A Study to Evaluate the Efficacy and Safety of Ruxolitinib Cream in Children (6 to < 12 Years Old) With Nonsegmental Vitiligo

A Phase 3, Randomized, Double-Blind, Efficacy, and Safety Study of Ruxolitinib Cream in Children (6 to < 12 Years Old) With Nonsegmental Vitiligo

The purpose of this study is to evaluate the efficacy and safety of ruxolitinib cream in children (6 to < 12 years old) with nonsegmental vitiligo.

Study Overview

• Status: Recruiting
• Conditions: NonSegmental Vitiligo
• Intervention / Treatment: Drug: Vehicle Cream; Drug: Ruxolitinib Cream

• Study Type: Interventional
• Enrollment (Estimated): 250
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Incyte Corporation Call Center (US); Phone Number: 1.855.463.3463; Email: medinfo@incyte.com
• Study Contact Backup: Name: Incyte Corporation Call Center (ex-US); Phone Number: +800 00027423; Email: eumedinfo@incyte.com

Study Locations

• Argentina
  • Buenos Aires, Argentina, C1181ACH
    • Not yet recruiting
    • Hospital Italiano de Buenos Aires
  • Buenos Aires, Argentina, C1425BEA
    • Not yet recruiting
    • Instituto de Neumonologia Y Dermatologia
  • Buenos Aires, Argentina, 01425
    • Not yet recruiting
    • Psoriahue
  • Buenos Aires, Argentina, C1426
    • Not yet recruiting
    • Consultorios Médicos Dr. Doreski
  • Buenos Aires, Argentina, C1426
    • Not yet recruiting
    • Derma Internacional S.A.
  • Mar del Plata, Argentina, B7600FYK
    • Not yet recruiting
    • Centro de Investigaciones Medicas Mar del Plata
  • Pilar, Argentina, B16290DT
    • Not yet recruiting
    • Hospital Universitario Austral
  • San Miguel de Tucumán, Argentina, 04000
    • Not yet recruiting
    • Hospital del Niño Jesus
• Austria
  • Graz, Austria, 08036
    • Recruiting
    • Universitaetsklinikum Graz
  • Innsbruck, Austria, 06020
    • Not yet recruiting
    • Medical University Innsbruck
• Belgium
  • Brussels, Belgium, 01070
    • Recruiting
    • Hopital Universitaire de Bruxelles (Academisch Ziekenhuis Brussel)
  • Ghent, Belgium, 09000
    • Recruiting
    • Universitair Ziekenhuis Gent
  • Leuven, Belgium, 03000
    • Recruiting
    • Universitair Ziekenhuis Leuven
  • Woluwe-Saint-Lambert, Belgium, 01200
    • Not yet recruiting
    • Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-Luc
• Bulgaria
  • Dupnitsa, Bulgaria, 02600
    • Recruiting
    • Medical Center Asklepii OOD
  • Gabrovo, Bulgaria, 05300
    • Recruiting
    • Mhat Dr. Tota Venkova Ad
  • Pleven, Bulgaria, 05800
    • Not yet recruiting
    • Medical Center Medkonsult Pleven Ood
  • Sevlievo, Bulgaria, 05400
    • Recruiting
    • Medical Center Unimed Eood
  • Sofia, Bulgaria, 01528
    • Recruiting
    • 28 Diagnostic and Consultative Center
  • Sofia, Bulgaria, 01431
    • Not yet recruiting
    • UMHAT Aleksandrovska
  • Sofia, Bulgaria, 01606
    • Recruiting
    • Mc Euroderma Clinic
  • Sofia, Bulgaria, 01618
    • Recruiting
    • Diagnostic Consultation Center Xx - Sofia Eood
• Denmark
  • Gandrup, Denmark, 09362
    • Not yet recruiting
    • Sanos Clinic
  • Hellerup, Denmark, 02900
    • Recruiting
    • Herlev and Gentofte Hospital
• France
  • Argenteuil, France, 95107
    • Not yet recruiting
    • Centre Hospitalier Victor Dupouy
  • Bordeaux, France, 33076
    • Recruiting
    • Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin
  • Créteil, France, 94010
    • Not yet recruiting
    • Centre Hospitalier Intercommunal de Créteil
  • Lyon, France, 69003
    • Recruiting
    • Hospices Civils de Lyon (Hcl) - Hopital Edouard Herriot (Heh)
  • Nantes, France, 44093
    • Not yet recruiting
    • Centre Hospitalier Universitaire de Nantes (Chu de Nantes) - Hopital Hotel Dieu
  • Nice, France, 06200
    • Not yet recruiting
    • Centre Hospitalier Universitaire de Nice,Hopital L Archet
  • Paris, France, 75015
    • Not yet recruiting
    • Hôpital Universitaire Necker Enfants Malades
  • Reims, France, 51100
    • Not yet recruiting
    • Polyclinique de Courlancy
  • Toulouse, France, 31059
    • Recruiting
    • Centre Hospitalier Universitaire (CHU) de Toulouse - Hopital Larrey
  • Vandœuvre-lès-Nancy, France, 54500
    • Not yet recruiting
    • CHU Nancy
• Germany
  • Berlin, Germany, 10117
    • Recruiting
    • Charite - Universitaetsmedizin Berlin - Campus Charite Mitte (CCM)
  • Bochum, Germany, 44791
    • Not yet recruiting
    • Universitaetsklinikum der Ruhr Universitaet Bochum (UKRUB), St. Josef Hospital
  • Chemnitz, Germany, 09117
    • Withdrawn
    • DRK Krankenhaus Chemnitz-Rabenstein
  • Erlangen, Germany, 91054
    • Not yet recruiting
    • Hautklinik Universitatsklinikum Erlangen
  • Frankfurt, Germany, 60590
    • Withdrawn
    • Universitaetsklinik Frankfurt
  • Hamburg, Germany, 22149
    • Recruiting
