acceptability of budesonide inhalation suspension

研究概览

简要总结

Acute and chronic pulmonary complications with concomitant inflammatory response are a leading cause of morbidity and mortality in children with sickle cell disease (SCD). Acute chest syndrome (ACS), defined broadly as an increase in respiratory effort, fever and new radiodensity on chest x-ray, is a major cause of death in children and adults with SCD. There is a high rate of ACS in children between 1 and 4 years of age that is associated with an asthma diagnosis, and children with ACS events before 4 years of age have a 50% rate of being hospitalized for either ACS or pain within 1 year of admission. For children with SCD that develop ACS, the investigators propose that the use of budesonide inhalation suspension (BIS) will attenuate pulmonary inflammation after an ACS episode and will decrease future vaso-occlusive pain and ACS episodes. Through a single-arm prospective feasibility trial and in preparation for a limited-institution randomized trial, the investigators plan to test the following primary hypothesis for a phase III definitive trial: In children with SCD admitted to the hospital for an ACS episode between 1 and 4 years of age, low dose BIS for 6 months will result in a 50% reduction in the recurrent incidence rate of ACS or pain requiring hospitalization. Through this trial, the investigators will determine the acceptability of and adherence to BIS in the study population. The investigators will track respiratory symptoms in cases versus controls over 6 months. Finally, the investigators will explore the impact of BIS on biological correlates (sVCAM-1).

研究设计

研究类型
Interventional
分配方式
Na
干预模型
Single Group
主要目的
Prevention
盲法
None

入排标准

年龄范围
1 Year 至 4 Years(Child)
性别
All
接受健康志愿者

入选标准

  • confirmed diagnosis of sickle cell disease (SCD)
  • age between 1 and 4 years (must have reached 1st but not yet 4th birthday)
  • a prior diagnosis of ACS, defined as acute respiratory illness with a new radiodensity on CXR, and one of the following: fever (temperature > 38.50C), decrease in oxygen saturation more than 3% from baseline, or increase in respiratory rate above baseline

排除标准

  • patients already taking inhaled corticosteroids
  • those receiving blood transfusions for elevated TCD or strokes
  • presents over 2 weeks after discharge from hospital following initial ACS episode.
  • Participants may be on hydroxyurea and participate in this trial.

研究组 & 干预措施

Budesonide inhalation suspension

Experimental

To determine the acceptability of budesonide inhalation suspension (BIS) 0.5 QD for 6 months for children with SCD that develop ACS between 1 and 4 years of age (n=10).

干预措施: Budesonide inhalation suspension (Drug)

结局指标

主要结局

The acceptability of budesonide inhalation suspension

时间窗: 6 Months

Specific Aim 1: To determine the acceptability of budesonide inhalation suspension (BIS) 0.5 QD for 6 months for children with SCD that develop ACS between 1 and 4 years of age (n=10). We will determine the proportions of eligible families who were willing to participate and families that enrolled and elected to stay throughout the six months of the trial. We will also assess adherence to BIS using the Morisky scale; this will be our primary outcome. If the participation rate for the trial is less than 60%, the dropout rate is greater than 20%, or if our adherence rate is poor as measured by the Morisky scale, then alternative strategies for recruitment, retention and adherence must be considered.

次要结局

  • The impact of BIS on biological correlates of inflammation.(12 weeks (or between 8-16 weeks) and at 24 weeks (or between 20-28 weeks))

研究者

申办方类型
Other
责任方
Principal Investigator
主要研究者

Michael DeBaun

Professor of Pediatrics and Medicine, Vice Chair of Clinical Research in Pediatrics, JC Peterson Endowed Chair in Pediatrics, Director, Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease

Vanderbilt University

研究点 (1)

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