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

Официальное название

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

Обзор

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.

Вмешательства

• Препарат Ruxolitinib Cream
  Ruxolitinib cream applied topically to the affected area as a thin film twice daily.
• Препарат Vehicle Cream
  Matching vehicle cream applied topically to the affected area as a thin film twice daily.

Первичные конечные точки

• Proportion of Participants Achieving a ≥ 75% Improvement From Baseline in the Facial Vitiligo Area Scoring Index (F-VASI75) [Срок оценки: Week 24]

Вторичные конечные точки (12)

• Proportion of Participants Achieving Facial Vitiligo Area Scoring Index (F-VASI50) [Срок оценки: Week 24]
• Proportion of Participants Achieving Facial Vitiligo Area Scoring Index (F-VASI90) [Срок оценки: Week 24]
• Proportion of Participants Achieving a ≥ 50% Improvement From Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) [Срок оценки: Week 24]
• Percentage change from baseline in F-BSA [Срок оценки: Week 24]
• Number of Treatment Emergent Adverse Events (TEAEs) [Срок оценки: Up to Week 52 and 30 days]
• Proportion of participants achieving a Vitiligo Noticeability Scale (VNS) score of 4 (a lot less noticeable) or 5 (no longer noticeable) [Срок оценки: Week 24 and Week 52]
• Proportion of participants in each category for the color-matching question [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: Week 24 and Week 52]
• Change from baseline in Psychosocial Impact Questionnaire for Pediatric Patients Who Have Nonsegmental Vitiligo (VIT-PIQ) [Срок оценки: Week 24 and Week 52]
• Change from baseline in Children's Dermatology Life Quality Index (CDLQI) [Срок оценки: Week 24 and Week 52]
• Change from baseline in Patient Global Impression of Bother - Vitiligo (PGIB-V) [Срок оценки: Week 24 and Week 52]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Тройное слепое

Основная цель

Лечение

Центры проведения

Франция · 10 центров

• Centre Hospitalier Victor Dupouy — Argenteuil
• Centre Hospitalier Universitaire (Chu) de Bordeaux - Groupe Hospitalier Pellegrin — Bordeaux
• Centre Hospitalier Intercommunal de Creteil — Créteil
• Hospices Civils de Lyon (Hcl) - Hopital Edouard Herriot (Heh) — Lyon
• Centre Hospitalier Universitaire de Nantes (Chu de Nantes) - Hopital Hotel Dieu — Nantes
• Centre Hospitalier Universitaire de Nice,Hopital L Archet — Nice
• Hopital Universitaire Necker Enfants Malades — Paris
• Polyclinique de Courlancy — Reims
• … и ещё 2 центра

Италия · 9 центров

• A.O. Policlinico Sant'Orsola Malpighi-Universita Degli Studi Di Bologna — Bologna
• Azienda Ospedaliera Spedali Civili Di Brescia-Universita Degli Studi Di Brescia — Brescia
• Azienda Ospedaliero-Universitaria Policlinico Vittorio Emanuele Ospedale Gaspare Rodolico — Catania
• Uoc Dermatologia Ospedale Maggiore Policlinico, Fondazione Irccs Ca' Granda — Milan
• Azienda Ospedaliero Universitaria Federico Ii Di Napoli — Naples
• Azienda Ospedale Universita Di Padova — Padua
• Istituto Di Ricovero E Cura A Carattere Scientifico (Ircss) — Roma
• Fondazione Policlinico Universitario Agostino Gemelli Irccs — Rome
• … и ещё 1 центр

Польша · 9 центров

• Centrum Badan Klinicznych Pi-House Sp. Z O.O. — Gdansk
• Care Clinic Sp. Z O.O. — Katowice
• Diamond Clinic Specjalistyczne Poradnie Lekarskie — Krakow
• Dermedic Iwona Zdybska — Lublin
• Twoja Przychodnia - Medyczne Nowa Sol — Nowa Sól
• Dermodent Centrum Medyczne Aldona Czajkowska Rafał Czajkowski — Osielsko
• Uniwersytecki Szpital Kliniczny Im. Fryderyka Chopina W Rzeszowie — Rzeszów
• Twoja Przychodnia Scm — Szczecin
• … и ещё 1 центр

