VitiligoPhase 2NCT01517893
Clinical Trial of Simvastatin to Treat Generalized Vitiligo
A Phase 2 study testing Simvastatin in people with Vitiligo, run by John Harris at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 64 years, men only
- Conditions the study is for
- Vitiligo
- What is being tested?
- Simvastatin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2012to Dec 2014
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 64 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 17Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Massachusetts Medical School Clinical Research Center
Worcester, Massachusetts, 01655
About this study
Official title: A Phase-II, Randomized, Placebo-controlled Trial of Simvastatin in Generalized Vitiligo
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.
Read the 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.
- Conditions
- Vitiligo
- Being tested
- Simvastatin, Placebo
- Keywords
- vitiligo, simvastatin
- Medical subject headings
- Vitiligo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SimvastatinDrug
Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
PlaceboDrug
Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
| Study group | Role | What they receive |
|---|---|---|
| Intervention arm | Experimental | Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Receives: Drug: Simvastatin |
| Placebo Arm | Placebo comparator | Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Assessed at baseline and final study visit, 6 months after randomization
Secondary outcomes (7)
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.
Measured: 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
Measured: 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
Measured: 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.
Measured: 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%
Measured: 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
Measured: 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
Measured: Assessed prior to treatment and periodically while on treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2012 (estimated)
- Study startJan 2012
- Primary completionDec 2013
- Study completionDec 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for VitiligoStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- John Harris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, John Harris, University of Massachusetts, Worcester
- Investigators
- John E. Harris, MD, PhD, Principal investigator, University of Massachusetts, Worcester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01517893
- Sponsor's protocol ID
- UM-DERM001
- Status last verified
- Oct 2018
- First submitted
- 13 Jan 2012
- Last updated
- 14 Nov 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase-II, Randomized, Placebo-controlled Trial of Simvastatin in Generalized Vitiligo
- ClinicalTrials.gov ID
- NCT01517893
- Acronym / sponsor ID
- UM-DERM001
- Conditions
- Vitiligo
- Keywords
- vitiligo, simvastatin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- John HarrisUniversity, hospital or other
- Study start
- Jan 2012
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 25 Jan 2012 (estimated)
- Results
- Posted 12 Mar 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intervention arm | Experimental | Drug: SimvastatinSig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated |
| Placebo Arm | Placebo comparator | Drug: PlaceboSig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated |
Who may qualify
- Ages
- 18 to 64 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (17 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SimvastatinSig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated | Drug | Intervention arm |
| PlaceboSig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated | Drug | Placebo Arm |
Primary outcome
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).
7 secondary outcomes
Number of Participants With Increase in Investigator's Global Assessment Score
Time frame: Assessed at baseline and final study visit, 6 months after randomization
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.
Change in 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)
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
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
Time frame: Assessed at baseline and final study visit, 6 months after randomization
CXCR3 Expression on CD8+ T Cells
Time frame: Assessed prior to treatment and periodically while on treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Nov 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John E. Harris, MD, PhD principal investigator
University of Massachusetts, Worcester
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Simvastatin in people with Vitiligo, run by John Harris at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Nov 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Mar 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Nov 2018.