Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease
Status:
Withdrawn
Trial end date:
2018-10-01
Target enrollment:
Participant gender:
Summary
Sickle cell anemia (SCA) is a life-threatening, monogenic disorder associated with early
death when compared to individuals without SCA. Pulmonary complications, namely acute chest
syndrome, obstructive lung disease and pulmonary hypertension, are the most common causes of
death in patients with SCA. Recent studies suggest that lung specific inflammation is a
hallmark of SCA and underlies pulmonary pathology. To date, no therapy has been shown to
improve the pulmonary complications of SCA. Macrolides have pleomorphic effects in the lung
with improvement in pulmonary function, symptoms and inflammatory markers demonstrated in
several inflammatory pulmonary conditions such as cystic fibrosis, asthma, COPD and
post-transplant bronchiolitis obliterans. Investigators hypothesize that low dose macrolide
therapy is well tolerated and can improve pulmonary function and symptoms in patients with
SCA. The objective of this project is to assess the feasibility of macrolides to attenuate or
reverse the decrease in %predicted FEV1 in adults with SCA in a single-site, randomized,
placebo-controlled feasibility trial.