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This study is a prospective, multicenter, real-world observational study to assess the clinical efficacy and safety of topical 1.5% Ruxolitinib phosphate cream used in combination with systemic corticosteroids in a real-world clinical setting. The study plans to observe patients aged 12 years and older with active, progressive non-segmental vitiligo involving the face. All treatments are prescribed based on standard routine clinical care and medical practice guidelines. Participants will apply Ruxolitinib cream twice daily to affected skin areas for up to 24 weeks alongside a standard corticosteroid regimen. The primary goal of the study is to evaluate how many patients achieve a 75% or greater improvement in their facial vitiligo patches after 12 weeks of combined treatment. Safety and side effects will also be closely monitored throughout the 24-week period.
This prospective, multicenter, real-world cohort study plans to enroll 300 patients with active, progressive non-segmental vitiligo. Following routine medical diagnosis and standard-of-care clinical decisions, patients will receive treatment combining topical 1.5% Ruxolitinib phosphate cream applied twice daily (maximum 2 tubes/200g per month) for up to 24 weeks with concurrent corticosteroid therapy. Corticosteroid regimens consist of either intramuscular Compound Betamethasone injection (1ml once monthly) or Dexamethasone oral low-dose pulse therapy (2.25mg single dose once daily on Saturdays and Sundays) for a maximum duration of 24 weeks.The sample size of 300 patients is mathematically powered to test a superiority hypothesis. While historical single-center real-world data for ruxolitinib cream monotherapy demonstrated a 12-week response rate of 24.7%, this study establishes a conservative historical target control threshold baseline (P0) of 24%. Assuming the real-world addition of corticosteroid pulse therapy achieves an improved true response rate (P1) of 32%, a sample size of 237 evaluable participants provides 80% statistical power (beta = 0.20) to reject the null hypothesis using a two-sided exact binomial test at a significance level of alpha = 0.05. Accounting for an expected 20% drop-out or loss-to-follow-up rate, the final enrollment target was set to 300 participants. Efficacy analyses for the primary endpoint at Week 12 will utilize Multiple Imputation methods to account for missing data under Missing at Random (MAR) assumptions.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 1.5% Ruxolitinib Phosphate Cream (Opzelura) | Drug | Applied topically twice daily (BID), maximum 2 tubes per month (100g/tube) for a total duration of 24 weeks | ||
| Corticosteroid Therapy | Drug | Either Compound Betamethasone injection (1ml, intramuscularly once a month) OR Dexamethasone oral low-dose pulse therapy (2.25mg, single dose once daily on Saturdays and Sundays) . Maximum hormone duration is 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Participants Achieving Facial Vitiligo Area Scoring Index 75 (F-VASI 75) | The percentage of patients achieving a ≥ 75% improvement from baseline in the Facial Vitiligo Area Scoring Index (F-VASI) score. F-VASI measures facial depigmentation using the Palmar method (1% Body Surface Area (BSA) corresponds to one hand surface area of the participant) across facial anatomical regions with a total score range of 0 to 3. Higher scores represent greater depigmentation. Missing primary endpoint data will be handled via Multiple Imputation based on Missing at Random (MAR) assumptions. | Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Participants Achieving F-VASI 50 and F-VASI 90 | Percentage of participants achieving a ≥50% or ≥90% improvement from baseline in the Facial Vitiligo Area Scoring Index (F-VASI). | Weeks 4, 8, 12, and 24 |
| Proportion of Participants Achieving F-VASI 75 at Remaining Timepoints |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Drug Reactions (ADRs) | Assessment of total safety events, with a specific focus on application-site reactions such as acne and pruritus. | Throughout the study period (Up to Week 24) |
| Proportion of Participants Discontinuing Treatment or Withdrawing Due to Adverse Events |
Inclusion Criteria:
Clinical signs including trichrome vitiligo, confetti-like depigmentation, or Koebner phenomenon.
The extent of lesions under Wood's lamp is larger than that visible to the naked eye.
Exclusion Criteria:
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Males and females aged 12 and older presenting with active, progressive non-segmental vitiligo with facial involvement and a total body surface area (BSA) depigmentation under 10%. Subjects are selected via a non-probability clinical recruitment strategy.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| ShanShan Li | Contact | +86 18201346463 | lishanshan@cms.net.cn |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Second People's Hospital of Huai'an | Huai'an | Jiangsu | China |
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Percentage of participants achieving a ≥75% improvement from baseline in the Facial Vitiligo Area Scoring Index (F-VASI). |
| Weeks 4, 8, and 24 |
| Proportion of Participants Achieving T-VASI 50, T-VASI 75, and T-VASI 90 | Percentage of participants achieving a ≥50%, ≥75%, or ≥90% improvement from baseline in the Total Vitiligo Area Scoring Index (T-VASI). | Weeks 4, 8, 12, and 24 |
| Change From Baseline in Vitiligo Disease Activity (VIDA) Score | Evaluation of the mean change in disease progression activity using the VIDA score ranking scale. | Weeks 4, 8, 12, and 24 |
| Change From Baseline in Facial Body Surface Area (F-BSA) and Total Body Surface Area (T-BSA) | Evaluation of the mean changes in percentage values for facial and total body surface area affected by vitiligo. | Weeks 4, 8, 12, and 24 |
| Change From Baseline in F-VASI and T-VASI Scores | Continuous absolute scoring changes from baseline evaluation across both indices. | Weeks 4, 8, 12, and 24 |
| Proportion of Participants Achieving a Vitiligo Noticeability Scale (VNS) Score of 4 or 5 | Percentage of participants rating their lesions as 4 ("a lot less noticeable") or 5 ("no longer noticeable") on the patient-reported VNS scale, alongside individual category breakdowns. | Weeks 4, 8, 12, and 24 |
Evaluation of tolerability based on safety discontinuation metrics. |
| Throughout the study period (Up to Week 24) |
| The Fourth Affiliated Hospital of Soochow University | Suzhou | Jiangsu | China |
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| Affiliated Hospital of Xuzhou Medical University | Xuzhou | Jiangsu | China |
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| Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine | Shanghai | Shanghai Municipality | China |
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| Changzhou First People's Hospital | Changzhou | China |
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| Dongying People's Hospital | Dongying | China |
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| The First People's Hospital of Lianyungang | Lianyungang | China |
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| Nanjing Drum Tower Hospital | Nanjing | China |
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| U-Family Clinic (Nanjing) Co., Ltd. | Nanjing | China |
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| Yueyang Hospital of Integrated Traditional Chinese and Western Medicine, Shanghai University of Traditional Chinese Medicine | Shanghai | China |
| Shenzhen Hospital, Southern Medical University | Shenzhen | China |
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| The First Affiliated Hospital of Soochow University | Suzhou | China |
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| ID | Term |
|---|---|
| C540383 | ruxolitinib |
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