Sickle Cell Disease Clinical Trial
Official title:
Phase I/II Study of Simvastatin (Zocor) Therapy in Sickle Cell Disease
Recent clinical and experimental data indicate that statins have effects beyond cholesterol lowering that may be beneficial in sickle cell disease by protecting the vascular endothelium. Statins have been shown to attenuate endothelial dysfunction through their anti-inflammatory, anti-oxidant and anti-thrombotic properties. This phase I/II dose-escalating trial is designed to assess the safety and potential clinical efficacy of oral simvastatin (Zocor)in adolescents and adults with sickle cell disease (SCD).
Although statins have been used extensively for their cholesterol-lowering effects, recent
clinical and experimental data indicate that statins regulate yet other processes, many of
which play a major role in sickle cell disease (SCD). Independent of their
cholesterol-lowering effects, statins have been shown to prevent damage to blood vessels in
several ways, through upregulation of endothelial nitric oxide (NO)and decreased
inflammation. Numerous studies documenting the protective effects of statins, together with
data showing the therapeutic role of NO in SCD, provide the basis for investigating the
potential clinical benefit of simvastatin in SCD.
Data supporting the safety and tolerability of simvastatin in patients with SCD are now
needed. For this phase I/II dose-escalation study of oral simvastatin in SCD, we propose the
following specific aims:
1. To obtain preliminary efficacy data on the effects of oral simvastatin on plasma
biomarkers of endothelial injury in patients with SCD, and
2. To assess the safety and tolerability of oral simvastatin in patients with SCD.
;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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