Open Label Phase 2 Proof-of-concept Pilot Trial of Topical Ruxolitinib in Repigmenting Adult Patients With Vitiligo
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- Percent Change in Vitiligo Area Severity Index (VASI) Score From Baseline to Week 20
研究概览
简要总结
The purpose of this study is to determine if topical ruxolitinib 1.5% will provide repigmentation in vitiligo lesions.
详细描述
The hypothesis is that JAK inhibitors can also successfully treat vitiligo. Lesional skin of both alopecia areata and vitiligo primarily contain T cells in a TH1 response as opposed to a mixed cell infiltrate such as in psoriasis or lichen planus. Both alopecia areata and vitiligo are TH1 mediated diseases dependent on the production of IFN-gamma to drive the response. CD8+ T cells are both necessary and sufficient for melanocyte destruction in vitiligo (van den Boorn JG et al 2009) and CD8+NKG2D+ T cells are also necessary and sufficient for hair loss in alopecia areata (Gilhar A et al 2013).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of vitiligo.
- •At Visit 1 (Baseline/Day 1), have had vitiligo covering at least 1% of total body surface area (BSA) on the scalp, trunk or limbs (excluding nails).
- •Female subjects of childbearing potential must agree to use a highly effective method of contraception throughout the study and for at least four weeks after the last dose of assigned treatment. Male subjects must also use contraception, such as barrier method with spermicide.
- •If receiving concomitant medications for any reason, must be on a stable regimen and willing to stay on a stable regimen.
- •Must be willing to washout of other vitiligo treatments. All treatments for vitiligo are prohibited during the course of the study.
排除标准
- •Other skin conditions at Baseline that would interfere with evaluation of vitiligo.
- •Pregnant/breastfeeding females, or females of childbearing potential not using highly effective contraception. Women of childbearing potential must test negative for pregnancy and use contraception for at least four weeks after last dose of drug.
- •Current or recent history of clinically significant medical/psychiatric condition or laboratory abnormality that may increase risk associated with the study participation or drug administration.
- •Have a history of any lymphoproliferative disorder, lymphoma, leukemia, history of disseminated herpes zoster or disseminated herpes simplex, or a recurrent localized, dermatomal herpes zoster.
- •Have a history of infection requiring parenteral or oral or topical antimicrobial therapy within 2 weeks prior to Baseline.
- •Vaccinated with live/attenuated live vaccine within 6 weeks prior to Baseline.
- •Previously participated in study of oral/topical ruxolitinib or tofacitinib (tofacitinib, CP-690,550, formerly tasocitinib) unless confirmed to have been randomized to and treated with placebo or placebo topical formulation (vehicle) only.
- •Received a prohibited concomitant medication within 7 days or 5 half-lives (whichever is longer) prior to Baseline.
- •Have participated in other studies within 4 weeks or 5 half-lives (whichever is longer) prior to Visit 1 (Baseline/Day 1). Subjects cannot participate in studies of other investigational or experimental therapies or procedures at any time during their participation in this study.
- •Subjects who are investigational site staff members or relatives of those site staff members or subjects who are Sponsor employees directly involved in the conduct of the trial.
- •In the opinion of the investigator or Sponsor, the subject is inappropriate for entry into this study, or unwilling/unable to comply with study procedures and lifestyle guidelines.
- •Screening laboratory abnormalities
- •CYP Inhibitor Exclusion: Subjects taking potent CYP3A4 inhibitors or fluconazole within 2 weeks or 5 half-lives, whichever is longer, before the baseline visit.
研究组 & 干预措施
Ruxolitinib 1.5% phosphate cream
Ruxolitinib 1.5% phosphate cream twice daily to vitiligo patches.
干预措施: Ruxolitinib 1.5% Phosphate Cream (Drug)
结局指标
主要结局
Percent Change in Vitiligo Area Severity Index (VASI) Score From Baseline to Week 20
时间窗: Baseline to Week 20
The VASI for each body region (hands, upper extremities, trunk, lower extremities, feet) is determined by the product of the area involved in hand prints and the extent of depigmentation within each hand print unit measured patch (0-100). Area involved is measured by hand prints (1 hand print = 1%) with a possible range of 0-100. Degree of depigmentation is measured as: 1.00 (100%) = complete depigmentation, no pigment present, 0.90(90%)=specks of pigment present, 0.75(75%)=depigmented area exceeds the pigmented area, 0.50(50%)=pigmented and depigmented areas are equal, 0.25(25%)=pigmented area exceeds depigmented area, 0.10(10%)=only specks of depigmentation present, and 0.0(0%)=no depigmentation present.
次要结局
- Mean Dermatology Life Quality Index (DLQI) Scores(Baseline and Week 20)
- Percent Change in Body Surface Area (BSA) of Repigmentation(Baseline and Week 20)
- Percent Change in Vitiligo European Task Force (VETF) Assessment - Disease Staging(Baseline and Week 20)
- Number of Subjects Who Achieve a Physician Global Vitiligo Assessment (PGVA) of Clear or Almost Clear(Baseline and Week 20)
- Percent Change in Vitiligo European Task Force (VETF) Assessment - Body Surface Area(Baseline and Week 20)
- Percent Change in Vitiligo European Task Force (VETF) Assessment - Disease Progression(Baseline and Week 20)