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.
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Descrizione dettagliata
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 di studio
Iscrizione (Stimato)
Fase
- Fase 3
Contatti e Sedi
Contatto studio
- Nome: Yunqing Ren, PhD
- Numero di telefono: 0571-86670281
- Email: yqren@zju.edu.cn
Backup dei contatti dello studio
- Nome: Jipeng Liu, PhD
- Numero di telefono: 0571-86670281
- Email: liujipeng@zju.edu.cn
Luoghi di studio
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Zhejiang
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Hangzhou, Zhejiang, Cina, 310052
- Children's Hospital, Zhejiang University School of Medicine
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Contatto:
- Yunqing Ren
- Numero di telefono: 0571-86670281
- Email: yqren@zju.edu.cn
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Investigatore principale:
- Yunqing Ren
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
- Bambino
Accetta volontari sani
Descrizione
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.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: N / A
- Modello interventistico: Assegnazione di gruppo singolo
- Mascheramento: Nessuno (etichetta aperta)
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
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Sperimentale: 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.
Altri nomi:
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Total-Vitiligo Area Scoring Index (T-VASI)
Lasso di tempo: 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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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Total-Vitiligo Area Scoring Index (T-VASI)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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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Collaboratori e investigatori
Investigatori
- Investigatore principale: Yunqing Ren, PhD, The Children's Hospital of Zhejiang University School of Medicine
Studiare le date dei record
Studia le date principali
Inizio studio (Stimato)
Completamento primario (Stimato)
Completamento dello studio (Stimato)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- 2026-IEC-0016
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