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Completed

NCT Number: NCT01702246

Effect of Simvastatin Treatment on Vaso-occlusive Pain in Sickle Cell Disease

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.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Inflammation Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

10 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Children's Hospital and Research Center Oakland

Oakland, California, 94609, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Sickle cell disease (HbSS or S/β0 thalassemia)
  • ≥ 3 vaso-occlusive pain episodes in the year prior to enrollment
  • Age ≥ 10 years
  • Weight > 30 kg

Exclusion criteria

  • Creatine kinase (CK) > UNL
  • Total cholesterol < 90 mg/dL, or triglycerides <30mg/dL
  • Renal dysfunction (Creatinine > 1.5-fold UNL)
  • Hepatic dysfunction (ALT > 2-fold UNL)
  • Treatment with drugs having known metabolic interactions with statins within the past 30 days
  • Vaso-occlusive pain requiring hospitalization within past 30 days
  • Red blood cell transfusion within the past 30 days
  • Pregnancy/lactation
  • Musculoskeletal disorder associated with an elevated CK level
  • Past or present history of substance abuse (alcohol, cocaine, amphetamines, heroin)
  • Chronic pain caused by avascular necrosis of the bone (AVN) or leg ulcers, and pain due to trauma or causes other than SCD.
  • Major cognitive or neurological impairments that may hamper the ability to use the smartphone or complete the electronic pain diary in this study

Treatment and study plan

simvastatin

Drug

40 mg, orally, once daily for 3 months

Other names: Zocor

Primary outcomes

  1. Change in Frequency of Vaso-occlusive Pain Events, Before and After Treatment With Simvastatin

    Time frame: Baseline and 3 months

    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.

Secondary outcomes

  1. Change in Plasma High Sensitivity C-reactive Protein

    Time frame: Baseline and 3 months

    Mean difference in plasma high sensitivity C-reactive protein level, before and after treatment with simvastatin

  2. Change From Baseline in Total Cholesterol Level After Treatment With Simvastatin

    Time frame: Baseline and 3 months

Sponsors and collaborators

Lead sponsor

UCSF Benioff Children's Hospital Oakland

Other

Collaborators

  • University of California, Los Angeles

Registry information

Official study title

Phase 2 Study of Simvastatin Treatment Effects on Vaso-occlusive Pain in Sickle Cell Disease

Important dates

Study start
2012
Primary completion
2015
Study completion
2015
First posted
Oct 8, 2012
Registry last updated
Dec 19, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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