Oral Tacrolimus vs Dexamethasone Micro-pulse Therapy in Pediatric Rapidly Progressing Vitiligo: A Multicenter RCT

Oral Tacrolimus Capsule Versus Dexamethasone Micro-pulse Therapy for the Treatment of Rapidly Progressing Vitiligo in Children: A Multicenter, Randomized, Controlled Study

This clinical study aims to compare the safety and effectiveness of two treatments-oral Tacrolimus capsules and Dexamethasone micro-pulse therapy-in children aged 4-12 years with rapidly progressing vitiligo. The study is a multicenter, randomized, controlled trial involving 90 participants, who will be divided equally into two groups. One group will receive daily Tacrolimus, while the other will take Dexamethasone on weekends. Over 24 weeks, doctors will monitor improvements in skin repigmentation, side effects, and overall health through regular check-ups and blood tests. The goal is to determine which treatment better controls disease progression and improves quality of life for children with vitiligo.

Key Points:

• For children with rapidly spreading vitiligo.
• Compares two common medications.
• Follows participants for 6 months.
• Focuses on safety and effectiveness.

Study Overview

• Status: Recruiting
• Conditions: Child; Progressive Disease; Vitiligo
• Intervention / Treatment: Drug: Tacrolimus; Drug: Dexamethasone

Detailed Description

Background:

Vitiligo is an autoimmune skin condition causing pigment loss, significantly impacting children's well-being. Current treatments like systemic corticosteroids (e.g., Dexamethasone) carry risks of long-term side effects. Tacrolimus, an immunosuppressant with a safer profile in other pediatric conditions, shows promise but lacks evidence for oral use in vitiligo. This trial addresses this gap by comparing Tacrolimus and Dexamethasone.

Study Design:

• Multicenter, randomized, controlled trial across 5 hospitals in China.
• 90 participants (4-12 years) with rapidly progressing non-segmental vitiligo (VIDA score 4).
• Interventions:
• Tacrolimus group: 0.1±0.05 mg/kg/day, divided into two doses.
• Dexamethasone group: 0.05±0.025 mg/kg/weekend pulse dosing.
• Duration: 24 weeks with follow-ups at 4, 8, 12, 16, 20, and 24 weeks.

Outcome Measures:

• Primary: Proportion achieving ≥50% improvement in Vitiligo Area Scoring Index (VASI 50) at 24 weeks.
• Secondary: VASI 75/90 response rates, Investigator Global Assessment (IGA) scores, and safety parameters (blood tests, metabolic panels, adverse events).

Statistical Analysis:

Data will be analyzed using chi-square tests to compare efficacy and safety between groups (significance: p ≤ 0.05). All analyses adhere to intention-to-treat principles.

Ethics & Compliance:

Approved by the Ethics Committee of the First Affiliated Hospital of Air Force Medical University. Informed consent is obtained from all participants' guardians.

• Study Type: Interventional
• Enrollment (Estimated): 90
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Zhe Jian, Associate Professor; Phone Number: +862984775406; Email: 13571826086@163.com

Study Locations

• China
  • Shaanxi
    • Xi'an, Shaanxi, China, 710032
      • Recruiting
      • Xijing Hospital
      • Contact: Zhe Jian, Associate Professor; Phone Number: +862984775406; Email: 13571826086@163.com
      • Contact: Yucheng Sun; Phone Number: +8618215408401; Email: 814572592@qq.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Children aged 4-12 years diagnosed with rapidly progressive non-segmental vitiligo (VIDA score ≥4, indicating disease progression within the past 6 weeks).

Total body surface area (BSA) affected by vitiligo between 1% and 50%. Guardians provide written informed consent for the child's participation.

Exclusion Criteria:

• Stable-phase childhood vitiligo. Segmental, mucosal, undetermined, or generalized vitiligo. Systemic immunosuppressive therapy within the past 4 weeks. Known hypersensitivity to tacrolimus, other macrolide drugs, or study drug excipients.

Comorbidities precluding oral tacrolimus use (e.g., severe hepatic/renal dysfunction).

Obesity or systemic diseases (e.g., tuberculosis, acute/chronic infections, hypertension, congenital cardiovascular disease).

Any condition deemed by investigators to increase participant risk or interfere with trial execution.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tacrolimus Treatment Group; Participants in this arm receive oral tacrolimus therapy following the specified dosage and administration schedule in the study protocol for treating rapidly progressive vitiligo in children.	Drug: Tacrolimus; Participants receive oral tacrolimus capsules at a dosage of 0.1 ± 0.05 mg/kg per day, divided into two administrations. The treatment duration is 24 weeks. Blood drug concentration is monitored to maintain trough levels between 7-15 ng/mL. Safety assessments include regular blood tests (hematology, liver/kidney function, blood glucose) and adverse event tracking.
Active Comparator: Dexamethasone Comparison Group; Participants in this arm receive oral dexamethasone as the comparative intervention, administered according to the study protocol for evaluating its efficacy and safety in treating rapidly progressive vitiligo in children.	Drug: Dexamethasone; Participants receive oral dexamethasone tablets at a dosage of 0.05 ± 0.025 mg/kg per day, administered as a single dose on weekends (Saturday and Sunday). The treatment duration is 24 weeks. Safety evaluations include monitoring of blood parameters (hematology, liver/kidney function, blood glucose), weight, and adrenal function (cortisol, ACTH levels).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Achieving VASI 50 Response	VASI (Vitiligo Area and Severity Index) 50 response is defined as a 50% or greater reduction in VASI score from baseline. Assessment is conducted by trained dermatologists using standardized VASI scoring criteria.	24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Vitiligo Area and Severity Index (VASI) Score	Calculate the difference between the VASI score at 24 weeks and the baseline VASI score. This reflects the improvement in vitiligo area and severity over the treatment period.	24 weeks
Proportion of Participants Achieving Investigator Global Assessment (IGA) Score Improvement	Assess participants' IGA scores at 24 weeks. Participants with an IGA score of "mild" or better (score ≤ 2) are defined as achieving improvement, and the proportion is calculated.	24 weeks
Incidence of Treatment-Emergent Adverse Events	Record all adverse events (e.g., gastrointestinal symptoms, metabolic abnormalities) occurring during the 24-week treatment period. Calculate the incidence rate and analyze their relationship with the intervention.	Throughout the 24-week treatment period

Collaborators and Investigators

• Sponsor: Xijing Hospital
• Investigators: Principal Investigator: Zhe Jian, Associate Professor, First Affiliated Hospital of Air Force Medical University

Study record dates

Study Major Dates

• Study Start (Actual): April 1, 2025
• Primary Completion (Estimated): April 1, 2026
• Study Completion (Estimated): April 1, 2026

Study Registration Dates

• First Submitted: March 23, 2025
• First Submitted That Met QC Criteria: March 23, 2025
• First Posted (Actual): March 28, 2025

Study Record Updates

• Last Update Posted (Actual): June 19, 2025
• Last Update Submitted That Met QC Criteria: June 16, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Keywords: Safety; Efficacy; Children; Tacrolimus; Dexamethasone; Multicenter Study; Vitiligo; Immunosuppressive Therapy; Randomized Controlled Trialy; VASI Score; IGA Score
• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Skin Diseases; Hypopigmentation; Pigmentation Disorders; Disease Progression; Vitiligo; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anti-Inflammatory Agents; Antiemetics; Autonomic Agents; Peripheral Nervous System Agents; Gastrointestinal Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Enzyme Inhibitors; Calcineurin Inhibitors; Dexamethasone; Tacrolimus
• Other Study ID Numbers: XJPF-LCY-V202421

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No