Phase 2
N=20
Simvastatin (Zocor) Therapy in Sickle Cell Disease
Sickle Cell Disease
Bottom Line
View on ClinicalTrials.gov: NCT00508027 ↗Enrolled (actual)
20
Serious AEs
4.8%
Results posted
Aug 2013
Primary outcome: Primary: Change in Total Cholesterol Level — -16; -18; -18 mg/dL
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Simvastatin (Drug)
- Age
- Pediatric, Adult, Older Adult · 13+ yrs
- Sex
- All
- Sponsor
- UCSF Benioff Children's Hospital Oakland
- Primary completion
- Dec 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Total Cholesterol Level |
-16; -18; -18 | — |
| PRIMARY Change in Hemoglobin Level |
-0.2; 0.1; -0.4 | — |
| PRIMARY Change in Serum Creatine Kinase Levels |
57; 20; 62 | — |
| PRIMARY Change in Serum Alanine Transaminase (ALT) Levels |
4; 3; -3 | — |
| PRIMARY Change in Serum Creatinine Levels |
0.03; 0.04; -0.1 | — |
Summary
Recent clinical and experimental data indicate that statins have effects beyond cholesterol lowering that may be beneficial in sickle cell disease by protecting the vascular endothelium. Statins have been shown to attenuate endothelial dysfunction through their anti-inflammatory, anti-oxidant and anti-thrombotic properties. This phase I/II dose-escalating trial is designed to assess the safety and potential clinical efficacy of oral simvastatin (Zocor)in adolescents and adults with sickle cell disease (SCD).
Eligibility Criteria
Inclusion Criteria
- Established diagnosis of sickle cell disease (HbSS, SC or Sβ-thalassemia)
- Age greater than or equal to thirteen years
- Weight greater than or equal to 35 kg
Exclusion Criteria
- Renal dysfunction (Serum Creatinine > 1.5 UNL)
- Hepatic dysfunction (ALT > 2X UNL)
- Pretreatment total cholesterol 1X UNL (215 U/L)
- Pregnancy/lactation
- RBC transfusion in the last 30 days
- Vaso-Occlusive Event needing hospitalization in the past 30 days
- Treatment with any statin drugs within the past 30 days
- Treatment with drugs having known metabolic interactions with statin drugs (e.g. cytochrome P450 3A4 metabolism), including ketoconazole, itraconazole, fluconazole, erythromycin, clarithromycin, azithromycin, niacin (nicotinic acid), digoxin, coumadin, sildenafil or amiodarone within the past 30 days
- Treatment (past or present) with amiodarone
- Musculoskeletal disorder associated with an elevated creatine kinase level
- Past or present history of substance abuse (alcohol, cocaine, amphetamines, heroin, PCP)
- Allergy to statins
Data sourced from ClinicalTrials.gov (NCT00508027). 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.