Clinical Trial of Simvastatin to Treat Generalized Vitiligo

NCT ID: NCT01517893

Last Updated: 2018-11-14

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-01-31
• Study Completion Date: 2014-12-31

Brief Summary

The investigators purpose is to initiate a phase II, randomized, placebo-controlled clinical trial to test simvastatin, an FDA-approved medication for hypercholesterolemia, as a new treatment for vitiligo. The aims of this placebo-controlled study seek to determine the safety and potential efficacy of simvastatin 80mg daily versus placebo in adult male patients with generalized vitiligo. Additionally, the investigators will collect blood to examine the effect of simvastatin on autoreactive CD8+ T cells in vitiligo patients.

Detailed Description

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.

Conditions

Vitiligo

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Intervention arm

Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated

Group Type: EXPERIMENTAL

Simvastatin

Intervention Type: DRUG

Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated

Placebo Arm

Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated

Interventions

Simvastatin

Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated

Intervention Type: DRUG

Placebo

Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 64 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

John Harris

OTHER

Sponsor Role: lead

Responsible Party

John Harris

Principal Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

John E. Harris, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

University of Massachusetts, Worcester

Locations

University of Massachusetts Medical School Clinical Research Center

Worcester, Massachusetts, United States

Countries

United States

Other Identifiers

UM-DERM001

Identifier Type: -

Identifier Source: org_study_id