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Clinical Trial of Simvastatin to Treat Generalized Vitiligo

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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

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Vitiligo
Interventions Drug: Simvastatin
Drug: Placebo
Enrollment 15

Recruitment Details  
Pre-assignment Details  
Arm/Group Title Intervention Arm Placebo Arm
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Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated

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

Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Period Title: Overall Study
Started 8 7
Completed 5 7
Not Completed 3 0
Reason Not Completed
Withdrawal by Subject             3             0
Arm/Group Title Intervention Arm Placebo Arm Total
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Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated

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

Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated Total of all reporting groups
Overall Number of Baseline Participants 8 7 15
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 8 participants 7 participants 15 participants
43.9
(28 to 55)
39
(32 to 49)
41.6
(28 to 55)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 7 participants 15 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
Male
8
 100.0%
7
 100.0%
15
 100.0%
Vitiligo area scoring index   [1] 
Mean (Full Range)
Unit of measure:  Percentage
Number Analyzed 8 participants 7 participants 15 participants
14.81
(3.23 to 40.93)
23.09
(5.12 to 46.26)
19.23
(3.23 to 46.26)
[1]
Measure Description: The vitiligo area scoring index (VASI) is a measure of the surface area affected by vitiligo in a given research subject. It ranges from 0% (no vitiligo) to 100% (complete depigmentation of the entire body surface area). In terms of treatment, a decrease in VASI is desirable as it indicates less surface area affected by vitiligo. The score represents an estimate made by the investigators, based on the approximation that one "hand unit," is roughly equivalent to 1% of the body surface area.
1.Primary Outcome
Title Number of Participants With a Decrease in Vitiligo Area Scoring Index (VASI) Score
Hide 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Overall number of participants for intervention (5) differs from enrollment (8) due to withdraw of 3 participants on intervention arm.
Arm/Group Title Intervention Arm Placebo Arm
Hide Arm/Group Description:

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

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

Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Overall Number of Participants Analyzed 5 7
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
2.Secondary Outcome
Title Number of Participants With Increase in Investigator's Global Assessment Score
Hide 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
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Hide Analysis Population Description
For placebo arm, only 6 out of 7 participants reported data.
Arm/Group Title Intervention Arm Placebo Arm
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Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated

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

Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Overall Number of Participants Analyzed 5 6
Measure Type: Count of Participants
Unit of Measure: Participants
1
  20.0%
0
   0.0%
3.Secondary Outcome
Title Number of Participants Experiencing Toxicity From of High-dose Simvastatin .
Hide 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.
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention Arm Placebo Arm
Hide Arm/Group Description:

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

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

Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Overall Number of Participants Analyzed 5 7
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
4.Secondary Outcome
Title Change in Sentinel Patch Area
Hide 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
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention Arm Placebo Arm
Hide Arm/Group Description:

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

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

Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Overall Number of Participants Analyzed 5 7
Mean (Standard Deviation)
Unit of Measure: cm2
-0.2272  (3.463) 3.8571  (9.17)
5.Secondary Outcome
Title Change in Quality of Life Score by Using DERMATOLOGY LIFE QUALITY INDEX (DLQI)
Hide 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
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention Arm Placebo Arm
Hide Arm/Group Description:

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

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

Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Overall Number of Participants Analyzed 5 7
Mean (Standard Deviation)
Unit of Measure: units on a scale
3.4  (4.159326869) 2.285714286  (2.690370837)
6.Secondary Outcome
Title Number of Participants With an Increase in Patient's Global Assessment Score
Hide 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
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Hide Analysis Population Description
Placebo arm reports 6 instead of enrolled 7 due to failure of participant to complete assessment.
Arm/Group Title Intervention Arm Placebo Arm
Hide Arm/Group Description:

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

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

Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Overall Number of Participants Analyzed 5 6
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
1
  16.7%
7.Secondary Outcome
Title Serum CXCL10 Levels From the First and Last Available Clinic Visits Were Measured Via ELISA
Hide 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
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Hide Analysis Population Description
Serum CXCL10 levels from the first and last available clinic visits were measured via ELISA. The mean and SEM are reported here. One participant in intervention group was not included because participant withdrew before a second CXCL10 level was obtained. Other withdrawn participants were included, using the CXCL10 from their final visits.
Arm/Group Title Intervention Arm Placebo Arm
Hide Arm/Group Description:

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

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

Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Overall Number of Participants Analyzed 7 7
Mean (Standard Error)
Unit of Measure: Fold change of baseline CXCL10 level
0.9148  (0.263) 0.6176  (0.1685)
8.Secondary Outcome
Title CXCR3 Expression on CD8+ T Cells
Hide 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
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Hide Analysis Population Description
No data collected as this outcome was abandoned due to budget constraints and strength of other study results.
Arm/Group Title Intervention Arm Placebo Arm
Hide Arm/Group Description:

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

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

Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 6 months (baseline to end of treatment)
Adverse Event Reporting Description Safety lab values and questionnaires collected for outcomes.
 
Arm/Group Title Intervention Arm Placebo Arm
Hide Arm/Group Description

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

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

Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
All-Cause Mortality
Intervention Arm Placebo Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Intervention Arm Placebo Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   1/8 (12.50%)   0/7 (0.00%) 
Skin and subcutaneous tissue disorders     
Burn  [1]  1/8 (12.50%)  0/7 (0.00%) 
Indicates events were collected by systematic assessment
[1]
Sustained at work, unrelated
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Intervention Arm Placebo Arm
Affected / at Risk (%) Affected / at Risk (%)
Total   4/8 (50.00%)   4/7 (57.14%) 
Ear and labyrinth disorders     
tinnitus   0/8 (0.00%)  1/7 (14.29%) 
Gastrointestinal disorders     
diarrhea   1/8 (12.50%)  2/7 (28.57%) 
abdominal pain   0/8 (0.00%)  1/7 (14.29%) 
flatulence   0/8 (0.00%)  1/7 (14.29%) 
stool odor   0/8 (0.00%)  1/7 (14.29%) 
Infections and infestations     
dental infection   1/8 (12.50%)  0/7 (0.00%) 
Musculoskeletal and connective tissue disorders     
back pain   0/8 (0.00%)  1/7 (14.29%) 
leg twinge   0/8 (0.00%)  1/7 (14.29%) 
pulled muscle   0/8 (0.00%)  1/7 (14.29%) 
neck pain   1/8 (12.50%)  0/7 (0.00%) 
myalgia   2/8 (25.00%)  0/7 (0.00%) 
arthrlagia   2/8 (25.00%)  0/7 (0.00%) 
Nervous system disorders     
sleep disturbance   1/8 (12.50%)  0/7 (0.00%) 
Renal and urinary disorders     
urine color change   1/8 (12.50%)  0/7 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
URI symptoms   1/8 (12.50%)  2/7 (28.57%) 
cough   0/8 (0.00%)  1/7 (14.29%) 
Skin and subcutaneous tissue disorders     
xerosis   0/8 (0.00%)  1/7 (14.29%) 
Indicates events were collected by systematic assessment
The following outcome measures were abandoned: 1.Decrease in CXCR3 exp. on CD8 positive T-Cells, 2.Corr. Among Var. Outcome Measures for Vitiligo: procedures not performed/no data collected due to strength of prior outcomes & budget limitation.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: John E. Harris, MD, PhD
Organization: University of Massachusetts Medical School
Phone: 508-856-1982
Responsible Party: John Harris, University of Massachusetts, Worcester
ClinicalTrials.gov Identifier: NCT01517893     History of Changes
Other Study ID Numbers: UM-DERM001
First Submitted: January 13, 2012
First Posted: January 25, 2012
Results First Submitted: February 19, 2015
Results First Posted: March 12, 2015
Last Update Posted: November 14, 2018