A Study to Assess the Safety and Efficacy of Ruxolitinib Cream in Participants With Genital Vitiligo

An Open-Label, Phase 2, Safety, and Efficacy Study of Ruxolitinib Cream in Participants With Genital Vitiligo

An open-label study in which participants with non-segmental vitiligo with genital involvement will apply ruxolitinib 1.5% cream twice a day (BID) to all depigmented areas (up to 10% BSA) for up to 48 weeks. Participants should continue to treat depigmented areas identified for treatment at baseline regardless of whether the area begins to improve or fully repigment.

Study Overview

• Status: Completed
• Conditions: Nonsegmental Vitiligo With Genital Involvement
• Intervention / Treatment: Drug: Ruxolitinib Cream

• Study Type: Interventional
• Enrollment (Actual): 49
• Phase: Phase 2

Expanded Access

Approved for sale to the public. See expanded access record.

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2J 7E1
      • Dermatology Research Institute
  • Ontario
    • Barrie, Ontario, Canada, L4M 7G1
      • Simcomed Health Ltd
    • Peterborough, Ontario, Canada, K9J 5K2
      • Skin Centre for Dermatology
    • Toronto, Ontario, Canada, M3H 5Y8
      • Research Toronto
• France
  • Bordeaux, France, 33075
    • Centre hospitalier universitaire de Bordeaux
  • Créteil, France, 94000
    • Hopital Henri Mondor Service de Dermatologie
  • Nice, France, 06202
    • Chu Nice Hopital Archet 2
• United States
  • California
    • Fremont, California, United States, 94538
      • Center for Dermatology Clinical Research, Inc
    • Los Angeles, California, United States, 90036
      • Vitiligo & Pigmentation Institute of Southern California
  • Michigan
    • Fort Gratiot, Michigan, United States, 48059-3526
      • Hamzavi Dermatology
  • Ohio
    • Mayfield, Ohio, United States, 44124
      • Apex Clinical Research Center
  • Texas
    • Pflugerville, Texas, United States, 78660
      • Austin Institute For Clinical Research Aicr Pflugerville
    • Plano, Texas, United States, 75024-4319
      • Innovative Dermatology: Legacy Medical Village

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Clinical diagnosis of nonsegmental vitiligo with genital involvement
• At least ≥ 0.25% BSA of nonsegmental vitiligo in the genital area.
• Pigmented hair within some of the genital vitiligo areas.
• At least 1 genital target lesion that is ≥ 0.1% BSA that has a pigmented hair within it.
• Vitiligo on areas of the body besides the genitals.
• Total body vitiligo area not exceeding 10% BSA.
• Willing to have genital photography conducted.
• Must 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:

• Other forms of vitiligo (eg, segmental) or other differential diagnosis of vitiligo or other skin depigmentation disorders.
• Prior or current use of depigmentation treatments (eg, monobenzone).
• Active or recurrent genital warts or herpes.
• Male participants with partners with known current/active cervical intraepithelial neoplasia or anal intraepithelial neoplasia.
• An active sexually transmitted disease, sexually transmitted infection, or other skin disorder affecting the genital area (eg, scabies, fungal infection, molluscum).
• Had ≥ 3 laser hair removal treatments in an area to be treated for vitiligo.
• Any serious illness or medical, physical, or psychiatric condition(s) that, in the investigator's opinion, would interfere with full participation in the study or interpretation of study data.
• Clinical laboratory test results outside of protocol defined ranges
• Pregnant or lactating participants, or those considering pregnancy during the period of their study participation.

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: Ruxolitinib Cream; Participants with non-segmental vitiligo with genital involvement will receive ruxolitinib 1.5% cream BID for up to 48 weeks	Drug: Ruxolitinib Cream; Ruxolitinib cream will be applied twice daily for upto 48 weeks; Other Names: Opzelura

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Achieving a Genital Vitiligo Noticeability Scale (VNS) of "4 - A Lot Less Noticeable" or "5 - No Longer Noticeable" at Week 48	The VNS is a patient-reported measure of vitiligo treatment success that has a 5-point scale. The participant was asked to respond to the following query: "Compared with before treatment, how noticeable is the vitiligo now? Responses: (1) more noticeable, (2) as noticeable, (3) slightly less noticeable, (4) a lot less noticeable, or (5) no longer noticeable. The baseline genital photograph was shown to the participant for reference. A VNS score of 4 or 5 can be interpreted as representing treatment success.	Baseline; Week 48

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)	An adverse event (AE) is any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered drug related. An AE can therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study cream. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug and within 30 days of the last administration of study drug.	up to approximately 14 months
Percentage of Participants Achieving Genital-Physician Global Vitiligo Assessment (PhGVA) of 0 or 1 at Week 48	The severity of genital vitiligo was assessed by the physician using the PhGVA, which has a 5-point scale: 0=clear, no signs of vitiligo or complete/near complete repigmentation; 1=almost clear, mostly pigmented areas with small depigmented or difficult to repigment areas (e.g., shaft of penis or labia minora); 2=mild disease, modest areas of depigmentation with approximately 50% pigmentation within vitiligo areas or significant perifollicular pattern present; 3=moderate disease, large areas of depigmented vitiligo areas/significant depigmentation within vitiligo areas; 4=severe disease, extensive areas of vitiligo with complete depigmentation. Response was reported for the genital region (Genital-PhGVA).	Week 48
Change From Baseline in Affected Body Surface Area (BSA) in the Genital Region at Weeks 24 and 48	BSA assessments were performed using the palmar method and was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Total BSA takes into account the depigmented areas for each of the following body regions: head/neck (including face and scalp), upper extremities (including axillae), hands, trunk (including genitalia), lower extremities (including buttocks), and feet. The BSA depigmented by vitiligo was assessed for the genitals (which represents approximately 1% of the trunk). Genital BSA takes into account the depigmented areas of the labia majora, labia minora, and perineum in females and the penis, scrotum, and perineum in males. Change from baseline was calculated as the post-baseline value minus the baseline value.	Baseline; Weeks 24 and 48
Percentage of Participants Achieving ≥50% Improvement in Total Body Vitiligo Area Scoring Index (T-VASI50) at Weeks 24 and 48	A T-VASI50 responder achieved ≥50% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement).	Baseline; Weeks 24 and 48
Percentage of Participants Achieving ≥75% Improvement in Total Body Vitiligo Area Scoring Index (T-VASI75) at Weeks 24 and 48	A T-VASI75 responder achieved ≥75% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement).	Baseline; Weeks 24 and 48
Percentage of Participants Achieving ≥90% Improvement in Total Body Vitiligo Area Scoring Index (T-VASI90) at Weeks 24 and 48	A T-VASI90 responder achieved ≥90% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement).	Baseline; Weeks 24 and 48
Percentage of Participants Achieving a Genital VNS of "4 - A Lot Less Noticeable" or "5 - No Longer Noticeable" at Week 24	The VNS is a patient-reported measure of vitiligo treatment success that has a 5-point scale. The participant was asked to respond to the following query: "Compared with before treatment, how noticeable is the vitiligo now? Responses: (1) more noticeable, (2) as noticeable, (3) slightly less noticeable, (4) a lot less noticeable, or (5) no longer noticeable. The baseline genital photograph was shown to the participant for reference. A VNS score of 4 or 5 can be interpreted as representing treatment success.	Baseline; Week 24
Percentage of Participants in Each Category of the Color-matching Question at Weeks 24 and 48	Participants were shown their baseline genital photograph for reference, and were asked to respond to the following query: "At this point of your treatment, how well does your genital skin color match between your treated vitiligo skin and normal skin?: Responses: (1) excellent, (2) very good, (3) good, (4) poor, or (5) very poor.	Baseline; Weeks 24 and 48

Collaborators and Investigators

• Sponsor: Incyte Corporation

Study record dates

Study Major Dates

• Study Start (Actual): April 11, 2023
• Primary Completion (Actual): March 6, 2025
• Study Completion (Actual): March 6, 2025

Study Registration Dates

• First Submitted: February 20, 2023
• First Submitted That Met QC Criteria: February 20, 2023
• First Posted (Actual): March 2, 2023

Study Record Updates

• Last Update Posted (Actual): March 20, 2026
• Last Update Submitted That Met QC Criteria: February 28, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: Vitiligo; INCB18424; Ruxolitinib Cream; Genital Vitiligo
• Additional Relevant MeSH Terms: Skin Diseases; Hypopigmentation; Pigmentation Disorders; Skin and Connective Tissue Diseases; Vitiligo; ruxolitinib
• Other Study ID Numbers: INCB 18424-219; 2023-503737-22-00 (Registry Identifier: EU CT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency

• IPD Sharing Time Frame: Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
• IPD Sharing Access Criteria: Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

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