Topical Ruxolitinib Evaluation in Immune-related Vitiligo-like Depigmentation (TRuE-VLD)

Topical Ruxolitinib for the Treatment of Immune-related Vitiligo-like Depigmentation: A Prospective, Multicentre Study of a Topical JAK-inhibitor

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Immune-related Vitiligo-like-Depigmentation
• Intervention / Treatment: Drug: Ruxolitinib Topical Cream

• Study Type: Interventional
• Enrollment (Estimated): 34
• Phase: Phase 2

Contacts and Locations

Study Locations

• Switzerland
  • Zurich, Switzerland, 8058
    • UniversitätsSpital Zürich

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

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

Exclusion Criteria:

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Topical ruxolitinib; This study will be performed as one-arm with no comparator.	Drug: Ruxolitinib Topical Cream; Ruxolitinib cream 1.5% will be applied topically twice daily over 52 weeks in patients with immune-related vitiligo-like depigmentation.; Other Names: Topical Ruxolitinib (OPZELURA)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of patients achieving a Facial vitiligo scoring index of 75% (F-VASI75) at 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.	week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of patients achieving a F-VASI of 25%, 50% and 90% (F-VASI25/50/90)	The F-VASI assesses the percentage of repigmentation on the face. Achieving an F-VASI of 50% means that a 50% improvement in facial repigmentation has been achieved. The same applies to an F-VASI of 25% or 90%; this corresponds to 25% respectively 90% repigmentation. 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.	week 24 and 52
Proportion of patients achieving a F-VASI 75 only at week 52	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. 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.	week 52
Proportion of patients achieving a Total Vitiligo Area Scoring Index of 25%, 50%, 75% and 90% (T-VASI25/50/75/90)	T-VASI is a measure used to evaluate the success of vitiligo treatment over the entire body. A T-VASI of for instance 75% means that a 75% improvement in repigmentation of the entire skin of the body has been achieved. The T-VASI is calculated by estimating and weighting the area of depigmented skin and the degree of repigmentation. Percentage of depigmentation (0% = no depigmentation present, 100% = completely depigmented skin) is multiplied by the affected body surface area in percent. The unit of "Proportion of patients achieving a Total Vitiligo Area Scoring Index of 25%, 50%, 75% and 90% (T-VASI25/50/75/90)" is a percentage. It is looked at what percentage of participants have achieved an improvement in their T-VASI score. It is looked at how many participants have achieved a 25% improvement, how many a 50% improvement, how many a 75% improvement and how many a 90% improvement in their T-VASI score.	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)	The VNS is an assessment tool developed to measure the visibility, conspicuousness and appearance of vitiligo spots. The score has a scale from one to five. One means more noticeable, five means no noticeability/visibility.	week 24 and 52
Percentage change from baseline in the Facial Body Surface Area (F-BSA) and Total Body Surface Area (T-BSA)	Calculation of the affected body surface area as a percentage. Once the affected surface on the face and once the affected surface on the entire integument.	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)	The F-PhGVA is an assessment tool used by physicians to evaluate the severity of facial vitiligo. It is based on a global assessment by the treating physician and takes into account various aspects of the disease, including the area and extent of depigmentation as well as the aesthetic impairment. The assessment is made on a numerical scale, categorizing different degrees of severity of facial vitiligo. The scale ranges from 0 to 4, with 0 corresponding to skin with no signs of vitiligo/complete repigmentation and 4 corresponding to very severe vitiligo. The T-PhGVA is a global assessment of the disease across the entire body. It has the same numerical scale as the F-PhGVA, ranging from 0 to 4, with 0 corresponding to skin with no signs of vitiligo/complete repigmentation and 4 corresponding to very severe vitiligo. Both scores are useful tools for evaluating the success of treatment for vitiligo on the face and the entire body and for documenting the progress of patients.	week 24 and 52
Change in disease stability	The change of disease stability is defined as an exclusion of progressive vitiligo over a 1-year period with no increase in size of existing vitiligo foci, no new vitiligo foci, no Koebner phenomenon	week 24 and 52
