Efficacy and Safety of Ruxolitinib Cream in Chinese Children Aged 2-11 Years With Non-segmental Vitiligo

Официальное название

Efficacy and Safety of Ruxolitinib Cream in Children Aged 2-11 Years With Non-segmental Vitiligo: A Single-Center, Real-World Study

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат Ruxolitinib 1.5% Cream BID
  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 (t

Первичные конечные точки

• Total-Vitiligo Area Scoring Index (T-VASI) [Срок оценки: 24 week]

Вторичные конечные точки (5)

• Total-Vitiligo Area Scoring Index (T-VASI) [Срок оценки: Weeks 4, 8, 12]
• Vitiligo Noticeability Scale (VNS) [Срок оценки: Weeks 4, 8, 12, 24]
• Total Body Surface Area (T-BSA) [Срок оценки: Weeks 4, 8, 12, 24]
• Children's Dermatology Life Quality Index (CDLQI) [Срок оценки: Weeks 4, 8, 12, 24]
• Pediatric Quality of Life Inventory (PedsQL) score [Срок оценки: Weeks 4, 8, 12, 24]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Children's Hospital, Zhejiang University School of Medicine — Ханчжоу

Идентификаторы

NCT: NCT07595939 · 2026-IEC-0016

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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