NonSegmental Vitiligo
Conditions
Keywords
NonSegmental Vitiligo, pediatric
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.
Interventions
DRUGRuxolitinib Cream
Ruxolitinib cream applied topically to the affected area as a thin film twice daily.
DRUGVehicle Cream
Matching vehicle cream applied topically to the affected area as a thin film twice daily.
Sponsors
Incyte Corporation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to 11 Years
Healthy volunteers
No
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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Participants Achieving a ≥ 75% Improvement From Baseline in the Facial Vitiligo Area Scoring Index (F-VASI75) | Week 24 | ≥75% improvement in facial Vitiligo Area Scoring Index. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of Participants Achieving Facial Vitiligo Area Scoring Index (F-VASI50) | Week 24 | ≥50% improvement in facial Vitiligo Area Scoring Index. |
| Proportion of Participants Achieving Facial Vitiligo Area Scoring Index (F-VASI90) | Week 24 | ≥90% improvement in facial Vitiligo Area Scoring Index. |
| Proportion of Participants Achieving a ≥ 50% Improvement From Baseline in the Total Body Vitiligo Area Scoring Index (T-VASI50) | Week 24 | ≥50% improvement in total body Vitiligo Area Scoring Index. |
| Percentage change from baseline in F-BSA | Week 24 | Facial body surface area (F-BSA) takes into account the facial depigmented areas as a percentage of the total body area. |
| Number of Treatment Emergent Adverse Events (TEAEs) | Up to Week 52 and 30 days | TEAE defined as any adverse event either reported for the first time or worsening of a pre-existing event after first dose of study drug. |
| Proportion of participants achieving a Vitiligo Noticeability Scale (VNS) score of 4 (a lot less noticeable) or 5 (no longer noticeable) | Week 24 and Week 52 | The VNS is a patient-reported measure of vitiligo treatment success that has a 5-point scale. |
| Proportion of participants in each category for the color-matching question | Week 24 and Week 52 | The color-matching question is a patient-reported measure of vitiligo treatment success that has a 5-point scale. |
| Proportion of participants who report facial assessment of Patient Global Impression of Change-Vitiligo (F-PaGIC-V) of very much improved or much improved | Week 24 and Week 52 | The F-PaGIC-V is an assessment of improvement by the participant that has a 7-point scale. |
| 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 | Week 24 and Week 52 | The T-PaGIC-V is an assessment of improvement by the participant that has a 7-point scale. |
| Change from baseline in Psychosocial Impact Questionnaire for Pediatric Patients Who Have Nonsegmental Vitiligo (VIT-PIQ) | Week 24 and Week 52 | The VIT-PIQ is a 17-item questionnaire to assess Quality of Life (QOL) of children with vitiligo. |
| Change from baseline in Children's Dermatology Life Quality Index (CDLQI) | Week 24 and Week 52 | The CDLQI is the youth/children's version of the Dermatology Life Quality Index. |
| Change from baseline in Patient Global Impression of Bother - Vitiligo (PGIB-V) | Week 24 and Week 52 | The PGIB-V is a single question with an 11-point scale ("not at all" to "a whole lot") to assess the participant's level of bother because of their vitiligo. |
| Mean satisfaction score | Week 24 and Week 52 | The treatment satisfaction tool is a fit-for-purpose questionnaire to assess treatment satisfaction in children with nonsegmental vitiligo. |
| Pharmacokinetic (PK) of Ruxolitinib: Trough concentrations | Weeks 6, 24, 30 and 52 | Trough is defined as the concentration reached by a drug immediately before the next dose is administered. |
Countries
Argentina, Austria, Belgium, Bulgaria, Denmark, France, Germany, Hungary, Italy, Netherlands, Poland, Portugal, Spain, United Kingdom
Contacts
CONTACTIncyte Corporation Call Center (US)
CONTACTIncyte Corporation Call Center (ex-US)
STUDY_DIRECTORIncyte Medical Monitor
Incyte Corporation
Outcome results
None listed