Effect of Simvastatin Treatment on Vaso-occlusive Pain in Sickle Cell Disease
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase 2 Study of Simvastatin Treatment Effects on Vaso-occlusive Pain in Sickle Cell Disease|
- Vaso-occlusive pain events [ Time Frame: 4 months ] [ Designated as safety issue: No ]The effect of simvastatin treatment will be assessed by measuring changes in the frequency and intensity of vaso-occlusive pain events from baseline in subjects treated with simvastatin.
- Correlation of plasma biomarkers with clinical measures of vaso-occlusive pain [ Time Frame: 4 months ] [ Designated as safety issue: No ]Changes in plasma biomarkers of vascular injury (NOx, IL-6, hs-CRP, VCAM-1, ICAM-1, E-selectin, VEGF) will be correlated with changes in vaso-occlusive pain within subjects at baseline and multiple timepoints during and after treatment with simvastatin.
- Clinical safety of simvastatin [ Time Frame: 4 months ] [ Designated as safety issue: Yes ]Clinical and laboratory monitoring for simvastatin-related adverse effects, including myopathy, will be monitored closely in all subjects. Clinical safety outcomes to be measured include changes in serum chemistries and blood cell counts, medication use and specific adverse events.
|Study Start Date:||February 2012|
|Estimated Study Completion Date:||June 2015|
|Estimated Primary Completion Date:||May 2015 (Final data collection date for primary outcome measure)|
Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months
40 mg, orally, once daily for 3 months
Other Name: Zocor
Sickle cell disease (SCD) is characterized by recurrent vaso-occlusive episodes and a chronic inflammatory state leading to progressive multi-organ injury. The pathophysiology of SCD is related to endothelial dysfunction driven largely by impaired nitric oxide (NO) homeostasis and chronic inflammation. Through multiple mechanisms, including upregulation of NO, statins have been shown to confer protection from endothelial injury, independent of their cholesterol-lowering properties.
By inhibiting inflammation and several common pathways leading to vascular damage,simvastatin may help prevent the acute and chronic complications of SCD. The objective of this study is to determine whether our preliminary results showing simvastatin-associated reductions in plasma markers of vascular injury will translate into a reduction in vaso-occlusive pain episodes in patients with SCD. A web-based, smartphone-accessible electronic pain diary will be used to monitor frequency and intensity of vaso-occlusive pain in SCD patients treated with a single daily dose of simvastatin.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01702246
|Contact: Carolyn Hoppe, M.D.||firstname.lastname@example.org|
|Contact: Lori Styles, M.D.||email@example.com|
|United States, California|
|Children's Hospital & Research Center Oakland||Recruiting|
|Oakland, California, United States, 94609|
|Contact: Carolyn Hoppe, M.D. 510-428-3193 firstname.lastname@example.org|
|Contact: Lori Styles, M.D. email@example.com|
|Principal Investigator: Carolyn Hoppe, M.D.|
|Sub-Investigator: Lori Styles, M.D.|
|Sub-Investigator: Frans Kuypers, Ph.D.|
|Principal Investigator:||Carolyn Hoppe, MD||Children's Hospital & Research Center Oakland|
|Study Chair:||Elliott P Vichinsky, MD||Children's Hospital & Research Center Oakland|