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NCT05750823

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

Completed Phase 2 Results posted Last updated 20 March 2026
What this trial tests

Phase 2 trial testing Ruxolitinib Cream in Nonsegmental Vitiligo With Genital Involvement in 49 participants. Completed in 6 March 2025.

Timeline
11 April 2023
Primary endpoint
6 March 2025
6 March 2025

Quick facts

Lead sponsorIncyte Corporation
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment49
Start date11 April 2023
Primary completion6 March 2025
Estimated completion6 March 2025
Sites13 locations across France, Canada, United States

Drugs / interventions tested

Conditions studied

Sponsor

Incyte Corporation — full company profile →

Who can join

18 and older, any sex, with Nonsegmental Vitiligo With Genital Involvement. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percentage of Participants Achieving a Genital Vitiligo Noticeability Scale (VNS) of "4 - A Lot Less Noticeable" or "5 - No Longer Noticeable" at Week 48 Primary · Baseline; 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.

GroupValue95% CI
Ruxolitinib 1.5% Cream BID27.0
Number of Participants With Any Treatment-emergent Adverse Event (TEAE) Secondary · up to approximately 14 months

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.

GroupValue95% CI
Ruxolitinib 1.5% Cream BID19
Percentage of Participants Achieving Genital-Physician Global Vitiligo Assessment (PhGVA) of 0 or 1 at Week 48 Secondary · 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, exten

GroupValue95% CI
Ruxolitinib 1.5% Cream BID10.8
Change From Baseline in Affected Body Surface Area (BSA) in the Genital Region at Weeks 24 and 48 Secondary · Baseline; 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 th

Baseline
GroupValue95% CI
Ruxolitinib 1.5% Cream BID0.762± 0.4899
Change from baseline at Week 24
GroupValue95% CI
Ruxolitinib 1.5% Cream BID-0.115± 0.4224
Change from baseline at Week 48
GroupValue95% CI
Ruxolitinib 1.5% Cream BID-0.185± 0.4129
Percentage of Participants Achieving ≥50% Improvement in Total Body Vitiligo Area Scoring Index (T-VASI50) at Weeks 24 and 48 Secondary · Baseline; 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 ar

Week 24
GroupValue95% CI
Ruxolitinib 1.5% Cream BID24.4
Week 48
GroupValue95% CI
Ruxolitinib 1.5% Cream BID35.1
Percentage of Participants Achieving ≥75% Improvement in Total Body Vitiligo Area Scoring Index (T-VASI75) at Weeks 24 and 48 Secondary · Baseline; 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 ar

Week 24
GroupValue95% CI
Ruxolitinib 1.5% Cream BID7.3
Week 48
GroupValue95% CI
Ruxolitinib 1.5% Cream BID16.2
Percentage of Participants Achieving ≥90% Improvement in Total Body Vitiligo Area Scoring Index (T-VASI90) at Weeks 24 and 48 Secondary · Baseline; 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 ar

Week 24
GroupValue95% CI
Ruxolitinib 1.5% Cream BID0.0
Week 48
GroupValue95% CI
Ruxolitinib 1.5% Cream BID8.1
Percentage of Participants Achieving a Genital VNS of "4 - A Lot Less Noticeable" or "5 - No Longer Noticeable" at Week 24 Secondary · Baseline; 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.

GroupValue95% CI
Ruxolitinib 1.5% Cream BID17.9
Percentage of Participants in Each Category of the Color-matching Question at Weeks 24 and 48 Secondary · Baseline; 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.

Week 24; Excellent
GroupValue95% CI
Ruxolitinib 1.5% Cream BID10.3
Week 24; Very good
GroupValue95% CI
Ruxolitinib 1.5% Cream BID15.4
Week 24; Good
GroupValue95% CI
Ruxolitinib 1.5% Cream BID25.6
Week 24; Poor
GroupValue95% CI
Ruxolitinib 1.5% Cream BID43.6
Week 24; Very poor
GroupValue95% CI
Ruxolitinib 1.5% Cream BID5.1
Week 48; Excellent
GroupValue95% CI
Ruxolitinib 1.5% Cream BID10.8
Week 48; Very good
GroupValue95% CI
Ruxolitinib 1.5% Cream BID16.2
Week 48; Good
GroupValue95% CI
Ruxolitinib 1.5% Cream BID40.5

Adverse events — posted to ClinicalTrials.gov

Time frame: up to approximately 14 months. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Ruxolitinib 1.5% Cream BID
Serious: 1/49 (2%)
Deaths: 0/49

Serious adverse events (1 terms)

ReactionSystemRuxolitinib 1.5% Cream BID
Substance-induced psychotic disorderPsychiatric disorders

Most-reported serious reactions: Substance-induced psychotic disorder.

Data from ClinicalTrials.gov NCT05750823 adverse events section.

Sponsor's own description

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.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Up-and-Coming Drugs for the Treatment of Vitiligo.
    Seong SH, Oh SH. · · 2024 · cited 8× · PMID 39082655 · DOI 10.5021/ad.24.038
  2. Novel Janus Kinase Inhibitors in the Treatment of Dermatologic Conditions.
    Ryguła I, Pikiewicz W, Kaminiów K. · · 2023 · cited 6× · PMID 38138551 · DOI 10.3390/molecules28248064
  3. Update vulval dermatology - diagnostics and therapy.
    Brägelmann C, Wölber L, Susok L, Anemüller W, et al · · 2025 · cited 2× · PMID 39711289 · DOI 10.1111/ddg.15541
  4. Emerging Therapeutic Innovations for Vitiligo Treatment.
    Li W, Dong P, Zhang G, Hu J, et al · · 2025 · cited 1× · PMID 40136446 · DOI 10.3390/cimb47030191
  5. Recent Advances in Vitiligo Treatment.
    Qiu Y, Su X, Chen J, Jiang S, et al · · 2026 · PMID 41940356 · DOI 10.2147/itt.s577653
  6. Update Vulvadermatologie – Diagnostik und Therapie
    Brägelmann C, Wölber L, Susok L, Anemüller W, et al · · 2025

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05750823.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing