Ruxolitinib Cream + Phototherapy for Vitiligo

Overseen By Incyte Corporation Call Center (US)

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Incyte Corporation

Must not be taking: Depigmentation treatments, JAK inhibitors

Disqualifiers: Segmental vitiligo, Thromboembolic events, Skin infections, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 12

Duration: 48 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness of Ruxolitinib cream, both alone and with light therapy, for individuals with non-segmental vitiligo. Non-segmental vitiligo causes skin to lose color in patches, and the study targets those with affected areas covering no more than 10% of their body. Participants will use the cream alone or with narrow-band ultraviolet B (NB-UVB) light therapy if additional improvement is needed after 12 weeks. This trial suits those with visible vitiligo on their face and body who have not found success with other recent treatments. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

You will need to stop using any treatments specifically for vitiligo during the study, but over-the-counter products approved by the study team and camouflage makeups are allowed. The protocol does not specify about other medications, so it's best to discuss with the study team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ruxolitinib cream is generally safe for treating vitiligo. Most people experience no serious side effects. Common side effects include mild skin reactions at the application site. Serious side effects, such as infections or blood clots, are rare.

When combined with narrow-band ultraviolet B (NB-UVB) light therapy, the treatment remains well tolerated. Studies indicate that this combination does not increase side effects compared to using the cream alone. Overall, this treatment appears safe for many individuals with non-segmental vitiligo. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike standard treatments for vitiligo, which often include topical steroids or calcineurin inhibitors, ruxolitinib cream offers a new approach by targeting the JAK-STAT pathway to reduce skin depigmentation. Researchers are excited about ruxolitinib because it addresses the underlying immune response, potentially offering more effective and sustained results. Additionally, the combination with narrow-band ultraviolet B (NB-UVB) phototherapy in certain cases could enhance treatment efficacy, providing a tailored approach based on individual response. This makes ruxolitinib a promising option for those who haven't seen significant improvements with existing therapies.

What evidence suggests that this trial's treatments could be effective for vitiligo?

Research has shown that ruxolitinib cream effectively treats vitiligo. In studies, about 50.3% of patients experienced a noticeable return of color to their vitiligo patches after using the cream for a year. In this trial, participants in Group A will initially apply ruxolitinib cream and may receive narrow-band ultraviolet B (NB-UVB) phototherapy if they show less than 25% improvement by week 12. This combination ranks among the most effective new treatments for vitiligo, as the light treatment can enhance the cream's effectiveness, particularly for those who don't see enough improvement with the cream alone. Participants in Group B will continue with ruxolitinib cream alone if they achieve sufficient improvement. Overall, ruxolitinib cream, with or without light treatment, offers a promising option for managing vitiligo. ^{6 7 8 9 10}

Who Is on the Research Team?

Haq Nawaz, MD

Principal Investigator

Incyte Corporation

Are You a Good Fit for This Trial?

This trial is for adolescents and adults with non-segmental vitiligo, where the affected area doesn't cover more than 10% of their body. Participants should have a certain level of facial and body depigmentation. They must stop other vitiligo treatments during the study and can't join if they have skin infections, other skin diseases, serious health issues, no pigmented hair in vitiligo areas on the face, used certain past treatments or had bad reactions to phototherapy.

Inclusion Criteria

You have a skin condition called nonsegmental vitiligo with a large depigmented area.

You have a score of 0.5 or higher on the F-VASI scale for facial skin condition.

You have large areas of vitiligo on your body, not including your face.

Exclusion Criteria

You have a current bacterial, fungal, or viral skin infection.

You have a skin condition that might make it hard to use the study cream or affect the study evaluations.

Any serious illness or medical, physical, or psychiatric condition(s) that pose a significant risk to the participant; or interfere with interpretation of study data

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants apply ruxolitinib cream with or without phototherapy. Phototherapy is added at Week 12 for those with less than 25% improvement.