Proportion of patient reported outcome: Dermatology Life Quality Index (DLQI)	Dermatology Life Quality Index (DLQI): 10 questions, summing the score of each question resulting in a minimum of 0 and a maximum of 30 points	baseline, week 24 and 52
Proportion of patient reported outcome: Treatment Satisfaction Questionnaire for Medication (TSQM)	Treatment Satisfaction Questionnaire for Medication (TSQM): questionnaire used to measure patient satisfaction with medication. The score ranges from 0 to 100 points	baseline, week 24 and 52
Proportion of patient reported outcome: Vitiligo Noticeability Scale (VNS)	Vitiligo Noticeability Scale (VNS): score with a scale from one to five. One means more noticeable, five means no noticeability/visibility.	baseline, week 24 and 52
Proportion of patient reported outcome: Color-matching questions	Color-matching questions: The patient assesses how well the color of the skin affected by VLD matches the surrounding, unaffected skin after treatment. Five answer options with a grading between "excellent" and "very poor" are possible.	baseline, week 24 and 52
Proportion of patient reported outcome: Facial Patient Global Impression of Change-Vitiligo (F-PaGIC-V)	Facial Patient Global Impression of Change-Vitiligo (F-PaGIC-V): Patients assess the change in VLD on their face since starting treatment with the drug. Seven answer options are possible with a grading of "very much improved" and "very much worse"	baseline, week 24 and 52
Proportion of patient reported outcome: Total Body Patient Global Impression of Change-Vitiligo (T-PaGIC-V)	Total Body Patient Global Impression of Change-Vitiligo (T-PaGIC-V): Patients assess the change of VLD on their total body since starting treatment with the drug. Seven answer options are possible with a grading of "very much improved" and "very much worse"	baseline, week 24 and 52
Proportion of patient reported outcome: Five Well-Being Index (WHO-5)	Five Well-Being Index (WHO-5): a short self-reported measure of current mental wellbeing. It consists of five statements, which respondents rate according to a scale between 0 to 5	baseline, week 24 and 52
Proportion of patient reported outcome: Hospital Anxiety and Depression Scale (HADS)	Hospital Anxiety and Depression Scale (HADS): is used to measure anxiety and depression in patients with physical illnesses. The severity of anxious and depressive symptoms during the past week is recorded by means of self-assessment on two subscales with seven items each. The total sum score can be used as a measure of general psychological impairment.	baseline, week 24 and 52
Proportion of patient reported outcome: Vitiligo Quality of Life Index (VitiQoL)	Vitiligo Quality of Life Index (VitiQoL): specially developed instrument to measure the quality of life of people with vitiligo. It captures the psychosocial and emotional impact of the disease on the daily lives of those affected. As vitiligo can have not only physical but also significant psychological and social effects, the VitiQoL is a valuable tool to assess these comprehensively. Contains 16 questions.	baseline, week 24 and 52
Proportion of patient reported outcome: load thermometer	The patient must indicate on a scale from zero to ten how much the VLD is bothering them. Grading from "not at all burdensome" to "extremely burdensome".	baseline, week 24 and 52

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assessment of skin shave biopsies	Differences in the cutaneous immune microenvironment in skin shave biopsies will be investigated using multi-omics technics. Histology, immune-histology, multiplex and single-cell RNA sequencing analyses will be performed.	Baseline and week 52

Collaborators and Investigators

• Sponsor: University of Zurich

Study record dates

Study Major Dates

• Study Start (Actual): July 25, 2024
• Primary Completion (Actual): July 7, 2026
• Study Completion (Estimated): August 31, 2026

Study Registration Dates

• First Submitted: May 31, 2024
• First Submitted That Met QC Criteria: July 20, 2026
• First Posted (Actual): July 24, 2026

Study Record Updates

• Last Update Posted (Actual): July 24, 2026
• Last Update Submitted That Met QC Criteria: July 20, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: Vitiligo; White spot disease
• Additional Relevant MeSH Terms: Lichenoid Eruptions; Skin Diseases, Papulosquamous; Skin Diseases; Hypopigmentation; Pigmentation Disorders; Skin and Connective Tissue Diseases; Vitiligo; Lichen Sclerosus et Atrophicus; ruxolitinib
• Other Study ID Numbers: 2023-01552

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No