Simvastatin (Zocor) Therapy in Sickle Cell Disease
This research study focuses on individuals with sickle cell disease (SCD). There is scientific evidence suggesting that treatment with the statin drug, simvastatin (Zocor), may be helpful for people with vascular diseases like SCD. This study looks at the effect this drug may have in preventing injury to the blood vessels. It will check for a change in the levels of certain substances in the blood that can damage blood vessels. The study will also help us find out whether, and at what dose, simvastatin is safe and useful for people with SCD.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase I/II Study of Simvastatin (Zocor) Therapy in Sickle Cell Disease|
- Evaluate the effect of simvastatin on plasma biomarkers of endothelial activation in three escalating treatment dosage (20mg/day,40mg/day,80mg/day) groups. [ Time Frame: 25 days ] [ Designated as safety issue: No ]
- Safety and tolerability of simvastatin in patients with sickle cell disease, as measured by changes in clinical adverse affects and laboratory (hematologic, renal, hepatic and lipid) profiles. [ Time Frame: 25 days ] [ Designated as safety issue: Yes ]
|Study Start Date:||June 2007|
|Study Completion Date:||December 2011|
|Primary Completion Date:||December 2011 (Final data collection date for primary outcome measure)|
Simvastatin, Dose Escalation
There are no arms in this study. Simvastatin will be given in a dose-escalating fashion to 3 sequential dosage groups (20 mg/day, 40 mg/day, 80 mg/day).
Comparison of 3 dosages of simvastatin given in a dose-escalating fashion. 20 mg, 40 mg, or 80 mg PO QD x 21 days followed by a drug taper x 4 days.
Other Name: Zocor
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:
- To determine specific dose-response effects of oral simvastatin on patients with SCD, and
- To assess the safety and tolerability of oral simvastatin in patients with SCD.
|United States, California|
|Children's Hospital and Research Center Oakland|
|Oakland, California, United States, 94609|
|Principal Investigator:||Carolyn C Hoppe, M.D.||Children's Hospital & Research Center Oakland|