A Study to Evaluate the Safety and Efficacy of Ruxolitinib Cream With Phototherapy in Participants With Vitiligo

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the efficacy and safety of Ruxolitinib cream with or without phototherapy in adolescent and adult participants with non-segmental vitiligo for whom vitiligo area (facial and nonfacial) does not exceed 10% body surface area (BSA).

Official Title
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A Safety and Efficacy Study of Ruxolitinib Cream Combined With Narrow-Band Ultraviolet B Phototherapy in Participants With Vitiligo

Conditions
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Vitiligo

Intervention / Treatment
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* Drug: Ruxolitinib 1.5% cream
* Device: NB-UVB phototherapy


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: A clinical diagnosis of nonsegmental vitiligo with depigmented area including all of the following: ≥ 0.5 F-VASI on the face ≥ 3.0 T-VASI (body areas not including the face) Total body vitiligo area (facial and nonfacial) not exceeding 10% BSA. Agree to discontinue all agents used to treat vitiligo from screening through the final safety follow-up visit. Over-the-counter preparations deemed acceptable by the investigator and camouflage makeups are permitted. Exclusion Criteria: No pigmented hair within any of the vitiligo areas on the face. Other forms of vitiligo (eg, segmental) or other differential diagnosis of vitiligo or other skin depigmentation disorders. Used depigmentation treatments (eg, monobenzone) for past treatment of vitiligo or other pigmented areas. Previous adverse reaction to NB-UVB phototherapy that caused discontinuation of therapy. Lack of response (little or no repigmentation) to prior NB-UVB phototherapy. History of thromboembolic events (eg, deep vein thrombosis, pulmonary embolism, ischemic stroke, myocardial infarction). Any other skin disease that, in the opinion of the investigator, would interfere with the study cream application or study assessments. Active acute bacterial, fungal, or viral skin infection (eg, herpes simplex, herpes zoster, chicken pox) within 1 week before baseline. Any serious illness or medical, physical, or psychiatric condition(s) that pose a significant risk to the participant; or interfere with interpretation of study data. Recent use of topical or systemic medications (including biologics or JAK inhibitors), or laser or phototherapy to treat vitiligo. Note: Recent may be defined differently for different treatments. Specific protocol-defined chemistry, hematology, and serological lab values. Those who are pregnant, lactating or considering pregnancy during the period of study participation.

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 99 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Group A: Ruxolitinib + Narrow-Band Ultraviolet B Phototherapy (NB-UVB)<br>Participants will initially apply ruxolitinib 1.5%mg cream as a monotherapy. At week 12, those who have < 25% improvement in total body Vitiligo Area Scoring Index (T-VASI25) will have NB-UVB phototherapy added to their ruxolitinib 1.5% cream BID regimen. NB-UVB will be given 3 times per week starting at Week 12 through Week 48 (36 weeks). For participants who receive combination therapy, NB-UVB machines will be supplied by the sponsor for at home use during the study. | Drug: Ruxolitinib 1.5% cream<br>* Ruxolitinib cream 1.5% applied twice a day (BID).<br>Device: NB-UVB phototherapy<br>* NB-UVB (311-312 nm) phototherapy is an established treatment modality for vitiligo. Starting dose will be 200 mJ/cm2 and dose may be increased by 10% at each visit<br>* Other names: Narrow-Band Ultraviolet B Phototherapy;|
| Experimental: Group B: Ruxolitinib Monotherapy<br>Participants will apply ruxolitinib 1.5% cream BID as monotherapy. Participants who have ≥ T-VASI25 at Week 12 will continue on ruxolitinib 1.5% cream BID alone. | Drug: Ruxolitinib 1.5% cream<br>* Ruxolitinib cream 1.5% applied twice a day (BID).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline in T-VASI | T-VASI is calculated using a formula that includes contributions from all body regions (possible range, 0-100). The body is divided into 6 separate and mutually exclusive sites (head/neck, hands, upper extremities [excluding hands], trunk, lower extremities [excluding feet], and feet), with percentage of vitiligo involvement estimated in hand units by the same investigator throughout the study. | Week 48 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of treatment-related adverse events | Defined as any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug. | Approximately 14 months | 
| Percentage of participants achieving F-VASI50/75/90 at each post-baseline visit | Defined as ≥ 50/75/90% improvement from baseline in Face Vitiligo Area Scoring Index (F-VASI) score. Facial VASI is measured by percentage of vitiligo involvement (%BSA) and the degree of depigmentation. | Weeks 4, 8, 12, 16, 24, 32, 40 and 48 | 
| Percentage of participants achieving T-VASI50/75/90 at each post-baseline visit | Defined as ≥ 50/75/90% improvement from baseline in Total Body Vitiligo Area Scoring Index (T-VASI) score. T-VASI is calculated using a formula that includes contributions from all body regions (possible range, 0-100). The body is divided into 6 separate and mutually exclusive sites (head/neck, hands, upper extremities [excluding hands], trunk, lower extremities [excluding feet], and feet), with percentage of vitiligo involvement estimated in hand units by the same investigator throughout the study. | Weeks 4, 8, 12, 16, 24, 32, 40 and 48 | 
| Percentage change from baseline in F-VASI at each post-baseline visit | Face Vitiligo Area Scoring Index (F-VASI) score is measured by percentage of vitiligo involvement (%BSA) and the degree of depigmentation. | Weeks 4, 8, 12, 16, 24, 32, 40 and 48 | 
| Percentage change from baseline in T-VASI at each post-baseline visit | T-VASI is calculated using a formula that includes contributions from all body regions (possible range, 0-100). The body is divided into 6 separate and mutually exclusive sites (head/neck, hands, upper extremities [excluding hands], trunk, lower extremities [excluding feet], and feet), with percentage of vitiligo involvement estimated in hand units by the same investigator throughout the study. | Weeks 4, 8, 12, 16, 24, 32, 40 and 48 | 
| Percentage change from baseline in F-BSA at each post-baseline visit | Facial BSA (F-BSA) takes into account the facial depigmented areas as a percentage of the total body area. | Weeks 4, 8, 12, 16, 24, 32, 40 and 48 | 
| Percentage change from baseline in T-BSA at each post-baseline visit | Total BSA (T-BSA) takes into account the depigmented areas for each of the following body regions: head/neck (including scalp), upper extremities (including axillae), hands, trunk (including genitalia), lower extremities (including buttocks), and feet. Body surface area assessment will be performed by the Palmar Method. | Weeks 4, 8, 12, 16, 24, 32, 40 and 48 | 
| Population-based (trough) plasma concentrations of ruxolitinib | Trough is defined as the concentration reached by a drug immediately before the next dose is administered. | Weeks 4, 12, and 16 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Non-segmental, Vitiligo, hypopigmentation, Pigment Disorders, Skin Diseases, JAK inhibitor