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Phase 2 N=24 Treatment

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

Sickle Cell Disease

Enrolled (actual)
24
Serious AEs
26.3%
Results posted
Dec 2016
Primary outcome: Primary: Change in Frequency of Vaso-occlusive Pain Events, Before and After Treatment With Simvastatin — 0.2365; 0.1311 proportion of pain days — p=0.005

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Simvastatin (Drug)
Age
Pediatric, Adult, Older Adult · 10+ yrs
Sex
All
Sponsor
UCSF Benioff Children's Hospital Oakland
Primary completion
May 2015

Outcome Measures

OutcomeResultp-value
PRIMARY
Change in Frequency of Vaso-occlusive Pain Events, Before and After Treatment With Simvastatin
0.2365; 0.1311 0.005 sig
SECONDARY
Change in Plasma High Sensitivity C-reactive Protein
7.2; 2.989 0.003 sig
SECONDARY
Change From Baseline in Total Cholesterol Level After Treatment With Simvastatin
124; 101 0.001 sig

Summary

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.

Eligibility 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 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
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01702246). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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