A Phase 3, Randomized, Double-Blind, Safety, and Efficacy Study ofRuxolitinib Cream in Pediatric Participants With Nonsegmental Vitiligo
David Rosmarin
Primary Investigator
Brief description of study
Detailed description of study
Approximately 180 participants will be randomized 1:1 to receive ruxolitinib 1.5% cream BID or
vehicle cream BID and stratified by age (2 to ≤ 6 and 7 to ≤ 11 years) and baseline F-BSA (0.5%
to < 0.75% versus ≥ 0.75%).
Participants (or parent/guardian) will apply study cream to depigmented vitiligo areas on the face
and body up to 10% BSA for 24 weeks (see Figure 1).
Participants who complete 24 weeks of treatment with no safety concerns will continue into the
28-week, open-label extension period to be treated with ruxolitinib 1.5% cream BID. The total
treated area should not exceed 10% BSA (facial and nonfacial).
During the treatment-extension period, if vitiligo areas become significantly more extensive, the
participant should contact the study site to discuss whether evaluation at the clinic is required.
After a visit to document the additional BSA, the participant (or parent/guardian) may apply
cream to the new areas up to a BSA of 10%. If BSA exceeds 10%, the participant and
investigator can determine which additional areas can be treated.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Nonsegmental vitiligo
-
Age: 2 years - 11 years
-
Gender: All
Inclusion Criteria:
- 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 from screening through 30 days after the last application of study cream.
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).
- 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.
- Current or previous use of JAK inhibitors, systemic or topical.
- 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.
