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| Name | Class |
|---|---|
| University of California, Los Angeles | OTHER |
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The purpose of this study is to determine whether simvastatin is effective in reducing the frequency and intensity of vaso-occlusive pain episodes in patients with sickle cell disease.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| simvastatin | Experimental | Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Simvastatin | Drug | 40 mg, orally, once daily for 3 months |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Frequency of Vaso-occlusive Pain Events, Before and After Treatment With Simvastatin | The effect of simvastatin treatment will be assessed by measuring the difference from baseline in the mean frequency (and intensity) of vaso-occlusive pain events, after treatment with simvastatin. Pain rate (proportion of pain days) was defined as the number of days reported with sickle cell disease-related pain divided by the number of daily pain diaries completed. | Baseline and 3 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Plasma High Sensitivity C-reactive Protein | Mean difference in plasma high sensitivity C-reactive protein level, before and after treatment with simvastatin | Baseline and 3 months |
| Change From Baseline in Total Cholesterol Level After Treatment With Simvastatin |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Carolyn Hoppe, MD | UCSF Benioff Children's Hospital Oakland | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital and Research Center Oakland | Oakland | California | 94609 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21477202 | Background | Hoppe C, Kuypers F, Larkin S, Hagar W, Vichinsky E, Styles L. A pilot study of the short-term use of simvastatin in sickle cell disease: effects on markers of vascular dysfunction. Br J Haematol. 2011 Jun;153(5):655-63. doi: 10.1111/j.1365-2141.2010.08480.x. Epub 2011 Apr 8. |
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publication
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| ID | Title | Description |
|---|---|---|
| FG000 | Simvastatin | Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months Simvastatin: 40 mg, orally, once daily for 3 months |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Simvastatin | Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months Simvastatin: 40 mg, orally, once daily for 3 months |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Frequency of Vaso-occlusive Pain Events, Before and After Treatment With Simvastatin | The effect of simvastatin treatment will be assessed by measuring the difference from baseline in the mean frequency (and intensity) of vaso-occlusive pain events, after treatment with simvastatin. Pain rate (proportion of pain days) was defined as the number of days reported with sickle cell disease-related pain divided by the number of daily pain diaries completed. | Posted | Mean | Standard Deviation | proportion of pain days | Baseline and 3 months |
|
Safety data were collected on each participant during the treatment period (3 months) and at 1 month follow up
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Simvastatin | Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months Simvastatin: 40 mg, orally, once daily for 3 months |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| sickle cell vaso-occlusive pain crisis | Blood and lymphatic system disorders | Systematic Assessment | Four participants were hospitalized for sickle cell related vaso-occlusive pain crisis (expected SAE) |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| facial rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Carolyn Hoppe | UCSF Benioff Children's Hospital Oakland | (510)428-3193 | choppe@mail.cho.org |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D007249 | Inflammation |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D019821 | Simvastatin |
| ID | Term |
|---|---|
| D008148 | Lovastatin |
| D009281 | Naphthalenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D006841 | Hydrocarbons, Aromatic |
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| Baseline and 3 months |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Gender | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Receiving hydroxyurea (HU) therapy | Number | participants |
|
|
|
|
| Secondary | Change in Plasma High Sensitivity C-reactive Protein | Mean difference in plasma high sensitivity C-reactive protein level, before and after treatment with simvastatin | Posted | Mean | Standard Deviation | mg/mL | Baseline and 3 months |
|
|
|
|
| Secondary | Change From Baseline in Total Cholesterol Level After Treatment With Simvastatin | change in baseline cholesterol level from baseline, after treatment with simvastatin | Posted | Mean | Standard Deviation | mmol/L | Baseline and 3 months |
|
|
|
|
| 5 |
| 19 |
| 1 |
| 19 |
|
| hyperhemolysis | Blood and lymphatic system disorders | Systematic Assessment | One participant was hospitalized for sickle cell related hyperhemolytic event (unrelated to study drug) |
|
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| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D006844 |
| Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D011083 | Polycyclic Compounds |