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.
調査の概要
詳細な説明
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.
研究の種類
入学 (推定)
段階
- フェーズ 3
連絡先と場所
研究連絡先
- 名前：Yunqing Ren, PhD
- 電話番号：0571-86670281
- メール：yqren@zju.edu.cn
研究連絡先のバックアップ
- 名前：Jipeng Liu, PhD
- 電話番号：0571-86670281
- メール：liujipeng@zju.edu.cn
研究場所
-
-
Zhejiang
-
Hangzhou、Zhejiang、中国、310052
- Children's Hospital, Zhejiang University School of Medicine
-
コンタクト：
- Yunqing Ren
- 電話番号：0571-86670281
- メール：yqren@zju.edu.cn
-
主任研究者：
- Yunqing Ren
-
-
参加基準
適格基準
就学可能な年齢
- 子
健康ボランティアの受け入れ
説明
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.
研究計画
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：なし
- 介入モデル：単一グループの割り当て
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：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.
|
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.
他の名前：
|
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Total-Vitiligo Area Scoring Index (T-VASI)
時間枠：24 week
|
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).
|
24 week
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Total-Vitiligo Area Scoring Index (T-VASI)
時間枠：Weeks 4, 8, 12
|
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).
|
Weeks 4, 8, 12
|
|
Vitiligo Noticeability Scale (VNS)
時間枠：Weeks 4, 8, 12, 24
|
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)
時間枠：Weeks 4, 8, 12, 24
|
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)
時間枠：Weeks 4, 8, 12, 24
|
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
時間枠：Weeks 4, 8, 12, 24
|
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.
|
Weeks 4, 8, 12, 24
|
協力者と研究者
捜査官
- 主任研究者：Yunqing Ren, PhD、The Children's Hospital of Zhejiang University School of Medicine
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
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