48 weeks

Weekly visits for phototherapy from Week 12 to Week 48

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• NB-UVB Phototherapy
• Ruxolitinib Cream

Trial Overview

The trial tests Ruxolitinib cream's effectiveness and safety when used alone or combined with NB-UVB phototherapy for treating non-segmental vitiligo. It focuses on participants whose condition affects less than 10% of their body surface area.

How Is the Trial Designed?

Group I: Group B: Ruxolitinib Monotherapy Experimental Treatment 1 Intervention

Participants will apply ruxolitinib 1.5% cream BID as monotherapy. Participants who have ≥ T-VASI25 at Week 12 will continue on ruxolitinib 1.5% cream BID alone.

Group II: Group A: Ruxolitinib + Narrow-Band Ultraviolet B Phototherapy (NB-UVB) Experimental Treatment 2 Interventions

Participants will initially apply ruxolitinib 1.5%mg cream as a monotherapy. At week 12, those who have \< 25% improvement in total body Vitiligo Area Scoring Index (T-VASI25) will have NB-UVB phototherapy added to their ruxolitinib 1.5% cream BID regimen. NB-UVB will be given 3 times per week starting at Week 12 through Week 48 (36 weeks). For participants who receive combination therapy, NB-UVB machines will be supplied by the sponsor for at home use during the study.

Ruxolitinib Cream is already approved in United States, European Union for the following indications:

Approved in United States as Opzelura for:

• Atopic Dermatitis
• Vitiligo

Approved in European Union as Jakavi for:

• Myelofibrosis
• Polycythaemia vera
• Steroid-refractory acute graft-versus-host disease
• Chronic graft-versus-host disease
• Non-segmental vitiligo

Who Is Running the Clinical Trial?

Incyte Corporation

Lead Sponsor

Trials

408

Recruited

66,800+

Steven Stein

Incyte Corporation

Chief Medical Officer since 2015

MD from University of Witwatersrand

Hervé Hoppenot

Incyte Corporation

Chief Executive Officer since 2014

MBA from ESSEC Business School

Published Research Related to This Trial

In a phase 2 study involving 157 adults with vitiligo, ruxolitinib cream demonstrated significant efficacy, with 45% of patients using the 1.5% twice daily dose achieving at least a 50% improvement in facial vitiligo area by week 24, compared to only 3% in the control group.

The treatment was well tolerated, with most adverse events being mild to moderate, suggesting that ruxolitinib cream could be a safe and effective option for repigmentation in vitiligo patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial. Rosmarin, D., Pandya, AG., Lebwohl, M., et al.[2021]

In a phase 2 trial involving patients with vitiligo, those using 1.5% ruxolitinib cream showed a significant improvement in facial Vitiligo Area Scoring Index scores, with a mean improvement of 79.9% at week 24 compared to only 1.1% in those who did not respond.

Proteomic analysis revealed distinct inflammatory biomarker profiles between patients who achieved at least a 50% improvement and those who did not, suggesting that these biomarkers could help identify which patients are more likely to benefit from ruxolitinib treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Baseline Levels of Circulating Inflammatory Biomarkers Stratify Patients with Vitiligo Who Significantly Repigment after Treatment with Ruxolitinib Cream. Howell, MD., Kuo, FI., Rumberger, B., et al.[2023]

In two 52-week phase 3 trials, 52% of patients treated with ruxolitinib 1.5% cream experienced at least a 75% improvement in their vitiligo, demonstrating its efficacy as a treatment.

Ruxolitinib is the first FDA-approved topical medication for repigmentation in vitiligo patients, showing it is both safe and effective, although its cost may limit accessibility for some patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Review on the Use of Topical Ruxolitinib for the Treatment of Vitiligo. Grossmann, MC., Haidari, W., Feldman, SR.[2023]

Topical ruxolitinib, a selective JAK1/2 inhibitor, has been approved for treating non-segmental vitiligo in patients over 12 years old, showing promising efficacy based on phase III clinical trials (TRuE-V1 and TRuE-V2).

