Topical Ruxolitinib for the Treatment of Immune-related Vitiligo-like Depigmentation: A Prospective, Multicentre Study of a Topical JAK-inhibitor
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 34
- 试验地点
- 1
- 主要终点
- Proportion of patients achieving a Facial vitiligo scoring index of 75% (F-VASI75) at week 24
研究概览
简要总结
White spot disease is a common side effect on the skin in the context of cancer treatments with immunotherapies. There is no standard therapy yet for white spot caused by cancer therapies. There is already research in humans on the treatment of white spot disease. Previous human studies with ruxolitinib cream have shown that approximately 30% of patients with white spot disease have achieved a 75% improvement (re-pigmentation) of their affected areas after 24 weeks and approximately 50% of patients have achieved a 75% improvement after 52 weeks. It is also know that a 50% improvement was observed in over 50% of patients after 24 weeks and a 90% improvement in over 15% of patients. However, these studies to date have not included patients with white spot disease caused by cancer therapy. Therefore, we do not know how well the cream works in this situation. In this study, the investigators are therefore examining whether the investigational substance ruxolitinib is effective and well tolerated in white spot disease caused by cancer therapy. The test substance has not yet been approved in Switzerland. Only once the efficacy of the investigational substance ruxolitinib has been scientifically investigated and proven can it be approved in Switzerland and used to treat white spot disease caused by cancer therapy.
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
- •Adult patients eligible for this inclusion in this study have to fulfil all the following criteria:
- •Age 18 years
- •Written informed consent of the patient
- •Sufficient fluency in German language skills to complete all questionnaires of the study without external assistance
- •Treatment-related, immune-related vitiligo-like depigmentation of at least 5% body surface area of one region (face or one upper or lower extremity or front or back side of the trunk)
- •Female participants must use effective contraceptives, either: An ovulation-inhibiting method (e.g., pill, injectable, implant, patch, or vaginal ring) or an intrauterine device (IUD). Contraception should extend one week post-study. If pregnancy occurs during or within one week after the study, participants should notify the overseeing physician immediately.
- •During participation in the study and up to one week afterwards, male participants may only have sexual intercourse with condoms. Female partners of male participants must use a highly effective contraceptive method during the partner's participation in the study and for up to 1 week afterwards
排除标准
- •Patients fulfilling any of the following criteria are not eligible for the inclusion in this study:
- •Acute psychiatric illness (except mild to moderate depression)
- •Contraindications to ruxolitinib, e.g. known hypersensitivity or allergy
- •Use of topical glucocorticosteroids, topical calcineurin inhibitors, UV light therapy, therapeutic biologics, other JAK inhibitors or potent immunosuppressants such as azathioprine or cyclosporine are not allowed during the study period (no wash out period required).
- •Women who are pregnant or breast feeding
- •Intention to become pregnant during the course of the study
- •Known or suspected non-compliance, drug or alcohol abuse.
- •Patients with an active, serious infection, including localized infections.
- •Inability to follow the procedures of the study due to language problems, psychological disorders, dementia of the participant.
- •Participation in another trial with topical or oral ruxolitinib within the 30 days preceding and during the present study.
- •Simultaneously participation in clinical trials with intervention with a drug other than ruxolitinib (except registry studies)
- •Previous enrolment into the current study.
- •Enrolment of the investigator, his/her family members, employees and other dependent persons.
结局指标
主要结局
Proportion of patients achieving a Facial vitiligo scoring index of 75% (F-VASI75) at week 24
时间窗: week 24
The F-VASI is a measure for assessing the success of treatment. The F-VASI assesses the percentage of repigmentation on the face. Achieving an F-VASI of 75% means that a 75% improvement in facial repigmentation has been achieved. This is determined by visually assessing the percentage of skin area that has returned to its normal color as a result of the treatment. The F-VASI is made up of the degree of depigmentation in percent and the size of the affected skin surface in percent. Percentage of depigmentation (0% = no depigmentation present, 100% = completely depigmented skin) is multiplied by the size of the affected skin area on the face in percent.
次要结局
- Proportion of patients achieving a F-VASI of 25%, 50% and 90% (F-VASI25/50/90)(week 24 and 52)
- Proportion of patients achieving a F-VASI 75 only at week 52(week 52)
- Proportion of patients achieving a Total Vitiligo Area Scoring Index of 25%, 50%, 75% and 90% (T-VASI25/50/75/90)(week 24 and 52)
- Proportion of patients achieving 4 points ("a lot less noticeable") or 5 points ("no longer noticeable") in the Vitiligo Noticeability Scale (VNS)(week 24 and 52)
- Percentage change from baseline in the Facial Body Surface Area (F-BSA) and Total Body Surface Area (T-BSA)(week 24 and 52)
- Percentage change from baseline in the Facial Physician's Global Vitiligo Assessment (F-PhGVA) and Total Physician's Global Vitiligo Assessment (T-PhGVA)(week 24 and 52)
- Change in disease stability(week 24 and 52)
- Proportion of patient reported outcome: Dermatology Life Quality Index (DLQI)(baseline, week 24 and 52)
- Proportion of patient reported outcome: Treatment Satisfaction Questionnaire for Medication (TSQM)(baseline, week 24 and 52)
- Proportion of patient reported outcome: Vitiligo Noticeability Scale (VNS)(baseline, week 24 and 52)
- Proportion of patient reported outcome: Color-matching questions(baseline, week 24 and 52)
- Proportion of patient reported outcome: Facial Patient Global Impression of Change-Vitiligo (F-PaGIC-V)(baseline, week 24 and 52)
- Proportion of patient reported outcome: Total Body Patient Global Impression of Change-Vitiligo (T-PaGIC-V)(baseline, week 24 and 52)
- Proportion of patient reported outcome: Five Well-Being Index (WHO-5)(baseline, week 24 and 52)
- Proportion of patient reported outcome: Hospital Anxiety and Depression Scale (HADS)(baseline, week 24 and 52)
- Proportion of patient reported outcome: Vitiligo Quality of Life Index (VitiQoL)(baseline, week 24 and 52)
- Proportion of patient reported outcome: load thermometer(baseline, week 24 and 52)
研究者
Lara Maul-Duwendag
Senior physician
University of Zurich