    • Katholisches Kinderkrankenhaus Wilhelmstift
  • Kiel, Germany, 24105
    • Recruiting
    • University Medical Center Schleswig-Holstein
  • Leipzig, Germany, 04103
    • Recruiting
    • Universitaetsklinikum Leipzig
  • Mainz, Germany, 55131
    • Not yet recruiting
    • Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz Iii
  • München, Germany, 81675
    • Recruiting
    • Klinikum Rechts Der Isar Der Technischen Universitaet Muenchen (Hopital Rechts Der Isar)
  • Münster, Germany, 48149
    • Recruiting
    • University hospital Muenster
• Hungary
  • Budapest, Hungary, 01033
    • Recruiting
    • Clinexpert
  • Debrecen, Hungary, 04032
    • Recruiting
    • Debreceni Egyetem Klinikai Kozpon Belgyogy Klinika
  • Pécs, Hungary, 07632
    • Not yet recruiting
    • University of Pecs-Clinical Center
  • Szeged, Hungary, 06720
    • Not yet recruiting
    • University of Szeged
• Italy
  • Bologna, Italy, 40138
    • Not yet recruiting
    • A.O. Policlinico Sant'Orsola Malpighi-Universita Degli Studi Di Bologna
  • Brescia, Italy, 25123
    • Not yet recruiting
    • Azienda Ospedaliera Spedali Civili di Brescia-Universita degli Studi Di Brescia
  • Catania, Italy, 95123
    • Not yet recruiting
    • Azienda Ospedaliero-Universitaria Policlinico Vittorio Emanuele Ospedale Gaspare Rodolico Di Catania
  • Milan, Italy, 20122
    • Not yet recruiting
    • Uoc Dermatologia Ospedale Maggiore Policlinico, Fondazione Irccs Ca' Granda
  • Napoli, Italy, 80131
    • Not yet recruiting
    • Università degli Studi di Napoli Federico II
  • Padua, Italy, 35100
    • Not yet recruiting
    • Azienda Ospedale Universita Di Padova
  • Roma, Italy, 00133
    • Withdrawn
    • Fondazione PTV Policlinico Tor Vergata
  • Roma, Italy, 00167
    • Not yet recruiting
    • Istituto Di Ricovero E Cura A Carattere Scientifico (Ircss)
  • Rome, Italy, 00168
    • Not yet recruiting
    • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
  • Rome, Italy, 00165
    • Not yet recruiting
    • IRCCS Ospedale Pediatrico Bambino Gesù
• Netherlands
  • Amsterdam, Netherlands, 1105 AZ
    • Recruiting
    • Amsterdam UMC Research BV
• Poland
  • Gdansk, Poland, 80-546
    • Recruiting
    • Centrum Badan Klinicznych PI-House Sp. z o.o.
  • Katowice, Poland, 40-568
    • Recruiting
    • Care Clinic Sp. Z O.O.
  • Krakow, Poland, 31-559
    • Recruiting
    • Diamond Clinic Specjalistyczne Poradnie Lekarskie
  • Lublin, Poland, 20-607
    • Recruiting
    • DERMEDIC Iwona Zdybska
  • Nowa Sól, Poland, 67-100
    • Recruiting
    • Twoja Przychodnia - Medyczne Nowa Sol
  • Osielsko, Poland, 86-031
    • Recruiting
    • Dermodent Centrum Medyczne Aldona Czajkowska Rafał Czajkowski
  • Ostrowiec Świętokrzyski, Poland, 27-400
    • Withdrawn
    • Dermedic Jacek Zdybski
  • Rzeszów, Poland, 35-055
    • Recruiting
    • Uniwersytecki Szpital Kliniczny Im. Fryderyka Chopina W Rzeszowie
  • Szczecin, Poland, 71-500
    • Recruiting
    • Twoja Przychodnia SCM
  • Warsaw, Poland, 02-625
    • Recruiting
    • Evimed Sp. Z O. O.
• Portugal
  • Coimbra, Portugal, 3000-602
    • Not yet recruiting
    • Centro Hospitalar E Universitario Coimbra
  • Lisbon, Portugal, 1169-050
    • Not yet recruiting
    • Centro Hospitalar de Lisboa Central - Hospital Santo Antonio Dos Capuchos
  • Porto, Portugal, 4099-001
    • Recruiting
    • Hospital Geral de Santo Antonio
• Spain
  • Alcorcón, Spain, 28922
    • Recruiting
    • Hospital Universitario Fundacion Alcorcon
  • Badalona, Spain, 08916
    • Recruiting
    • Hospital Universitari Germans Trias i Pujol
  • Barcelona, Spain, 08026
    • Recruiting
    • Hospital De La Santa Creu I Sant Pau
  • Esplugues de Llobregat, Spain, 08950
    • Recruiting
    • Hospital Sant Joan de Deu Barcelona
  • Madrid, Spain, 28009
    • Recruiting
    • Hospital Infantil Unversitario Nino Jesus
  • Zaragoza, Spain, 50009
    • Recruiting
    • Hospital Universitario Miguel Servet de Zaragoza
• United Kingdom
  • Croydon, United Kingdom, CR7 7YE
    • Not yet recruiting
    • Croydon University Hospital
  • London, United Kingdom, SE1 9RT
    • Not yet recruiting
    • Guys Hospital
  • London, United Kingdom, WC1N 3JH
    • Not yet recruiting
    • Great Ormond Street Hospital For Children NHS Foundation Trust
  • London, United Kingdom, E1 1FR
    • Not yet recruiting
    • The Royal London Hospital - Barts Health NHS Trust
  • Nottingham, United Kingdom, NG7 2UH
    • Not yet recruiting
    • Queen's Medical Centre - Nottingham University Hospitals NHS Trust
  • Sheffield, United Kingdom, S10 2TH
    • Not yet recruiting
    • Sheffield Childrens Hospital
  • Walsall, United Kingdom, WS2 9PS
    • Not yet recruiting
    • Manor Hospital (Walsall) - Walsall Healthcare Nhs Trust

