Efficacy and Safety of Ruxolitinib Cream in Chinese Children Aged 2-11 Years With Non-segmental Vitiligo
Efficacy and Safety of Ruxolitinib Cream in Children Aged 2-11 Years With Non-segmental Vitiligo: A Single-Center, Real-World Study
The goal of this clinical trial is to evaluate the efficacy and safety of Ruxolitinib Phosphate Cream in treating non-segmental vitiligo in children aged 2 to 11 years under real-world conditions. The main question[s] it aims to answer are:
Primary Efficacy: What is the improvement rate (e.g., proportion of participants achieving F-VASI75) after 24 weeks of treatment with Ruxolitinib Phosphate Cream in this population? Safety Profile: What is the safety profile of the treatment over 24 weeks, specifically regarding the incidence of Application Site Acne, Application Site Pruritus, and other adverse events?
Participants will:
Apply Ruxolitinib Phosphate Cream topically to vitiligo lesions as prescribed by the investigator.
Attend scheduled clinic visits at Weeks 4, 8, 12, and 24 for efficacy and safety assessments.
Undergo standardized clinical photography and severity scoring of their vitiligo lesions at each visit.
Report any adverse events or skin reactions experienced during the study period to the research team.
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Descrição detalhada
This is a single-center, prospective, interventional, real-world study conducted in China. The study aims to evaluate the efficacy and safety of Ruxolitinib Phosphate Cream in the treatment of non-segmental vitiligo in children aged 2 to 11 years.
A total of eligible participants will receive topical Ruxolitinib Cream applied to vitiligo lesions as prescribed by the investigator. The treatment regimen and dosage follow the product labeling (if applicable) or standard clinical practice, with no additional randomization or blinding procedures.
The study duration is approximately 3 years, including enrollment, a 24-week treatment observation period, and follow-up. Clinical assessments are scheduled at baseline (Week 0) and at Weeks 4, 8, 12, and 24 post-treatment initiation.
Primary Outcome Measures:
- Proportion of participants achieving ≥75% improvement from baseline in the Facial Vitiligo Area Scoring Index (F-VASI75) at Week 24.
Secondary Outcome Measures:
- Proportion of participants achieving F-VASI50, F-VASI90, and Total Vitiligo Area Scoring Index (T-VASI50, T-VASI75) at Weeks 4, 8, 12, and 24.
- Change from baseline in quality of life assessed by the Children's Dermatology Life Quality Index (CDLQI) or appropriate pediatric dermatology quality of life scale.
- Proportion of participants with ≥50% improvement in T-VASI (T-VASI50) at Week 24.
Safety Outcome Measures:
- Incidence and severity of adverse events (AEs), serious adverse events (SAEs), and adverse drug reactions (ADRs) throughout the study period, with special attention to application site acne, application site pruritus, and other local skin reactions.
- Proportion of participants discontinuing treatment or withdrawing from the study due to AEs.
Eligibility Criteria (Key):
- Inclusion: Children aged 2-11 years, clinically diagnosed with non-segmental vitiligo, with depigmented BSA ≥0.1% and T-VASI ≥0.1, total body depigmentation area ≤10% BSA. Legal guardian provides signed informed consent.
- Exclusion: Diagnosis of segmental or other types of vitiligo; other depigmentation disorders; prior use of depigmenting treatments; history of JAK inhibitor failure; allergy to study drug; participation in another clinical trial; judged unsuitable by investigator.
Data will be collected using standardized Case Report Forms (CRFs) and clinical photography. All statistical analyses will be descriptive. This real-world study will provide critical evidence on the use of Ruxolitinib Phosphate Cream in young children with non-segmental vitiligo, filling the current evidence gap in this age group.
Tipo de estudo
Inscrição (Estimado)
Estágio
- Fase 3
Contactos e Locais
Contato de estudo
- Nome: Yunqing Ren, PhD
- Número de telefone: 0571-86670281
- E-mail: yqren@zju.edu.cn
Estude backup de contato
- Nome: Jipeng Liu, PhD
- Número de telefone: 0571-86670281
- E-mail: liujipeng@zju.edu.cn
Locais de estudo
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Zhejiang
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Hangzhou, Zhejiang, China, 310052
- Children's Hospital, Zhejiang University School of Medicine
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Contato:
- Yunqing Ren
- Número de telefone: 0571-86670281
- E-mail: yqren@zju.edu.cn
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Investigador principal:
- Yunqing Ren
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-
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Filho
Aceita Voluntários Saudáveis
Descrição
Inclusion Criteria:
- Children aged 2 to 11 years, regardless of sex.
- Clinical diagnosis of non-segmental vitiligo, with affected Body Surface Area (BSA) ≥0.1%, T-VASI score ≥0.1, and total depigmented area not exceeding 10% BSA.
- Legal guardian voluntarily provides signed informed consent and agrees to scheduled follow-ups.
Exclusion Criteria:
- Diagnosis of other forms of vitiligo (e.g., segmental vitiligo).
- Presence of other hypopigmentary or depigmentary disorders that could interfere with efficacy assessment (e.g., pityriasis alba, leprosy, post-inflammatory hypopigmentation, progressive macular hypomelanosis, nevus depigmentosus, and tinea versicolor).
- Prior or current use of any treatment for depigmentation.
- History of failure with any systemic or topical JAK inhibitor therapy for vitiligo or any other inflammatory condition.
- Known hypersensitivity to the study drug or its excipients.
- Concurrent participation in another clinical trial.
- Considered by the investigator to be unsuitable for the study for any other reason.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: N / D
- Modelo Intervencional: Atribuição de grupo único
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
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Experimental: Ruxolitinib treatment
In this single-arm clinical trial, all eligible subjects will receive the same investigational intervention.
The study drug is Ruxolitinib Phosphate Cream, 1.5% (w/w), supplied in 100 g tubes.
The dosing regimen consists of applying a thin layer of the cream to all vitiligo lesions twice daily, with approximately 12 hours between applications.
Throughout the treatment period, the total application area must not exceed 10% of the Body Surface Area, and the maximum usage is limited to two tubes (totaling 200 g) per month.
The entire investigational intervention will last for 24 weeks, unless a subject meets early discontinuation criteria beforehand.
It must be emphasized that this drug is for cutaneous use only and is strictly prohibited for application to mucous membranes (e.g., inside the mouth, vagina) or in or around the eyes.
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In this single-arm clinical trial, all eligible subjects will receive the same investigational intervention.
The study drug is Ruxolitinib Phosphate Cream, 1.5% (w/w), supplied in 100 g tubes.
The dosing regimen consists of applying a thin layer of the cream to all vitiligo lesions twice daily, with approximately 12 hours between applications.
Throughout the treatment period, the total application area must not exceed 10% of the Body Surface Area, and the maximum usage is limited to two tubes (totaling 200 g) per month.
The entire investigational intervention will last for 24 weeks, unless a subject meets early discontinuation criteria beforehand.
It must be emphasized that this drug is for cutaneous use only and is strictly prohibited for application to mucous membranes (e.g., inside the mouth, vagina) or in or around the eyes.
Outros nomes:
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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Total-Vitiligo Area Scoring Index (T-VASI)
Prazo: 24 week
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Improvement from baseline in the Total Vitiligo Area Scoring Index (T-VASI) at Week 24.
The T-VASI ranges from 0 to 100, with higher scores indicating worse outcome (greater depigmentation).
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24 week
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Total-Vitiligo Area Scoring Index (T-VASI)
Prazo: Weeks 4, 8, 12
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Improvement from baseline in the Total Vitiligo Area Scoring Index (T-VASI) at Week 4, 8, 12.
The T-VASI ranges from 0 to 100, with higher scores indicating worse outcome (greater depigmentation).
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Weeks 4, 8, 12
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Vitiligo Noticeability Scale (VNS)
Prazo: Weeks 4, 8, 12, 24
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Proportion of patients with Vitiligo Noticeability Scale (VNS) score of 4 or 5 at Weeks 4, 8, 12, and 24.
The VNS is a patient-reported outcome measure ranging from 1 to 5, where 1 = more noticeable and 5 = no longer noticeable.
Higher scores indicate better outcome.
The proportion of patients achieving a score of 4 ("less noticeable") or 5 ("no longer noticeable") will be reported, including the breakdown of patients in each category.
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Weeks 4, 8, 12, 24
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Total Body Surface Area (T-BSA)
Prazo: Weeks 4, 8, 12, 24
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Change from baseline in Total Body Surface Area (T-BSA) affected at Weeks 4, 8, 12, and 24.
T-BSA is measured as the percentage of body surface area affected by vitiligo, ranging from 0% to 100%.
Higher percentage indicates worse outcome.
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Weeks 4, 8, 12, 24
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Children's Dermatology Life Quality Index (CDLQI)
Prazo: Weeks 4, 8, 12, 24
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Change from baseline in Children's Dermatology Life Quality Index (CDLQI) score at Weeks 4, 8, 12, and 24.
The CDLQI is a validated pediatric dermatology quality of life questionnaire ranging from 0 to 30, where 0 = no impact on quality of life and 30 = maximum impact.
Higher scores indicate worse outcome.
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Weeks 4, 8, 12, 24
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Pediatric Quality of Life Inventory (PedsQL) score
Prazo: Weeks 4, 8, 12, 24
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Change from baseline in Pediatric Quality of Life Inventory (PedsQL) score at Weeks 4, 8, 12, and 24.
The PedsQL is a generic quality of life scale ranging from 0 to 100, where 0 = worst quality of life and 100 = best quality of life.
Higher scores indicate better outcome.
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Weeks 4, 8, 12, 24
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Colaboradores e Investigadores
Investigadores
- Investigador principal: Yunqing Ren, PhD, The Children's Hospital of Zhejiang University School of Medicine
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- 2026-IEC-0016
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