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

This study has been completed.
Sponsor:
Collaborator:
University of California, Los Angeles
Information provided by (Responsible Party):
Carolyn Hoppe, Children's Hospital & Research Center Oakland
ClinicalTrials.gov Identifier:
NCT01702246
First received: October 4, 2012
Last updated: October 26, 2016
Last verified: January 2016
  Purpose
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.

Condition Intervention Phase
Sickle Cell Disease Drug: Simvastatin Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Study of Simvastatin Treatment Effects on Vaso-occlusive Pain in Sickle Cell Disease

Resource links provided by NLM:


Further study details as provided by Carolyn Hoppe, Children's Hospital & Research Center Oakland:

Primary Outcome Measures:
  • 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 Outcome Measures:
  • 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

  • Change From Baseline in Total Cholesterol Level After Treatment With Simvastatin [ Time Frame: Baseline and 3 months ]

Enrollment: 24
Study Start Date: February 2012
Study Completion Date: June 2015
Primary Completion Date: May 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: simvastatin
Simvastatin (Zocor), 40mg tablet, 40mg orally once daily, for 3 months
Drug: Simvastatin
40 mg, orally, once daily for 3 months
Other Name: Zocor

Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   10 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01702246

Locations
United States, California
Children's Hospital and Research Center Oakland
Oakland, California, United States, 94609
Sponsors and Collaborators
Children's Hospital & Research Center Oakland
University of California, Los Angeles
Investigators
Principal Investigator: Carolyn Hoppe, MD Children's Hospital & Research Center Oakland
  More Information

Publications:
Responsible Party: Carolyn Hoppe, Associate Hematologist-Oncologist, Children's Hospital & Research Center Oakland
ClinicalTrials.gov Identifier: NCT01702246     History of Changes
Other Study ID Numbers: DDCF-ICRA-2011
Study First Received: October 4, 2012
Results First Received: January 15, 2016
Last Updated: October 26, 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: publication

Keywords provided by Carolyn Hoppe, Children's Hospital & Research Center Oakland:
sickle cell disease
statins
vaso-occlusive pain
inflammation
biomarkers

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Simvastatin
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors

ClinicalTrials.gov processed this record on August 18, 2017