Study of Propranolol as Anti-Adhesive Therapy in Sickle Cell Disease (SCD)
Status:
Completed
Trial end date:
2013-12-01
Target enrollment:
Participant gender:
Summary
An open label, prospective, randomized cross-over phase II study in up to 60 sickle cell
patients who are either homozygous for Hb S or have HbSB0 thalassemia. Initially, each
patient will be treated for 6 weeks with placebo or a standard dose of propranolol (40 mg)
every 12 hrs. This will be followed by a 2-week washout period after which, patients will
receive the other treatment modality (placebo or propranolol).
We Hypothesize that propranolol administered in vivo on a daily basis for 6 weeks (1) will
decrease baseline adhesion to endothelial cells and will substantially abrogate
epinephrine-stimulated adhesion to endothelial cells, as measured in vitro; (2) will improve
biomarkers of endothelial activation and dysfunction; and (3) can be safely used in patients
with SCD. Thus, the use of propranolol in SCD may represent a safe and effective means of
anti-adhesive therapy in SCD.
Study Objectives:
Primary Objective:
• To establish the safety and efficacy of long-term therapy with propranolol as an
anti-adhesive therapy for SCD.
Secondary Objective:
• To evaluate changes in soluble markers of endothelial activation and dysfunction.
Correlative Science Objective:
• To determine whether response to propranolol therapy is associated with polymorphisms in
genes encoding the proteins involved in the upregulation of Sickle Red Blood Cell (SS RBC)
adhesion by epinephrine.