Clinical trial NCT06804811: A Study to Evaluate the Efficacy and Safety of Ruxolitinib Cream in Children (6 to < 12 Years Old) With Nonsegmental Vitiligo
Official title: A Phase 3, Randomized, Double-Blind, Efficacy, and Safety Study of Ruxolitinib Cream in Children (6 to < 12 Years Old) With Nonsegmental Vitiligo
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: NonSegmental Vitiligo
Keywords: NonSegmental Vitiligo
pediatric
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2025-02-03
Enrollment: 250
Sex: ALL
Minimum age (years): 6
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy and safety of ruxolitinib cream in children (6 to \< 12 years old) with nonsegmental vitiligo.


Eligibility criteria: Inclusion Criteria:

* Aged 6 to \< 12 years at the time of signing the ICF.
* Clinical diagnosis of nonsegmental vitiligo with depigmented area including ≥ 0.5% BSA on the face, ≥ 0.5 F-VASI, ≥ 3% BSA on nonfacial areas, ≥ 3 T-VASI.
* Total body vitiligo area does not exceed 10% BSA.
* Pigmented hair within some of the areas of vitiligo on the face.
* Must agree to discontinue all agents used to treat vitiligo from screening through the final safety follow-up visit.
* For sexually active participants (except participants who are prepubescent) willingness to avoid pregnancy or fathering a child as defined in the protocol.

Exclusion Criteria:

* Diagnosis of other forms of vitiligo (eg, segmental).
* Other differential diagnosis of vitiligo or other skin depigmentation disorders (eg, piebaldism, pityriasis alba, leprosy, postinflammatory hypopigmentation, progressive macule hypomelanosis, nevus anemicus, chemical leukoderma, and tinea versicolor).
* Any other skin disease that, in the opinion of the investigator, would interfere with the study drug application or study assessments.
* Prior or current use of depigmentation treatments (eg, monobenzone).
* Concurrent conditions and history of protocol-defined diseases
* Any serious illness or medical, physical, or psychiatric condition(s) that, in the investigator's opinion, would interfere with full participation in the study, including application of study cream and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data.
* Use of protocol-defined treatments within the indicated washout period before baseline.
* History of treatment failure with any systemic or topical JAK inhibitor for vitiligo or any other inflammatory condition.
* Protocol-defined clinically significant abnormal laboratory values at screening.
* BMI-for-age \< 5th percentile or ≥ 85th percentile according to the CDC BMI Percentile Calculator for Child and Teen.
* Pregnant or lactating participants or those considering pregnancy during the period of their study participation.
* In the opinion of the investigator, unable or unlikely to comply with the application schedule and study evaluations.
* Living with anyone participating in any current Incyte-sponsored ruxolitinib cream study.
* Employees of the sponsor or investigator or are otherwise dependents of them.
* Known allergy or reaction to any component of the study cream formulation.
* The following participants are excluded in France: vulnerable populations according to article L.1121-6 of the French Public Health Code and adults under legal protection, or who are unable to express their consent per article L.1121-8 of the French Public Health Code, not affiliated to a social security per article L.1121-8-1 of the French Public Health code.
* In the EU, participants considered incapacitated according to EU CTR No. 536/2014 Articles 2 and 31.

Other protocol-defined Inclusion/Exclusion Criteria may apply.

Primary outcomes:
measure: Proportion of Participants Achieving a ≥ 75% Improvement From Baseline in the Facial Vitiligo Area Scoring Index (F-VASI75)
timeFrame: Week 24
Secondary outcomes:
measure: Proportion of Participants Achieving Facial Vitiligo Area Scoring Index (F-VASI50)
timeFrame: Week 24
measure: Proportion of Participants Achieving Facial Vitiligo Area Scoring Index (F-VASI90)
timeFrame: Week 24
measure: Proportion of Participants Achieving a ≥ 50% Improvement From Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50)
timeFrame: Week 24
measure: Percentage change from baseline in F-BSA
timeFrame: Week 24
measure: Number of Treatment Emergent Adverse Events (TEAEs)
timeFrame: Up to Week 52 and 30 days
measure: Proportion of participants achieving a Vitiligo Noticeability Scale (VNS) score of 4 (a lot less noticeable) or 5 (no longer noticeable)
timeFrame: Week 24 and Week 52
measure: Proportion of participants in each category for the color-matching question
timeFrame: Week 24 and Week 52
measure: Proportion of participants who report facial assessment of Patient Global Impression of Change-Vitiligo (F-PaGIC-V) of very much improved or much improved
timeFrame: Week 24 and Week 52
measure: Proportion of participants who report total body assessment of Patient Global Impression of Change-Vitiligo (T-PaGIC-V) of very much improved or much improved
timeFrame: Week 24 and Week 52
measure: Change from baseline in Psychosocial Impact Questionnaire for Pediatric Patients Who Have Nonsegmental Vitiligo (VIT-PIQ)
timeFrame: Week 24 and Week 52
measure: Change from baseline in Children's Dermatology Life Quality Index (CDLQI)
timeFrame: Week 24 and Week 52
measure: Change from baseline in Patient Global Impression of Bother - Vitiligo (PGIB-V)
timeFrame: Week 24 and Week 52
measure: Mean satisfaction score
timeFrame: Week 24 and Week 52
measure: Pharmacokinetic (PK) of Ruxolitinib: Trough concentrations
timeFrame: Weeks 6, 24, 30 and 52
Other outcomes:
Locations:
Study officials:
affiliation: Incyte Corporation
name: Incyte Medical Monitor
role: STUDY_DIRECTOR
References: