Efficacy and Safety Evaluation of Ruxolitinib Cream in Pediatric Patients Aged 6 to <12 Years with Nonsegmental Vitiligo: A Phase 3 Randomized, Double-Blind Study
- Trial ID
- 2024-513171-41-00
- Protocol
- INCB 18424-309
- Sponsor
- Incyte Corp.
Trial statistics
Diseases & Conditions
Objectives
The primary objective of this study is to evaluate the **efficacy** of ruxolitinib cream in pediatric participants with nonsegmental vitiligo. This is clinically relevant as nonsegmental vitiligo is a chronic skin condition characterized by the loss of skin pigmentation, which can significantly impact the quality of life and psychological well-being of affected children. Effective treatment options are limited, and assessing the efficacy of ruxolitinib cream could provide a new therapeutic avenue for managing this condition.
Secondary objectives include:
- To further assess the efficacy of ruxolitinib cream.
- To evaluate the safety and tolerability of ruxolitinib cream.
- To determine the effect of ruxolitinib cream on participant quality of life (QOL).
- To determine participant satisfaction with treatment.
- To evaluate the pharmacokinetics (PK) of ruxolitinib cream.
Participants
The clinical trial involves a total of **48 participants** diagnosed with **nonsegmental vitiligo**, specifically focusing on pediatric subjects. The study population comprises both male and female participants aged between 6 to less than 12 years. Participants were selected based on their ability to comprehend and willingness to sign an informed consent form, or through a legally designated representative, with assent from the participant when possible. The trial includes individuals with a clinical diagnosis of nonsegmental vitiligo, characterized by specific depigmented areas on the body. Participants must agree to discontinue all agents used to treat vitiligo during the study period, although over-the-counter preparations deemed acceptable by the investigator and camouflage makeups are permitted. The trial population includes a vulnerable group, as it involves children. The study does not specify any particular lifestyle considerations such as diet or physical activity. Key inclusion criteria include having a total body vitiligo area not exceeding 10% body surface area and the presence of pigmented hair within some vitiligo areas on the face. The trial aims to evaluate the efficacy of ruxolitinib cream in this specific population.
Plans and Procedures
The clinical trial is designed to evaluate the **efficacy** and safety of **ruxolitinib** cream in pediatric participants aged 6 to less than 12 years with nonsegmental **vitiligo**. This is a Phase 3, randomized, double-blind, controlled study. Participants will be randomly assigned to receive either the active treatment, **ruxolitinib** cream, or a vehicle (placebo) cream. The trial is expected to last until October 2027, with recruitment starting in September 2025. The maximum treatment period for participants is 52 weeks.
The study involves several key visits. The inclusion visit, or screening, will determine eligibility based on criteria such as age, clinical diagnosis of nonsegmental vitiligo, and agreement to discontinue other treatments for vitiligo. Participants must have a total body vitiligo area not exceeding 10% body surface area (BSA) and pigmented hair within some vitiligo areas on the face. Follow-up visits will occur at regular intervals to assess the primary endpoint, which is the proportion of participants achieving F-VASI75 at Week 24. Secondary endpoints include various measures of vitiligo improvement and safety assessments, such as changes in vital signs and laboratory data. The end-of-study visit will evaluate the long-term effects of the treatment and collect final data.
Participant involvement is expected to last up to 52 weeks, with conditions for early termination including withdrawal of consent, adverse events, or non-compliance with the study protocol. The study aims to provide comprehensive data on the safety and efficacy of **ruxolitinib** cream in treating pediatric nonsegmental vitiligo, contributing valuable insights into potential therapeutic options for this condition.
Treatment
The clinical trial involves the use of **Ruxolitinib (INCB018424) cream**, an experimental medication formulated as a cream for **cutaneous use**. The active substance in this medication is **ruxolitinib**, a chemical entity developed by Incyte Corporation. The cream is applied topically with a maximum daily dose of 8.5 grams and a total maximum dose of 3120 grams over a treatment period of up to 52 weeks. The cream is not a pediatric formulation, and its role in the trial is to evaluate its efficacy and safety in children aged 6 to less than 12 years with nonsegmental vitiligo.
In addition to the experimental treatment, a **vehicle (placebo) cream** is used as a comparator in the study. This placebo cream does not contain any active pharmaceutical ingredients and serves as a control to assess the efficacy of the ruxolitinib cream. The placebo is also applied topically, following the same administration schedule as the experimental cream, to ensure consistency in the trial's methodology.
Efficacy
The efficacy of Ruxolitinib cream in the treatment of nonsegmental vitiligo in pediatric participants will be assessed through a series of primary and secondary endpoints. The primary endpoint is the proportion of participants achieving a **F-VASI75** (Facial Vitiligo Area Scoring Index 75) at Week 24. Secondary endpoints include the proportion of participants achieving F-VASI50 and F-VASI90 at Week 24, as well as T-VASI50 (Total Vitiligo Area Scoring Index 50) at the same time point. Additionally, the percentage change from baseline in F-BSA (Facial Body Surface Area) at Week 24 will be evaluated.
Further secondary endpoints involve assessments of adverse events (AEs) through changes in vital signs, clinical evaluations, height, weight, and laboratory data. The study will also measure the proportion of participants achieving a VNS (Vitiligo Noticeability Scale) score of 4 or 5 at Weeks 24 and 52, and the proportion of participants in each category for the color-matching question at these time points. Patient-reported outcomes will be collected, including F-PaGIC-V and T-PaGIC-V scores, indicating very much improved or much improved conditions at Weeks 24 and 52. Changes from baseline in VIT-PIQ, CDLQI, and PGIB-V will be assessed at Weeks 24 and 52, along with mean satisfaction scores. Preapplication plasma concentrations of ruxolitinib will be measured at Weeks 4, 24, 40, and 52.
Inclusion and Exclusion Criteria
Inclusion Criteria
- Ability to comprehend and willingness to sign an ICF or written informed consent of the legally designated representative and a verbal or written assent from the participant when possible
- 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 (facial and nonfacial) 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 safety follow-up visit. Over-the-counter preparations deemed acceptable by the investigator and camouflage makeups are permitted as per Section 6.6.1.
- With the exception of participants who are prepubescent, willingness to avoid pregnancy or fathering children based on the criteria below. Permitted methods in preventing pregnancy (see Appendix A) should be communicated to the participants and their understanding confirmed. a. Male participants with reproductive potential must agree to take appropriate precautions to avoid fathering children from screening through 90 days (a spermatogenesis cycle) after the last application of study cream. b. Female participants who have reached menarche must have a negative urine pregnancy test at screening and before the first application of study cream on Day 1 (if the interval between screening and Day 1 is > 2 weeks) and must agree to take appropriate precautions to avoid pregnancy from screening through 30 days (1 menstrual cycle) 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 disorder (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 study cream application or study assessments
- Prior or current use of depigmentation treatments (eg, monobenzone)
- Concurrent conditions and history of other diseases as follows: a. Immunocompromised (eg, lymphoma, acquired immunodeficiency syndrome, Wiskott-Aldrich syndrome). b. History of malignant disease within 5 years before the Day 1 visit, except for adequately treated, nonmetastatic, nonmelanoma, skin cancer. c. Current and/or history of thrombosis, including deep venous thrombosis and pulmonary embolism. d. Current and/or history of liver disease, including known hepatitis B or C virus infection, with hepatic or biliary abnormalities. e. Current and/or history of HIV infection. f. Current and/or history of active tuberculosis; or current and/or history of latent tuberculosis unless adequately treated.
- 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. Examples include but are not limited to the following: a. Chronic or acute infection requiring treatment with systemic antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungals within 2 weeks before the Day 1 visit. b. Active acute bacterial, fungal, or viral skin infection (eg, herpes simplex, herpes zoster, chickenpox, clinically infected AD, impetigo) within 1 week before the Day 1 visit. c. Clinically significant or uncontrolled cardiovascular disease, including unstable angina, acute myocardial infarction, or stroke within 6 months before the Day 1 visit; New York Heart Association Class III or IV congestive heart failure; or arrhythmia requiring therapy or uncontrolled hypertension (blood pressure > 150/90 mmHg), unless approved by the medical monitor/sponsor. d. On maintenance dialysis. e. Committed to an institution by virtue of an order issued by either judicial or administrative authorities
- Use of any of the following treatments within the indicated washout period before Day 1: a. 1 week: Topical drugs (eg, corticosteroids, calcineurin and phosphodiesterase type 4 inhibitors, and retinoids) when used on the vitiligo areas. b. 2 weeks: Immunizations with live-attenuated vaccines. Note: Live-attenuated vaccines are not recommended during the double-blind, vehicle-controlled period. c. 4 weeks: − Melanocyte-stimulating agents (eg, afamelanotide). − Immunomodulating systemic medications (eg, corticosteroids, methotrexate, and cyclosporine). − Any other systemic therapies that could increase the skin sensitivity to UV/visible light or impact skin pigmentation (eg, tetracyclines and methoxypsoralens). d. 8 weeks: Laser or any kind of phototherapy, including tanning bed or intentional UV exposure. e. 5 half-lives or 12 weeks, whichever is longer, for any of the following if used to treat vitiligo: Biologic agents, investigational therapies, experimental therapies, or procedures. Investigational biologic agents should be discussed with the sponsor to determine whether a longer period of discontinuation is required
- History of treatment failure with any systemic or topical JAK inhibitor (eg, ruxolitinib, tofacitinib, baricitinib, abrocitinib, and upadacitinib) for vitiligo or any other inflammatory condition
- Any of the following clinically significant, abnormal laboratory values at screening: a. Cytopenias: − Hemoglobin < 10 g/dL − Absolute neutrophil count < 1500/µL − Platelet count < 100,000/µL b. Liver function tests: − AST or ALT ≥ 2 × ULN − ALP and/or bilirubin > 1.5 × ULN (isolated bilirubin > 1.5 × ULN is acceptable if bilirubin is fractionated and direct bilirubin is < 35%) c. Estimated glomerular filtration rate < 30 mL/min/1.73 m (using the Revised (bedside) Schwartz equation). d. Clinically significant, abnormal TSH or free T4 as determined by the investigator. e. Any other clinically significant laboratory result that, in the opinion of the investigator, poses a significant risk to the participant.
- Pregnant or lactating or considering pregnancy during the period of 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
Trial Status by Country
| Country | Status | Start of Recruitment | Planned Patients |
|---|---|---|---|
 Austria | Recruiting | 15 Sept 2025 | 3 |
 Belgium | Recruiting | 15 Sept 2025 | 12 |
 Bulgaria | Recruiting | 15 Sept 2025 | 24 |
 Denmark | Recruiting | 15 Sept 2025 | 9 |
 France | Recruiting | 15 Sept 2025 | 21 |
 Germany | Recruiting | 15 Sept 2025 | 30 |
 Hungary | Recruiting | 15 Sept 2025 | 12 |
 Italy | Recruiting | 15 Sept 2025 | 27 |
 The Netherlands | Recruiting | 15 Sept 2025 | — |
 Poland | Recruiting | 15 Sept 2025 | 31 |
Sites & Investigators
Research sites
Investigators
Investigational Products
Details about the medicinal products being studied in this clinical trial.
| Product Name | Role in Trial | Formulation | Administration | Max Daily Dose | Treatment Duration | EU MP Number |
|---|---|---|---|---|---|---|
Ruxolitinib (INCB018424) cream | Test | CREAM | CUTANEOUS USE | 8.5 | 52 | PRD10399242 |
Vehicle (placebo) cream | Placebo | N/A | — | — | — | N/A |
Ruxolitinib (INCB018424) cream | Test | CREAM | CUTANEOUS USE | 8.5 | 52 | PRD12075367 |