Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Clinical Trial of Simvastatin to Treat Generalized Vitiligo

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01517893
Recruitment Status : Completed
First Posted : January 25, 2012
Results First Posted : March 12, 2015
Last Update Posted : November 14, 2018
Sponsor:
Information provided by (Responsible Party):
John Harris, University of Massachusetts, Worcester

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.

Condition or disease Intervention/treatment Phase
Vitiligo Drug: Simvastatin Drug: Placebo Phase 2

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase-II, Randomized, Placebo-controlled Trial of Simvastatin in Generalized Vitiligo
Study Start Date : January 2012
Actual Primary Completion Date : December 2013
Actual Study Completion Date : December 2014

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Vitiligo
Drug Information available for: Simvastatin

Arm Intervention/treatment
Experimental: Intervention arm
Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated
Drug: Simvastatin
Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated

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




Primary Outcome Measures :
  1. Number of Participants With a Decrease in Vitiligo Area Scoring Index (VASI) Score [ Time Frame: Assessed at baseline and final study visit, 6 months after randomization ]

    Number of participants with 33% decrease in the Vitiligo Area Scoring Index (VASI) from baseline to the last available study visit.

    Decrease in VASI score means improvement. Minimum value is 0, that means no vitiligo. maximum value is 100, that means 100% of the body surface area has vitiligo (total body surface area).



Secondary Outcome Measures :
  1. Number of Participants With Increase in Investigator's Global Assessment Score [ Time Frame: Assessed at baseline and final study visit, 6 months after randomization ]

    Increase in Investigator Global Assessment Scores of 30% or more from baseline to last available visit.

    Increase in score means improvement. 0% is no improvement at all. 100% is complete recovery.


  2. Number of Participants Experiencing Toxicity From of High-dose Simvastatin . [ Time Frame: Assessed at baseline, then monthly until final study visit, six months after randomization. ]
    The number of participants who experienced toxicity based upon monitored lab values (Liver Function Test) and patient symptoms for evidence of simvastatin toxicity

  3. Change in Sentinel Patch Area [ Time Frame: Assessed at baseline and final study visit, 6 months after randomization ]

    Change in percent depigmentation of sentinel patch lesion from baseline to last available study visit ( 6 months after randomization).

    positive numbers mean increase or worsening of sentinel patch area negative numbers mean decrease or improvement of sentinel patch area


  4. Change in Quality of Life Score by Using DERMATOLOGY LIFE QUALITY INDEX (DLQI) [ Time Frame: Assessed at baseline and final study visit, 6 months after randomization ]

    The aim of this questionnaire is to measure how much your skin problem has affected your life. We measured change in questionnaire score from baseline to end of study (at 6 months after randomization) of subjects randomized to treatment with simvastatin versus placebo. Change was measured as a drop in score at the end of 6 months of treatment.

    Minimum score is 0, maximum is 30. Higher value means worse score.


  5. Number of Participants With an Increase in Patient's Global Assessment Score [ Time Frame: Assessed at baseline and final study visit, 6 months after randomization ]
    Increase in Patient's Global Assessment Scores of 30% or more from baseline to last available visit Increase means improvement. minimum is 0% and maximum is 100%

  6. Serum CXCL10 Levels From the First and Last Available Clinic Visits Were Measured Via ELISA [ Time Frame: Assessed at baseline and final study visit, 6 months after randomization ]
    Determination of the effects of simvastatin treatment on Serum CXCL10 levels from the first and last available clinic visits were measured via ELISA in the blood of patients with vitiligo treated with simvastatin versus placebo

  7. CXCR3 Expression on CD8+ T Cells [ Time Frame: Assessed prior to treatment and periodically while on treatment ]
    Determination of the effects of simvastatin treatment on CXCR3 expression in melanocyte-specific, autoreactive CD8+ T cells in the blood of patients with vitiligo treated with simvastatin versus placebo



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 64 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01517893


Locations
Layout table for location information
United States, Massachusetts
University of Massachusetts Medical School Clinical Research Center
Worcester, Massachusetts, United States, 01655
Sponsors and Collaborators
John Harris
Investigators
Layout table for investigator information
Principal Investigator: John E. Harris, MD, PhD University of Massachusetts, Worcester

Layout table for additonal information
Responsible Party: John Harris, Principal Investigator, University of Massachusetts, Worcester
ClinicalTrials.gov Identifier: NCT01517893     History of Changes
Other Study ID Numbers: UM-DERM001
First Posted: January 25, 2012    Key Record Dates
Results First Posted: March 12, 2015
Last Update Posted: November 14, 2018
Last Verified: October 2018

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by John Harris, University of Massachusetts, Worcester:
vitiligo
simvastatin

Additional relevant MeSH terms:
Layout table for MeSH terms
Vitiligo
Hypopigmentation
Pigmentation Disorders
Skin Diseases
Simvastatin
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors