Ruxolitinib Cream Combined With Corticosteroids for Progressive Non-Segmental Vitiligo: A Multicenter Real-World Study

Официальное название

Efficacy and Safety of Ruxolitinib Phosphate Cream Combined With Corticosteroids in Patients With Progressive Non-Segmental Vitiligo: A Multicenter Real-World Study

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат 1.5% Ruxolitinib Phosphate Cream (Opzelura)
  Applied topically twice daily (BID), maximum 2 tubes per month (100g/tube) for a total duration of 24 weeks
• Препарат Corticosteroid Therapy
  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

Первичные конечные точки

• Proportion of Participants Achieving Facial Vitiligo Area Scoring Index 75 (F-VASI 75) [Срок оценки: Week 12]

Вторичные конечные точки (7)

• Proportion of Participants Achieving F-VASI 50 and F-VASI 90 [Срок оценки: Weeks 4, 8, 12, and 24]
• Proportion of Participants Achieving F-VASI 75 at Remaining Timepoints [Срок оценки: Weeks 4, 8, and 24]
• Proportion of Participants Achieving T-VASI 50, T-VASI 75, and T-VASI 90 [Срок оценки: Weeks 4, 8, 12, and 24]
• Change From Baseline in Vitiligo Disease Activity (VIDA) Score [Срок оценки: Weeks 4, 8, 12, and 24]
• Change From Baseline in Facial Body Surface Area (F-BSA) and Total Body Surface Area (T-BSA) [Срок оценки: Weeks 4, 8, 12, and 24]
• Change From Baseline in F-VASI and T-VASI Scores [Срок оценки: Weeks 4, 8, 12, and 24]
• Proportion of Participants Achieving a Vitiligo Noticeability Scale (VNS) Score of 4 or 5 [Срок оценки: Weeks 4, 8, 12, and 24]

Критерии участия

Критерии включения

• Age ≥ 12 years at the time of signing informed consent.
• Clinical diagnosis of non-segmental vitiligo with facial involvement, and total body surface area (BSA) depigmentation ≤ 10%.
• Diagnosed with progressive/active vitiligo meeting at least one of the following: Vitiligo Disease Activity (VIDA) score ≥ 3.

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.

• Agrees to have no pregnancy plans and uses effective contraception during the study and up to 4 weeks after the last dose.
• Voluntarily signs the informed consent form and agrees to regular follow-up visits.

Критерии исключения

• Diagnosis of other clinical forms of vitiligo (e.g., segmental vitiligo).
• Clinically diagnosed with stable vitiligo.
• Co-existing skin depigmentation disorders affecting efficacy evaluation (e.g., pityriasis alba, leprosy, post-inflammatory hypopigmentation, progressive macular hypomelanosis, nevus anemicus, chemical leukoderma, tinea versicolor).
• Leukoderma lesions with more than 1/3 white hair, or lesions accompanied by white hair where the investigator assesses the probability of benefit from participating is low based on clinical experience.
• Contraindications to systemic corticosteroid use.
• Known hypersensitivity or allergy to the study medications or excipients.
• Known end-stage renal disease (ESRD) or currently undergoing renal replacement therapy (e.g., dialysis).
• Concurrent participation in other clinical studies.
• Any other condition which, in the investigator's judgment, makes the patient unsuitable for the study

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Китай · 12 центров

• The Second People's Hospital of Huai'an — Huai'an
• The Fourth Affiliated Hospital of Soochow University — Сучжоу
• Affiliated Hospital of Xuzhou Medical University — Xuzhou
• Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine — Шанхай
• Changzhou First People's Hospital — Changzhou
• Dongying People's Hospital — Dongying
• The First People's Hospital of Lianyungang — Lianyungang
• Nanjing Drum Tower Hospital — Нанкин
• … и ещё 4 центра

Идентификаторы

NCT: NCT07673640 · Opzelura-CHN-IIT-001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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