Clinical trial NCT04052425: Topical Ruxolitinib Evaluation in Vitiligo Study 1 (TRuE-V1)
Official title: Topical Ruxolitinib Evaluation in Vitiligo Study 1 (TRuE-V1): A Phase 3, Double-Blind, Randomized, Vehicle-Controlled, Efficacy and Safety Study of Ruxolitinib Cream Followed by an Extension Period in Participants With Vitiligo
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Non-segmental Vitiligo
Keywords: Vitiligo
non-segmental
JAK inhibitor
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2019-08-09
Enrollment: 330
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: Participants will receive ruxolitinib cream or vehicle for 24 weeks, after which they will be offered the opportunity to continue in the treatment extension period. Participants initially randomized to vehicle will be crossed over to active drug, and participants treated with ruxolitinib cream will receive an additional 28 weeks of treatment with ruxolitinib cream.
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy and safety of ruxolitinib cream in adolescent and adult participants with non-segmental vitiligo for whom total body involved vitiligo area (facial and nonfacial) does not exceed 10% body surface area (BSA).


Eligibility criteria: Key Inclusion Criteria:

* Clinical diagnosis of non-segmental vitiligo with depigmented area including ≥ 0.5% BSA on the face, ≥ 0.5 F-VASI, ≥ 3% BSA on nonfacial areas, ≥ 3 T-VASI, and 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.
* Must be willing to take appropriate contraceptive measures to avoid pregnancy or fathering a child for the duration of study participation.

Key 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 (eg, piebaldism, pityriasis alba, leprosy, postinflammatory hypopigmentation, progressive macule hypomelanosis, nevus anemicus, chemical leukoderma, and tinea versicolor).
* Have used depigmentation treatments (eg, monobenzone) for past treatment of vitiligo or other pigmented areas.
* Use of protocol-defined treatments within the indicated washout period before baseline.

