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

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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Non-Segmental Vitiligo (NSV)
• Intervention / Treatment: Drug: 1.5% Ruxolitinib Phosphate Cream (Opzelura); Drug: Corticosteroid Therapy

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.

• Study Type: Observational
• Enrollment (Estimated): 300

Contacts and Locations

• Study Contact: Name: ShanShan Li; Phone Number: +86 18201346463; Email: lishanshan@cms.net.cn

Study Locations

• China
  • Changzhou, China
    • Changzhou First People's Hospital
    • Contact: Yu Hua Yang; Phone Number: +86 15995044892; Email: manyinyan@163.com
  • Dongying, China
    • Dongying People's Hospital
    • Contact: Hai Ying Wang; Phone Number: +86 15504562533; Email: whydf001@163.com
  • Lianyungang, China
    • The First People's Hospital of Lianyungang
    • Contact: Wen Long Hu; Phone Number: +86 18961325126; Email: lyghuwl@163.com
  • Nanjing, China
    • Nanjing Drum Tower Hospital
    • Contact: Jun Bao; Phone Number: +86 13327809366; Email: baojun@sina.com
  • Nanjing, China
    • U-Family Clinic (Nanjing) Co., Ltd.
    • Contact: Hong Jia; Phone Number: +86 13512516585; Email: jhff2015@163.com
  • Shanghai, China
    • Yueyang Hospital of Integrated Traditional Chinese and Western Medicine, Shanghai University of Traditional Chinese Medicine
  • Shenzhen, China
    • Shenzhen Hospital, Southern Medical University
    • Contact: Yanhua Liang; Phone Number: +86 18665086898; Email: liangdoctor@163.com
  • Suzhou, China
    • The First Affiliated Hospital of Soochow University
    • Contact: Yu Qian Zhu; Phone Number: +86 18136088888; Email: zhuyueqian0711@126.com
  • Jiangsu
    • Huai'an, Jiangsu, China
      • The Second People's Hospital of Huai'an
      • Contact: Chunsheng Yang; Phone Number: +86 18752409168; Email: 8yung@sina.com
    • Suzhou, Jiangsu, China
      • The Fourth Affiliated Hospital of Soochow University
      • Contact: Xuejun Zhang; Phone Number: +86 13722781112; Email: ayzxj@vip.sina.com
    • Xuzhou, Jiangsu, China
      • Affiliated Hospital of Xuzhou Medical University
      • Contact: Guan Jiang; Phone Number: +86 13013941798; Email: dr.guanjiang@gmail.com
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China
      • Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
      • Contact: Pan Meng; Phone Number: +86 13601698537; Email: pm_ClinicalTrial@126.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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.

Description

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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Secondary Outcome Measures

Outcome Measure	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	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

Other Outcome Measures

Outcome Measure	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	Evaluation of tolerability based on safety discontinuation metrics.	Throughout the study period (Up to Week 24)

Collaborators and Investigators

• Sponsor: ShanShan Li

Study record dates

Study Major Dates

• Study Start (Estimated): June 20, 2026
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: June 23, 2026
• First Submitted That Met QC Criteria: June 23, 2026
• First Posted (Actual): June 29, 2026

Study Record Updates

• Last Update Posted (Actual): June 29, 2026
• Last Update Submitted That Met QC Criteria: June 23, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: Ruxolitinib phosphate cream, Corticosteroid therapy, Multicenter study, Real-world study, Active Vitiligo
• Additional Relevant MeSH Terms: ruxolitinib
• Other Study ID Numbers: Opzelura-CHN-IIT-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No