A Phase 1/2 interventional study of Simvastatin in Sickle Cell Disease, sponsored by UCSF Benioff Children's Hospital Oakland. Completed at 1 site in United States. Open to participants aged 10 Years and older. Per ClinicalTrials.gov, last updated 2016-12-19.
Sponsored by UCSF Benioff Children's Hospital Oakland · Phase 1/2, Interventional, and Treatment
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.
1,103 studies on the registry are indexed under Anemia, Sickle Cell; 235 are open to participants now.
This study's enrollment of 24 is below the median of 40 across 750 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →UCSF Benioff Children's Hospital Oakland is the lead sponsor of 53 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months
Drug: Simvastatin
40 mg, orally, once daily for 3 months
Also known as: Zocor
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.
Time frame: Baseline and 3 months
Change in Plasma High Sensitivity C-reactive Protein
Mean difference in plasma high sensitivity C-reactive protein level, before and after treatment with simvastatin
Time frame: Baseline and 3 months
Change From Baseline in Total Cholesterol Level After Treatment With Simvastatin
Time frame: Baseline and 3 months
| Milestone | Simvastatin |
|---|---|
| Started | 24 |
| Completed | 19 |
| Not completed | 5 |
| Withdrew: Lost to follow-up | 2 |
| Withdrew: Withdrawal by subject | 2 |
| Withdrew: Physician decision | 1 |
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.
| proportion of pain days | Baseline | Simvastatin |
|---|---|---|
| Change in Frequency of Vaso-occlusive Pain Events, Before and After Treatment With Simvastatin | 0.2365 ± 0.21 | 0.1311 ± 0.15 |
Mean difference in plasma high sensitivity C-reactive protein level, before and after treatment with simvastatin
| mg/mL | Baseline | Simvastatin |
|---|---|---|
| Change in Plasma High Sensitivity C-reactive Protein | 7.2 ± 11 | 2.989 ± 4.169 |
| mmol/L | Baseline Cholesterol | Simvastatin Treatment |
|---|---|---|
| Change From Baseline in Total Cholesterol Level After Treatment With Simvastatin | 124 ± 27 | 101 ± 17 |
Collected over Safety data were collected on each participant during the treatment period (3 months) and at 1 month follow up. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Simvastatin | — | 5/19 (26.3%) | 1/19 (5.3%) |
| Event | Simvastatin |
|---|---|
| sickle cell vaso-occlusive pain crisisBlood and lymphatic system disorders | 4/19 |
| hyperhemolysisBlood and lymphatic system disorders | 1/19 |
| Event | Simvastatin |
|---|---|
| facial rashSkin and subcutaneous tissue disorders | 1/19 |
| Age, Categorical(Participants) | Simvastatin |
|---|---|
| <=18 years | 11 |
| Between 18 and 65 years | 8 |
| >=65 years | 0 |
| Age, Continuous(years) | Simvastatin |
|---|---|
| Mean | 18.5 ± 6.8 |
| Gender(Participants) | Simvastatin |
|---|---|
| Female | 13 |
| Male | 6 |
| Race (NIH/OMB)(Participants) | Simvastatin |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 18 |
| White | 1 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Receiving hydroxyurea (HU) therapy(participants) | Simvastatin |
|---|---|
| Number | 10 |
Plan to share: No — publication
This study is completed, as verified in Jan 2016. You cannot join it, but the record below documents what was studied.
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UCSF Benioff Children's Hospital Oakland