Primary outcomes:
measure: Percentage of Participants Achieving a ≥ 75% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI75) Score at Week 24
timeFrame: Baseline; Week 24
Secondary outcomes:
measure: Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI50) Score at Week 24
timeFrame: Baseline; Week 24
measure: Percentage of Participants Achieving a ≥ 90% Improvement From Baseline in the Face Vitiligo Area Scoring Index (F-VASI90) Score at Week 24
timeFrame: Baseline; Week 24
measure: Percentage of Participants Achieving a ≥ 50% Improvement From Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) Score at Week 24
timeFrame: Baseline; Week 24
measure: Percentage of Participants Achieving a Vitiligo Noticeability Scale (VNS) of 4 or 5 at Week 24
timeFrame: Baseline; Week 24
measure: Percentage Change From Baseline in Facial Body Surface Area (F-BSA) at Week 24
timeFrame: Baseline; Week 24
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Double-Blind Period
timeFrame: from the time of Informed Consent Form signing until at least 30 days after the last application of study drug (up to Week 24)
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Treatment-Extension Period
timeFrame: from the completion of the Week 24 assessments until at least 30 days after the last application of study drug (up to Week 52 + 30 days)
measure: Percentage of Participants Achieving a ≥ 25% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25) Score at Week 24
timeFrame: Baseline; Week 24
measure: Percentage of Participants Achieving a ≥ %25, ≥ %50, ≥ 75%, and ≥ 90% Improvement in the Face Vitiligo Area Scoring Index (F-VASI25/50/75/90) Score at Week 52
timeFrame: Baseline; Week 52
measure: Percentage Change From Baseline in F-VASI at Week 24
timeFrame: Baseline; Week 24
measure: Percentage Change From Baseline in F-VASI at Week 52
timeFrame: Baseline; Week 52
measure: Percentage Change From Baseline in F-BSA at Week 52
timeFrame: Baseline; Week 52
measure: Percentage Change From Baseline in T-VASI at Week 24
timeFrame: Baseline; Week 24
measure: Percentage Change From Baseline in T-VASI at Week 52
timeFrame: Baseline; Week 52
measure: Percentage Change From Baseline in T-BSA at Week 24
timeFrame: Baseline; Week 24
measure: Percentage Change From Baseline in T-BSA at Week 52
timeFrame: Baseline; Week 52
measure: Percentage of Participants Achieving a ≥ 25%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/75/90) Score at Week 24
timeFrame: Baseline; Week 24
measure: Percentage of Participants Achieving a ≥ 25%, ≥ 50%, ≥ 75%, and ≥ 90% Improvement in the Total Body Vitiligo Area Scoring Index (T-VASI25/50/75/90) Score at Week 52
timeFrame: Baseline; Week 52
measure: Percentage of Participants in Each Category of VNS During the Treatment Period (Double-Blind and Treatment-Extension Periods)
timeFrame: Baseline; Week 24 and Week 52
measure: Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 24
timeFrame: Baseline; Week 24
measure: Change From Baseline in DLQI at Week 52
timeFrame: Baseline; Week 52
measure: Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) During the Treatment Period (Double-Blind and Treatment-Extension Periods)
timeFrame: Baseline; Week 24 and Week 52
measure: Trough Plasma Concentrations of Ruxolitinib at Weeks 4, 24, and 40
timeFrame: pre-dose at Weeks 4, 24, and 40
Other outcomes:
Locations:
city: Hoover
country: United States
facility: Cahaba Dermatology
state: Alabama
city: Scottsdale
country: United States
facility: Cognitive Clinical Trials Scottsdale Btc Ppds
state: Arizona
city: Hot Springs National Park
country: United States
facility: Burke Pharmaceutical Research
state: Arkansas
city: Fountain Valley
country: United States
facility: First Oc Dermatology
state: California
city: Huntington Beach
country: United States
facility: Marvel Clinical Research Llc
state: California
city: San Diego
country: United States
facility: Rady Children'S Hospital - San Diego
state: California
city: San Francisco
country: United States
facility: University of California San Francisco Sub Location
state: California
city: Danbury
country: United States
facility: Clinical Research Center of Ct
state: Connecticut
city: Hialeah
country: United States
facility: Harmony Medical Research Institute
state: Florida
city: Miami Lakes
country: United States
facility: San Marcus Research Clinic Inc.
state: Florida
city: Tampa
country: United States
facility: ForCare Medical Center
state: Florida
city: Tampa
country: United States
facility: Forcare Clinical Research Fcr Forward Clinical Trials, Inc
state: Florida
city: West Palm Beach
country: United States
facility: Metabolic Research Institute Inc
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Metairie
country: United States
facility: Clinical Trials Management Llc
state: Louisiana
city: Bay City
country: United States
facility: Great Lakes Research Group Inc
state: Michigan
city: Brighton
country: United States
facility: Dermatology Specialists of Brighton
state: Michigan
city: Brooklyn
country: United States
facility: Suny Downstate Medical Center
state: New York
city: Forest Hills
country: United States
facility: Forest Hills Dermatology Group
state: New York
city: New York
country: United States
facility: The Dermatology Specialists Greenwich
state: New York
city: Raleigh
country: United States
facility: Wake Research Associates Llc
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest University
state: North Carolina
city: Broomall
country: United States
facility: Kgl Skin Study Center
state: Pennsylvania
city: Plymouth Meeting
country: United States
facility: Dermatology Associates of Plymouth Meeting
state: Pennsylvania
city: Anderson
country: United States
facility: Palmetto Clinical Trial Services
state: South Carolina
city: Murfreesboro
country: United States
facility: International Clinical Research Tennessee Llc
state: Tennessee
city: San Antonio
country: United States
facility: Progressive Clinical Research
state: Texas
city: San Antonio
country: United States
facility: Dermatology Clinical Research Center of San Antonio
state: Texas
city: Spokane
country: United States
facility: Dermatology Specialists of Spokane
state: Washington
city: Pleven
country: Bulgaria
facility: Multiprofile Hospital For Active Treatement - Clinic of Dermatology and Venerology
city: Sofia
country: Bulgaria
facility: DCC 28
city: Sofia
country: Bulgaria
facility: Medical Center Eurohealth
city: Calgary
country: Canada
facility: Dermatology Research Institute
state: Alberta
city: Calgary
country: Canada
facility: Institute For Skin Advancement
state: Alberta
city: Peterborough
country: Canada
facility: Skin Centre For Dermatology
state: Ontario
city: Windsor
country: Canada
facility: Windsor Clinical Research Inc
state: Ontario
city: Montreal
country: Canada
facility: McGill University Health Centre / Carey/Wang Clinic
state: Quebec
city: Westmount
country: Canada
facility: Siena Medical Reserch Corporation
state: Quebec
city: Nantes
country: France
facility: Centre Hospitalier Universitaire de Nantes
city: Nice Cedex 3
country: France
facility: Chu de Nice - Hopital L'Archet 1
city: Rouen
country: France
facility: Hopital Charles Nicolle Chu Rouen - Hopital de Bois-Guillaume
city: Toulouse
country: France
facility: Chu de Toulouse Hopital Larrey Centre de Reference Des Maladies Rares de La Peau Service de Dermatol
city: Dresden
country: Germany
facility: University Clinic Carl Gustav Carus, Technical University Dresden
city: Muenster
country: Germany
facility: Universitatsklinik Munster Dermatologie
city: Firenze
country: Italy
facility: Presidio Ospedaliero Piero Palagi
city: Rome
country: Italy
facility: Istituto Dermatologico San Gallicano
city: Gdansk
country: Poland
facility: Synexus - Polska Sp Z Oo Oddzial W Gdansk
city: Gdynia
country: Poland
facility: Synexus Polska Sp. Z O.O. Oddzial W Gdyni
city: Katowice
country: Poland
facility: Synexus - Sp Z Oo Oddzial W Katowice
city: Ostrowiec
country: Poland
facility: Dermedic Dr. Zdybski
city: Poznan
country: Poland
facility: Synexus Polska Sp. Z O.O. Oddzial W Poznaniu
city: Torun
country: Poland
facility: Poradnia Dermatologiczno-Wenerologiczna Mediderm S.C. Nzoz
city: Wroclaw
country: Poland
facility: Synexus Polska Sp. Z O.O. Oddzial We Wroclawiu
city: Barcelona
country: Spain
facility: Hospital Cima Sanitas
city: Barcelona
country: Spain
facility: Hospital Clinic de Barcelona
city: Granada
country: Spain
facility: Hospital Universitario San Cecilio
city: Pamplona
country: Spain
facility: Clinica Universidad de Navarra (Cun)
Study officials:
affiliation: Incyte Corporation
name: Kathleen Butler, MD
role: STUDY_DIRECTOR
References:
citation: Rosmarin D, Passeron T, Pandya AG, Grimes P, Harris JE, Desai SR, Lebwohl M, Ruer-Mulard M, Seneschal J, Wolkerstorfer A, Kornacki D, Sun K, Butler K, Ezzedine K; TRuE-V Study Group. Two Phase 3, Randomized, Controlled Trials of Ruxolitinib Cream for Vitiligo. N Engl J Med. 2022 Oct 20;387(16):1445-1455. doi: 10.1056/NEJMoa2118828.