Non-Segmental Vitiligo (NSV)
Conditions
Keywords
Ruxolitinib phosphate cream, Corticosteroid therapy, Multicenter study, Real-world study, Active Vitiligo
Brief summary
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.
Detailed description
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.
Interventions
DRUG1.5% Ruxolitinib Phosphate Cream (Opzelura)
Applied topically twice daily (BID), maximum 2 tubes per month (100g/tube) for a total duration of 24 weeks
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
Sponsors
ShanShan Li
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Participants Achieving Facial Vitiligo Area Scoring Index 75 (F-VASI 75) | Week 12 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Participants Achieving F-VASI 50 and F-VASI 90 | Weeks 4, 8, 12, and 24 | Percentage of participants achieving a ≥50% or ≥90% improvement from baseline in the Facial Vitiligo Area Scoring Index (F-VASI). |
| Proportion of Participants Achieving F-VASI 75 at Remaining Timepoints | Weeks 4, 8, and 24 | Percentage of participants achieving a ≥75% improvement from baseline in the Facial Vitiligo Area Scoring Index (F-VASI). |
| Proportion of Participants Achieving T-VASI 50, T-VASI 75, and T-VASI 90 | Weeks 4, 8, 12, and 24 | Percentage of participants achieving a ≥50%, ≥75%, or ≥90% improvement from baseline in the Total Vitiligo Area Scoring Index (T-VASI). |
| Change From Baseline in Vitiligo Disease Activity (VIDA) Score | Weeks 4, 8, 12, and 24 | Evaluation of the mean change in disease progression activity using the VIDA score ranking scale. |
| Change From Baseline in Facial Body Surface Area (F-BSA) and Total Body Surface Area (T-BSA) | Weeks 4, 8, 12, and 24 | Evaluation of the mean changes in percentage values for facial and total body surface area affected by vitiligo. |
| Change From Baseline in F-VASI and T-VASI Scores | Weeks 4, 8, 12, and 24 | Continuous absolute scoring changes from baseline evaluation across both indices. |
| Proportion of Participants Achieving a Vitiligo Noticeability Scale (VNS) Score of 4 or 5 | Weeks 4, 8, 12, and 24 | 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. |
Countries
China
Contacts
CONTACTShanShan Li
Outcome results
None listed