The 1.5% ruxolitinib cream is considered effective for vitiligo treatment, offering a localized therapy option that may help avoid the long-term side effects associated with systemic treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Topical ruxolitinib: A new treatment for vitiligo. Tavoletti, G., Avallone, G., Conforti, C., et al.[2023]

Topical ruxolitinib cream has been shown to significantly improve vitiligo symptoms, with 58% of patients achieving a 50% improvement in Vitiligo Area Scoring Index (VASI) after 52 weeks of treatment, making it an effective option for repigmentation.

The treatment has a favorable safety profile, with only mild adverse effects reported, such as erythema and pruritus, and it offers significant clinical improvements compared to traditional therapies, which may have more severe side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Review of Ruxolitinib for Treatment of Non-Segmental Vitiligo. Hwang, JR., Driscoll, MS.[2023]

Ruxolitinib (Opzelura) has been approved by the FDA as a topical treatment for nonsegmental vitiligo in individuals aged 12 and older, indicating its efficacy for this skin condition.

The most common side effects reported include application site acne, itching, and redness, as well as other mild issues like headaches and urinary tract infections, suggesting that while generally safe, users should be aware of these potential reactions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New Indication for Topical Ruxolitnib. Aschenbrenner, DS.[2023]

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40156697/?utm_source=FeedFetcher&utm_medium=rss&utm_campaign=None&utm_content=1R3QZIpFgeaXQJt8K9w8_oRDFOUkgIho18PKxDpWTR1UFZLoNX&fc=None&ff=20250331013223&v=2.18.0.post9+e462414

Efficacy and Safety of Ruxolitinib Cream in Vitiligo by ...

Results: The TRuE-V studies enrolled 674 patients. Week 52 F-VASI75 was achieved by 50.3% (176/350) of patients who applied ruxolitinib cream ...

2.

investor.incyte.com

investor.incyte.com/news-releases/news-release-details/incyte-announces-results-phase-3-clinical-trials-evaluating

Incyte Announces Results of Phase 3 Clinical Trials ...

Significant itch improvements were observed with ruxolitinib cream 1.5% versus vehicle control at Day 7 (22.4% vs 8.0%; P=0.0064), with ...

3.

opzelura.com

opzelura.com/vitiligo/clinical-trial-results

Repigmentation Results

75% improvement to vitiligo areas on the face was achieved at 6 months in 29.9% of patients using OPZELURA in 2 separate studies compared with 7.5% and 12.9% of ...

4.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12033138/

Efficacy and Safety of Ruxolitinib Cream in Vitiligo by ...

These results show that ruxolitinib cream is an effective and safe option to treat vitiligo across a diverse population of patients.

5.

nejm.org

nejm.org/doi/full/10.1056/NEJMoa2118828

Two Phase 3, Randomized, Controlled Trials of Ruxolitinib ...

In two phase 3 trials, application of ruxolitinib cream resulted in greater repigmentation of vitiligo lesions than vehicle control through 52 weeks.

6.

opzelura.com

opzelura.com/vitiligo/safety-side-effects

Safety & Side Effects - Vitiligo

OPZELURA may cause serious side effects including serious infections, cancer and immune system problems, blood clots, low blood cell counts, and increases in ...

7.

opzelurahcp.com

opzelurahcp.com/vitiligo/adverse-event-profile

safety data through 2 years 1 - Opzelura HCP

Serious lower respiratory tract infections were reported in the clinical development program with topical ruxolitinib. No cases of active tuberculosis (TB) were ...

8.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC10866801/

Real-World Use of Ruxolitinib Cream: Safety Analysis at 1 ...

Postmarketing safety data from the year following approval suggest ruxolitinib cream is generally well tolerated, without significant systemic ...

9.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/label/2025/215309s007lbl.pdf

OPZELURA® (ruxolitinib) cream, for topical use

The safety and effectiveness of OPZELURA for the topical treatment of nonsegmental vitiligo have been established in pediatric patients ages 12 years and older.

10.

ema.europa.eu

ema.europa.eu/en/medicines/human/EPAR/opzelura

Opzelura | European Medicines Agency (EMA)

In terms of safety, the side effects of Opzelura are considered acceptable. While ruxolitinib medicines taken by mouth are associated with serious side effects, ...