Clinical Trial of Simvastatin to Treat Generalized Vitiligo

Vitiligo is an autoimmune disease caused by autoreactive CD8+ T lymphocytes that target melanocytes, and interferon-γ-induced CXCL10 plays an important role.1 Simvastatin inhibits interferon-γ signaling by blocking activation of STAT12 and prevented and reversed disease in our mouse model.3 A case report described a patient with vitiligo who repigmented with simvastatin.4 We conducted a small, randomized, double-blind, placebo-controlled, phase II clinical trial to test simvastatin as a treatment for vitiligo. After obtaining informed consent, we enrolled men ages 18 to 64 years with vitiligo affecting 3% to 50% of their body surface area (BSA). We excluded patients with a segmental presentation; those already taking 3-hydroxy-3-methylglutaryl-coenzyme A reductase inhibitor; those with existing thyroid disease; and women, based on their increased risk of simvastatin-induced myopathy.

Inclusion criteria

• male gender
• ages 18-64
• at least one vitiligo skin lesion measuring at least 2x2 cm in size
• willing and able to understand and sign informed consent
• able to complete study and comply with study procedures

Exclusion criteria

• history of segmental vitiligo
• allergy to statin medications
• use of statin medications due to cardiac risks.
• use of any medications contraindicated with use of simvastatin
• use of topical vitiligo treatments in past 4 weeks
• use of laser or light-based vitiligo treatments within the past 8 weeks
• treatment with immunomodulating oral medications in the past 4 weeks
• use of statin medications in the past 8 weeks
• evidence of hepatic dysfunction, personal or family history of non-alcoholic steatotic hepatitis, or personal history of hepatitis
• evidence of renal dysfunction
• history of myopathy or rhabdomyolysis, or elevated baseline creatinine kinase
• recent history of alcohol or drug abuse
• history of diabetes
• untreated hypothyroidism
• other conditions that require the use of interfering topical or systemic therapy
• other current conditions that might interfere with study assessments such as, but not limited to, atopic dermatitis and psoriasis
• clinically significant abnormal findings or conditions which might, in the opinion of the Principal Investigator, interfere with study evaluations or pose a risk to subject safety during the study.