Clinical trial NCT01517893: Clinical Trial of Simvastatin to Treat Generalized Vitiligo
Official title: A Phase-II, Randomized, Placebo-controlled Trial of Simvastatin in Generalized Vitiligo
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Vitiligo
Keywords: vitiligo
simvastatin
Lead sponsor: University of Massachusetts, Worcester
Sponsor class: OTHER
First posted: 2012-01-25
Enrollment: 15
Sex: MALE
Minimum age (years): 18
Maximum age (years): 64
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Number of Participants With a Decrease in Vitiligo Area Scoring Index (VASI) Score
timeFrame: Assessed at baseline and final study visit, 6 months after randomization
Secondary outcomes:
measure: Number of Participants With Increase in Investigator's Global Assessment Score
timeFrame: Assessed at baseline and final study visit, 6 months after randomization
measure: Number of Participants Experiencing Toxicity From of High-dose Simvastatin .
timeFrame: Assessed at baseline, then monthly until final study visit, six months after randomization.
measure: Change in Sentinel Patch Area
timeFrame: Assessed at baseline and final study visit, 6 months after randomization
measure: Change in Quality of Life Score by Using DERMATOLOGY LIFE QUALITY INDEX (DLQI)
timeFrame: Assessed at baseline and final study visit, 6 months after randomization
measure: Number of Participants With an Increase in Patient's Global Assessment Score
timeFrame: Assessed at baseline and final study visit, 6 months after randomization
measure: Serum CXCL10 Levels From the First and Last Available Clinic Visits Were Measured Via ELISA
timeFrame: Assessed at baseline and final study visit, 6 months after randomization
measure: CXCR3 Expression on CD8+ T Cells
timeFrame: Assessed prior to treatment and periodically while on treatment
Other outcomes:
Locations:
city: Worcester
country: United States
facility: University of Massachusetts Medical School Clinical Research Center
state: Massachusetts
Study officials:
affiliation: University of Massachusetts, Worcester
name: John E. Harris, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: