Nonsegmental Vitiligo With Genital Involvement
Conditions
Keywords
Vitiligo, Genital Vitiligo, INCB18424, Ruxolitinib Cream
Brief summary
An open-label study in which participants with non-segmental vitiligo with genital involvement will apply ruxolitinib 1.5% cream twice a day (BID) to all depigmented areas (up to 10% BSA) for up to 48 weeks. Participants should continue to treat depigmented areas identified for treatment at baseline regardless of whether the area begins to improve or fully repigment.
Interventions
DRUGRuxolitinib Cream
Ruxolitinib cream will be applied twice daily for upto 48 weeks
Sponsors
Incyte Corporation
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Clinical diagnosis of nonsegmental vitiligo with genital involvement * At least ≥ 0.25% BSA of nonsegmental vitiligo in the genital area. * Pigmented hair within some of the genital vitiligo areas. * At least 1 genital target lesion that is ≥ 0.1% BSA that has a pigmented hair within it. * Vitiligo on areas of the body besides the genitals. * Total body vitiligo area not exceeding 10% BSA. * Willing to have genital photography conducted. * Must agree to discontinue all agents used to treat vitiligo from screening through the final safety follow-up visit. Over-the-counter preparations deemed acceptable by the investigator and camouflage makeups are permitted.
Exclusion criteria
* Other forms of vitiligo (eg, segmental) or other differential diagnosis of vitiligo or other skin depigmentation disorders. * Prior or current use of depigmentation treatments (eg, monobenzone). * Active or recurrent genital warts or herpes. * Male participants with partners with known current/active cervical intraepithelial neoplasia or anal intraepithelial neoplasia. * An active sexually transmitted disease, sexually transmitted infection, or other skin disorder affecting the genital area (eg, scabies, fungal infection, molluscum). * Had ≥ 3 laser hair removal treatments in an area to be treated for vitiligo. * Any serious illness or medical, physical, or psychiatric condition(s) that, in the investigator's opinion, would interfere with full participation in the study or interpretation of study data. * Clinical laboratory test results outside of protocol defined ranges * Pregnant or lactating participants, or those considering pregnancy during the period of their study participation.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Achieving a Genital Vitiligo Noticeability Scale (VNS) of "4 - A Lot Less Noticeable" or "5 - No Longer Noticeable" at Week 48 | Baseline; Week 48 | The VNS is a patient-reported measure of vitiligo treatment success that has a 5-point scale. The participant was asked to respond to the following query: "Compared with before treatment, how noticeable is the vitiligo now? Responses: (1) more noticeable, (2) as noticeable, (3) slightly less noticeable, (4) a lot less noticeable, or (5) no longer noticeable. The baseline genital photograph was shown to the participant for reference. A VNS score of 4 or 5 can be interpreted as representing treatment success. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | up to approximately 14 months | An adverse event (AE) is any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered drug related. An AE can therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study cream. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug and within 30 days of the last administration of study drug. |
| Percentage of Participants Achieving Genital-Physician Global Vitiligo Assessment (PhGVA) of 0 or 1 at Week 48 | Week 48 | The severity of genital vitiligo was assessed by the physician using the PhGVA, which has a 5-point scale: 0=clear, no signs of vitiligo or complete/near complete repigmentation; 1=almost clear, mostly pigmented areas with small depigmented or difficult to repigment areas (e.g., shaft of penis or labia minora); 2=mild disease, modest areas of depigmentation with approximately 50% pigmentation within vitiligo areas or significant perifollicular pattern present; 3=moderate disease, large areas of depigmented vitiligo areas/significant depigmentation within vitiligo areas; 4=severe disease, extensive areas of vitiligo with complete depigmentation. Response was reported for the genital region (Genital-PhGVA). |