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Aged 6 to < 12 years at the time of signing the ICF.
• Clinical diagnosis of nonsegmental vitiligo with depigmented area including ≥ 0.5% BSA on the face, ≥ 0.5 F-VASI, ≥ 3% BSA on nonfacial areas, ≥ 3 T-VASI.
• Total body vitiligo area does not exceed 10% BSA.
• Pigmented hair within some of the areas of vitiligo on the face.
• Must agree to discontinue all agents used to treat vitiligo from screening through the final safety follow-up visit.
• For sexually active participants (except participants who are prepubescent) willingness to avoid pregnancy or fathering a child as defined in the protocol.

Exclusion Criteria:

• Diagnosis of other forms of vitiligo (eg, segmental).
• Other differential diagnosis of vitiligo or other skin depigmentation disorders (eg, piebaldism, pityriasis alba, leprosy, postinflammatory hypopigmentation, progressive macule hypomelanosis, nevus anemicus, chemical leukoderma, and tinea versicolor).
• Any other skin disease that, in the opinion of the investigator, would interfere with the study drug application or study assessments.
• Prior or current use of depigmentation treatments (eg, monobenzone).
• Concurrent conditions and history of protocol-defined diseases
• Any serious illness or medical, physical, or psychiatric condition(s) that, in the investigator's opinion, would interfere with full participation in the study, including application of study cream and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data.
• Use of protocol-defined treatments within the indicated washout period before baseline.
• History of treatment failure with any systemic or topical JAK inhibitor for vitiligo or any other inflammatory condition.
• Protocol-defined clinically significant abnormal laboratory values at screening.
• BMI-for-age < 5th percentile or ≥ 85th percentile according to the CDC BMI Percentile Calculator for Child and Teen.
• Pregnant or lactating participants or those considering pregnancy during the period of their study participation.
• In the opinion of the investigator, unable or unlikely to comply with the application schedule and study evaluations.
• Living with anyone participating in any current Incyte-sponsored ruxolitinib cream study.
• Employees of the sponsor or investigator or are otherwise dependents of them.
• Known allergy or reaction to any component of the study cream formulation.
• The following participants are excluded in France: vulnerable populations according to article L.1121-6 of the French Public Health Code and adults under legal protection, or who are unable to express their consent per article L.1121-8 of the French Public Health Code, not affiliated to a social security per article L.1121-8-1 of the French Public Health code.
• In the EU, participants considered incapacitated according to EU CTR No. 536/2014 Articles 2 and 31.

