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Clinical Trial of Simvastatin to Treat Generalized Vitiligo

Primary Purpose

Vitiligo

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Simvastatin
Placebo
Sponsored by
John Harris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Vitiligo focused on measuring vitiligo, simvastatin

Eligibility Criteria

18 Years - 64 Years (Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • University of Massachusetts Medical School Clinical Research Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Intervention arm

Placebo Arm

Arm Description

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

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

Outcomes

Primary Outcome Measures

Number of Participants With a Decrease in Vitiligo Area Scoring Index (VASI) Score
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

Number of Participants With Increase in Investigator's Global Assessment Score
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.
Number of Participants Experiencing Toxicity From of High-dose Simvastatin .
The number of participants who experienced toxicity based upon monitored lab values (Liver Function Test) and patient symptoms for evidence of simvastatin toxicity
Change in Sentinel Patch Area
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
Change in Quality of Life Score by Using DERMATOLOGY LIFE QUALITY INDEX (DLQI)
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.
Number of Participants With an Increase in Patient's Global Assessment Score
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%
Serum CXCL10 Levels From the First and Last Available Clinic Visits Were Measured Via ELISA
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
CXCR3 Expression on CD8+ T Cells
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

Full Information

First Posted
January 13, 2012
Last Updated
October 17, 2018
Sponsor
John Harris
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1. Study Identification

Unique Protocol Identification Number
NCT01517893
Brief Title
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

2. Study Status

Record Verification Date
October 2018
Overall Recruitment Status
Completed
Study Start Date
January 2012 (undefined)
Primary Completion Date
December 2013 (Actual)
Study Completion Date
December 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
John Harris

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Vitiligo
Keywords
vitiligo, simvastatin

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Intervention arm
Arm Type
Experimental
Arm Description
Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated
Arm Title
Placebo Arm
Arm Type
Placebo Comparator
Arm Description
Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Intervention Type
Drug
Intervention Name(s)
Simvastatin
Intervention Description
Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Primary Outcome Measure Information:
Title
Number of Participants With a Decrease in Vitiligo Area Scoring Index (VASI) Score
Description
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).
Time Frame
Assessed at baseline and final study visit, 6 months after randomization
Secondary Outcome Measure Information:
Title
Number of Participants With Increase in Investigator's Global Assessment Score
Description
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.
Time Frame
Assessed at baseline and final study visit, 6 months after randomization
Title
Number of Participants Experiencing Toxicity From of High-dose Simvastatin .
Description
The number of participants who experienced toxicity based upon monitored lab values (Liver Function Test) and patient symptoms for evidence of simvastatin toxicity
Time Frame
Assessed at baseline, then monthly until final study visit, six months after randomization.
Title
Change in Sentinel Patch Area
Description
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
Time Frame
Assessed at baseline and final study visit, 6 months after randomization
Title
Change in Quality of Life Score by Using DERMATOLOGY LIFE QUALITY INDEX (DLQI)
Description
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.
Time Frame
Assessed at baseline and final study visit, 6 months after randomization
Title
Number of Participants With an Increase in Patient's Global Assessment Score
Description
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%
Time Frame
Assessed at baseline and final study visit, 6 months after randomization
Title
Serum CXCL10 Levels From the First and Last Available Clinic Visits Were Measured Via ELISA
Description
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
Time Frame
Assessed at baseline and final study visit, 6 months after randomization
Title
CXCR3 Expression on CD8+ T Cells
Description
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
Time Frame
Assessed prior to treatment and periodically while on treatment

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
John E. Harris, MD, PhD
Organizational Affiliation
University of Massachusetts, Worcester
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Massachusetts Medical School Clinical Research Center
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Clinical Trial of Simvastatin to Treat Generalized Vitiligo

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