| Change From Baseline in Affected Body Surface Area (BSA) in the Genital Region at Weeks 24 and 48 | Baseline; Weeks 24 and 48 | BSA assessments were performed using the palmar method and was estimated to the nearest 0.1%. The approximate size of the participant's entire palmar surface (i.e., the palm plus 5 digits) was considered as 1% BSA, and the approximate size of the participant's thumb was considered as 0.1% BSA. Total BSA takes into account the depigmented areas for each of the following body regions: head/neck (including face and scalp), upper extremities (including axillae), hands, trunk (including genitalia), lower extremities (including buttocks), and feet. The BSA depigmented by vitiligo was assessed for the genitals (which represents approximately 1% of the trunk). Genital BSA takes into account the depigmented areas of the labia majora, labia minora, and perineum in females and the penis, scrotum, and perineum in males. Change from baseline was calculated as the post-baseline value minus the baseline value. |
| Percentage of Participants Achieving ≥50% Improvement in Total Body Vitiligo Area Scoring Index (T-VASI50) at Weeks 24 and 48 | Baseline; Weeks 24 and 48 | A T-VASI50 responder achieved ≥50% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). |
| Percentage of Participants Achieving ≥75% Improvement in Total Body Vitiligo Area Scoring Index (T-VASI75) at Weeks 24 and 48 | Baseline; Weeks 24 and 48 | A T-VASI75 responder achieved ≥75% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). |
| Percentage of Participants Achieving ≥90% Improvement in Total Body Vitiligo Area Scoring Index (T-VASI90) at Weeks 24 and 48 | Baseline; Weeks 24 and 48 | A T-VASI90 responder achieved ≥90% improvement from Baseline in T-VASI, calculated with contributions from 6 sites. The percentage of vitiligo involvement was estimated in hand units (percentage of BSA estimated to the nearest 0.1%) by the Investigator using the Palmar Method. The Investigator used his/her hand to mimic the participant's hand size to evaluate percent BSA vitiligo involvement. The degree of depigmentation for each site was estimated to the nearest percentage: 0% (no depigmentation present), 10% (only specks of depigmentation present), 25% (pigmented area exceeded depigmented area), 50% (depigmented and pigmented area was equal), 75% (depigmented area exceeded pigmented area), 90% (specks of pigment present), 100% (no pigment present). T-VASI was then derived by multiplying the values assessed for the vitiligo involvement by the percentage of affected skin for each site and summing the values (range: 0-100; lower scores indicate increased improvement). |
| Percentage of Participants Achieving a Genital VNS of "4 - A Lot Less Noticeable" or "5 - No Longer Noticeable" at Week 24 | Baseline; Week 24 | The VNS is a patient-reported measure of vitiligo treatment success that has a 5-point scale. The participant was asked to respond to the following query: "Compared with before treatment, how noticeable is the vitiligo now? Responses: (1) more noticeable, (2) as noticeable, (3) slightly less noticeable, (4) a lot less noticeable, or (5) no longer noticeable. The baseline genital photograph was shown to the participant for reference. A VNS score of 4 or 5 can be interpreted as representing treatment success. |
| Percentage of Participants in Each Category of the Color-matching Question at Weeks 24 and 48 | Baseline; Weeks 24 and 48 | Participants were shown their baseline genital photograph for reference, and were asked to respond to the following query: "At this point of your treatment, how well does your genital skin color match between your treated vitiligo skin and normal skin?: Responses: (1) excellent, (2) very good, (3) good, (4) poor, or (5) very poor. |
Countries
Canada, France, United States
Participant flow
Pre-assignment details
This study was conducted at 12 study centers in Canada, France, and the United States.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 41.9 years STANDARD_DEVIATION 13.77 |
| Race/Ethnicity, Customized Asian | 9 Participants |
| Race/Ethnicity, Customized Asian, White | 1 Participants |
| Race/Ethnicity, Customized Black/African-American | 2 Participants |
| Race/Ethnicity, Customized Colombian | 1 Participants |
| Race/Ethnicity, Customized Egyptian | 1 Participants |
| Race/Ethnicity, Customized Indian | 1 Participants |
| Race/Ethnicity, Customized Native Hawaiian/Pacific Islander | 1 Participants |
| Race/Ethnicity, Customized Not Reported | 5 Participants |
| Race/Ethnicity, Customized Persian | 1 Participants |
| Race/Ethnicity, Customized Unknown | 1 Participants |
| Race/Ethnicity, Customized White | 26 Participants |
| Sex: Female, Male Female | 20 Participants |
| Sex: Female, Male Male | 29 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 49 |
| other Total, other adverse events | 0 / 49 |
| serious Total, serious adverse events | 1 / 49 |
Outcome results
None listed