Inhaled Mometasone to Reduce Painful Episodes in Patients With Sickle Cell Disease
Status:
Completed
Trial end date:
2017-11-01
Target enrollment:
Participant gender:
Summary
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.