Simvastatin (Zocor) Therapy in Sickle Cell Disease

• Status: Completed
• Trial end date: 2011-12-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital & Research Center Oakland; UCSF Benioff Children's Hospital Oakland
• Collaborators: Department of Health and Human Services; FDA Office of Orphan Products Development
• Treatments: Simvastatin

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 < 100 mg/dL or triglycerides < 30 mg/dL

• Pretreatment baseline creatine kinase >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