Аргентина · 8 центров

• Psoriahue — Buenos Aires
• Hospital Italiano de Buenos Aires — Buenos Aires
• Instituto de Neumonologia Y Dermatologia — Buenos Aires
• Consultorios Medicos Dr. Doreski — Buenos Aires
• Derma Internacional S.A. — Buenos Aires
• Hospital Universitario Austral — Derqui-pilar
• Centro de Investigaciones Medicas Mar Del Plata — Mar del Plata
• Hospital Del Nino Jesus — San Miguel de Tucumán

Германия · 8 центров

• Charite - Universitaetsmedizin Berlin - Campus Charite Mitte (Ccm) — Berlin
• Hautklinik Universitatsklinikum Erlangen — Erlangen
• Katholisches Kinderkrankenhaus Wilhelmstift — Hamburg
• University Medical Center Schleswig-Holstein — Kiel
• Universitaetsklinikum Leipzig — Leipzig
• Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz Iii — Mainz
• Klinikum Rechts Der Isar Der Technischen Universitaet Muenchen (Hopital Rechts Der Isar) — München
• University Hospital Muenster — Münster

Болгария · 7 центров

• Medical Center Asklepii Ood — Dupnitsa
• Mhat Dr. Tota Venkova Ad — Gabrovo
• Medical Center Unimed Eood — Sevlievo
• Umhat Aleksandrovska — Sofia
• 28 Diagnostic and Consultative Center — Sofia
• Mc Euroderma Clinic — Sofia
• Diagnostic Consultation Center Xx - Sofia Eood — Sofia

Испания · 6 центров

• Hospital Universitario Fundacion Alcorcon — Alcorcón
• Hospital Universitari Germans Trias I Pujol — Badalona
• Hospital de La Santa Creu I Sant Pau — Barcelona
• Hospital Sant Joan de Deu Barcelona — Esplugues de Llobregat
• Hospital Infantil Unversitario Nino Jesus — Madrid
• Hospital Universitario Miguel Servet de Zaragoza — Zaragoza

Великобритания · 6 центров

• Croydon University Hospital — Croydon
• The Royal London Hospital - Barts Health Nhs Trust — London
• Guys Hospital — London
• Great Ormond Street Hospital For Children Nhs Foundation Trust — London
• Sheffield Childrens Hospital — Sheffield
• Manor Hospital (Walsall) - Walsall Healthcare Nhs Trust — Walsall

Бельгия · 4 центра

• Hopital Universitaire de Bruxelles (Academisch Ziekenhuis Brussel) — Brussels
• Universitair Ziekenhuis Gent — Ghent
• Universitair Ziekenhuis Leuven — Leuven
• Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-Luc — Woluwe-Saint-Lambert

Венгрия · 4 центра

• Clinexpert — Budapest
• Debreceni Egyetem Klinikai Kozpon Belgyogy Klinika — Debrecen
• University of Pecs-Clinical Center — Pécs
• University of Szeged — Szeged

Португалия · 3 центра

• Centro Hospitalar E Universitario Coimbra — Coimbra
• Centro Hospitalar de Lisboa Central - Hospital Santo Antonio Dos Capuchos — Lisbon
• Hospital Geral de Santo Antonio — Porto

Австрия · 2 центра

• Universitaetsklinikum Graz — Graz
• Medical University Innsbruck — Innsbruck

Дания · 2 центра

• Herlev and Gentofte Hospital — Hellerup
• Sanos Clinic — Herlev

Нидерланды · 1 центр

• Amsterdam Umc Research Bv — Amsterdam

Идентификаторы

NCT: NCT06804811 · INCB18424-309 · 2024-513171-41-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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