Clinical Trial: Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Inhaled Corticosteroid Use to Prevent Acute Chest Syndrome Recurrence in Children Between 1 and 4 With Sickle Cell Disease: a Feasibility Trial
Brief Summary: 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).
Detailed Summary:
Sponsor: Vanderbilt University
Current Primary Outcome: The acceptability of budesonide inhalation suspension [ Time Frame: 6 Months ]
Original Primary Outcome: The acceptability of budesonide inhalation suspension [ Time Frame: 6 Months ]
Current Secondary Outcome: The impact of BIS on biological correlates of inflammation. [ Time Frame: 12 weeks (or between 8-16 weeks) and at 24 weeks (or between 20-28 weeks) ]
Original Secondary Outcome: The impact of BIS on biological correlates of inflammation. [ Time Frame: 12 weeks and at 24 weeks ]
Information By: Vanderbilt University Medical Center
Dates:
Date Received: June 27, 2014
Date Started: June 2014
Date Completion:
Last Updated: February 16, 2017
Last Verified: February 2017