Other protocol-defined Inclusion/Exclusion Criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ruxolitinib 1.5 % Cream; Participants received ruxolitinib 1.5% cream, applied topically to the affected area as defined in the protocol.	Drug: Ruxolitinib Cream; Ruxolitinib cream applied topically to the affected area as a thin film twice daily.; Other Names: INCB018424 Phosphate Cream
Placebo Comparator: Vehicle Cream; Participants received vehicle cream, applied topically to the affected area as defined by the protocol.	Drug: Vehicle Cream; Matching vehicle cream applied topically to the affected area as a thin film twice daily.
Experimental: Ruxolitinib 0.75 % Cream; Participants received ruxolitinib 0.75% cream, applied topically to the affected area as defined in the protocol.	Drug: Ruxolitinib Cream; Ruxolitinib cream applied topically to the affected area as a thin film twice daily.; Other Names: INCB018424 Phosphate Cream

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of Participants Achieving a ≥ 75% Improvement From Baseline in the Facial Vitiligo Area Scoring Index (F-VASI75)	≥75% improvement in facial Vitiligo Area Scoring Index.	Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of Participants Achieving Facial Vitiligo Area Scoring Index (F-VASI50)	≥50% improvement in facial Vitiligo Area Scoring Index.	Week 24
Proportion of Participants Achieving Facial Vitiligo Area Scoring Index (F-VASI90)	≥90% improvement in facial Vitiligo Area Scoring Index.	Week 24
Proportion of Participants Achieving a ≥ 50% Improvement From Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50)	≥50% improvement in total body Vitiligo Area Scoring Index.	Week 24
Percentage change from baseline in F-BSA	Facial body surface area (F-BSA) takes into account the facial depigmented areas as a percentage of the total body area.	Week 24
Number of Treatment Emergent Adverse Events (TEAEs)	TEAE defined as any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug.	Up to Week 52 and 30 days
Pharmacokinetic (PK) of Ruxolitinib: Trough concentrations	Trough is defined as the concentration reached by a drug immediately before the next dose is administered.	Weeks 6, 24, 30 and 52
Proportion of participants achieving a Vitiligo Noticeability Scale (VNS) score of 4 (a lot less noticeable) or 5 (no longer noticeable)	The VNS is a patient-reported measure of vitiligo treatment success that has a 5-point scale.	Week 24 and Week 52
Proportion of participants in each category for the color-matching question	The color-matching question is a patient-reported measure of vitiligo treatment success that has a 5-point scale.	Week 24 and Week 52
Proportion of participants who report facial assessment of Patient Global Impression of Change-Vitiligo (F-PaGIC-V) of very much improved or much improved	The F-PaGIC-V is an assessment of improvement by the participant that has a 7-point scale.	Week 24 and Week 52
Proportion of participants who report total body assessment of Patient Global Impression of Change-Vitiligo (T-PaGIC-V) of very much improved or much improved	The T-PaGIC-V is an assessment of improvement by the participant that has a 7-point scale.	Week 24 and Week 52
Change from baseline in Psychosocial Impact Questionnaire for Pediatric Patients Who Have Nonsegmental Vitiligo (VIT-PIQ)	The VIT-PIQ is a 17-item questionnaire to assess Quality of Life (QOL) of children with vitiligo.	Week 24 and Week 52
Change from baseline in Children's Dermatology Life Quality Index (CDLQI)	The CDLQI is the youth/children's version of the Dermatology Life Quality Index.	Week 24 and Week 52
Change from baseline in Patient Global Impression of Bother - Vitiligo (PGIB-V)	The PGIB-V is a single question with an 11-point scale ("not at all" to "a whole lot") to assess the participant's level of bother because of their vitiligo.	Week 24 and Week 52
Mean satisfaction score	The treatment satisfaction tool is a fit-for-purpose questionnaire to assess treatment satisfaction in children with nonsegmental vitiligo.	Week 24 and Week 52

Collaborators and Investigators

• Sponsor: Incyte Corporation
• Investigators: Study Director: Incyte Medical Monitor, Incyte Corporation

Publications and helpful links

Helpful Links

• A Study to Evaluate the Efficacy and Safety of Ruxolitinib Cream in Children (6 to < 12 Years Old) With Nonsegmental Vitiligo

Study record dates

Study Major Dates

• Study Start (Actual): November 13, 2025
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: January 27, 2025
• First Submitted That Met QC Criteria: January 27, 2025
• First Posted (Actual): February 3, 2025

Study Record Updates

• Last Update Posted (Actual): December 17, 2025
• Last Update Submitted That Met QC Criteria: December 11, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: pediatric; NonSegmental Vitiligo
• Additional Relevant MeSH Terms: Skin Diseases; Hypopigmentation; Pigmentation Disorders; Vitiligo
• Other Study ID Numbers: INCB18424-309; 2024-513171-41-00 (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