A Phase 2 interventional study of Simvastatin and Placebo in Vitiligo, sponsored by John Harris. Completed at 1 site in United States. Open to male participants aged 18 Years to 64 Years. Per ClinicalTrials.gov, last updated 2018-11-14.
Sponsored by John Harris · Phase 2, Interventional, and Treatment
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.
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.
298 studies on the registry are indexed under Vitiligo; 69 are open to participants now.
This study's enrollment of 15 is below the median of 30 across 227 interventional studies indexed under Vitiligo.
Browse Vitiligo studies →This is the only study on the registry with John Harris as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
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
Drug: Placebo
Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
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).
Time frame: Assessed at baseline and final study visit, 6 months after randomization
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.
Time frame: Assessed at baseline and final study visit, 6 months after randomization
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
Time frame: Assessed at baseline, then monthly until final study visit, six months after randomization.
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
Time frame: Assessed at baseline and final study visit, 6 months after randomization
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.
Time frame: Assessed at baseline and final study visit, 6 months after randomization
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%
Time frame: Assessed at baseline and final study visit, 6 months after randomization
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
Time frame: Assessed at baseline and final study visit, 6 months after randomization
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
Time frame: Assessed prior to treatment and periodically while on treatment
| Milestone | Intervention Arm | Placebo Arm |
|---|---|---|
| Started | 8 | 7 |
| Completed | 5 | 7 |
| Not completed | 3 | 0 |
| Withdrew: Withdrawal by subject | 3 | 0 |
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).
| Participants | Intervention Arm | Placebo Arm |
|---|---|---|
| Number of Participants With a Decrease in Vitiligo Area Scoring Index (VASI) Score | 0 | 0 |
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.
| Participants | Intervention Arm | Placebo Arm |
|---|---|---|
| Number of Participants With Increase in Investigator's Global Assessment Score | 1 | 0 |
The number of participants who experienced toxicity based upon monitored lab values (Liver Function Test) and patient symptoms for evidence of simvastatin toxicity
| Participants | Intervention Arm | Placebo Arm |
|---|---|---|
| Number of Participants Experiencing Toxicity From of High-dose Simvastatin . | 0 | 0 |
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
| cm2 | Intervention Arm | Placebo Arm |
|---|---|---|
| Change in Sentinel Patch Area | -0.2272 ± 3.463 | 3.8571 ± 9.17 |
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.
| units on a scale | Intervention Arm | Placebo Arm |
|---|---|---|
| Change in Quality of Life Score by Using DERMATOLOGY LIFE QUALITY INDEX (DLQI) | 3.4 ± 4.159326869 | 2.285714286 ± 2.690370837 |
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%
| Participants | Intervention Arm | Placebo Arm |
|---|---|---|
| Number of Participants With an Increase in Patient's Global Assessment Score | 0 | 1 |
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
| Fold change of baseline CXCL10 level | Intervention Arm | Placebo Arm |
|---|---|---|
| Serum CXCL10 Levels From the First and Last Available Clinic Visits Were Measured Via ELISA | 0.9148 ± 0.263 | 0.6176 ± 0.1685 |
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
No measurements were reported for this outcome.
Collected over 6 months (baseline to end of treatment). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Intervention Arm | — | 1/8 (12.5%) | 4/8 (50%) |
| Placebo Arm | — | 0/7 (0%) | 4/7 (57.1%) |
| Event | Intervention Arm | Placebo Arm |
|---|---|---|
| BurnSkin and subcutaneous tissue disorders | 1/8 | 0/7 |
| Event | Intervention Arm | Placebo Arm |
|---|---|---|
| diarrheaGastrointestinal disorders | 1/8 | 2/7 |
| URI symptomsRespiratory, thoracic and mediastinal disorders | 1/8 | 2/7 |
| myalgiaMusculoskeletal and connective tissue disorders | 2/8 | 0/7 |
| arthrlagiaMusculoskeletal and connective tissue disorders | 2/8 | 0/7 |
| back painMusculoskeletal and connective tissue disorders | 0/8 | 1/7 |
| leg twingeMusculoskeletal and connective tissue disorders | 0/8 | 1/7 |
| abdominal painGastrointestinal disorders | 0/8 | 1/7 |
| tinnitusEar and labyrinth disorders | 0/8 | 1/7 |
| flatulenceGastrointestinal disorders | 0/8 | 1/7 |
| stool odorGastrointestinal disorders | 0/8 | 1/7 |
| Age, Continuous(years) | Intervention Arm | Placebo Arm | Total |
|---|---|---|---|
| Mean | 43.9 (28 to 55) | 39 (32 to 49) | 41.6 (28 to 55) |
| Sex: Female, Male(Participants) | Intervention Arm | Placebo Arm | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 8 | 7 | 15 |
| Vitiligo area scoring index(percentage) | Intervention Arm | Placebo Arm | Total |
|---|---|---|---|
| Mean | 14.81 (3.23 to 40.93) | 23.09 (5.12 to 46.26) | 19.23 (3.23 to 46.26) |
This study is completed, as verified in Oct 2018. You cannot join it, but the record below documents what was studied.
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