A Phase-II, Randomized, Placebo-controlled Trial of Simvastatin in Generalized Vitiligo
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Number of Participants With a Decrease in Vitiligo Area Scoring Index (VASI) Score
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 64 Years（Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
研究组 & 干预措施
Intervention arm
Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated
干预措施: Simvastatin (Drug)
Placebo Arm
Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants With a Decrease in Vitiligo Area Scoring Index (VASI) Score
时间窗: 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).
次要结局
- Change in Sentinel Patch Area(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(Assessed at baseline and final study visit, 6 months after randomization)
- Number of Participants With Increase in Investigator's Global Assessment Score(Assessed at baseline and final study visit, 6 months after randomization)
- Change in Quality of Life Score by Using DERMATOLOGY LIFE QUALITY INDEX (DLQI)(Assessed at baseline and final study visit, 6 months after randomization)
- Number of Participants With an Increase in Patient's Global Assessment Score(Assessed at baseline and final study visit, 6 months after randomization)
- Number of Participants Experiencing Toxicity From of High-dose Simvastatin .(Assessed at baseline, then monthly until final study visit, six months after randomization.)
- CXCR3 Expression on CD8+ T Cells(Assessed prior to treatment and periodically while on treatment)
研究者
John Harris
Principal Investigator
University of Massachusetts, Worcester