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

55 Participants Needed

Trial Summary

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.

What data supports the effectiveness of the drug Ruxolitinib Cream for treating vitiligo?

Research shows that Ruxolitinib Cream, a topical treatment, is effective for vitiligo, a skin condition causing loss of color. It has been approved for use in people aged 12 and older, with studies demonstrating its ability to help repigment the skin. ^{1 2 3 4 5}

Is Ruxolitinib Cream safe for treating vitiligo?

Ruxolitinib cream, approved for treating vitiligo in people aged 12 and older, is generally safe but may cause side effects like acne, itching, common cold, headache, urinary tract infection, redness, and fever at the application site. ^{1 3 4 5 6}

What makes ruxolitinib cream unique for treating vitiligo?

Ruxolitinib cream is unique because it is a topical treatment specifically approved for non-segmental vitiligo, working as a selective JAK1/2 inhibitor to help repigment the skin, unlike other treatments that have limited efficacy and are often used off-label. ^{1 2 4 5 6}

What is the purpose of this trial?

This trial is testing a special cream called Ruxolitinib, sometimes combined with light therapy, on teenagers and adults with a type of vitiligo that affects less than 10% of their skin. The cream works by calming the immune system in the skin, and the light therapy helps improve skin color. The cream has shown repigmentation effects in previous studies for vitiligo.

Research Team

Haq Nawaz, MD

Principal Investigator

Incyte Corporation

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

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]

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]

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]

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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Review of Ruxolitinib for Treatment of Non-Segmental Vitiligo. [2023]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Baseline Levels of Circulating Inflammatory Biomarkers Stratify Patients with Vitiligo Who Significantly Repigment after Treatment with Ruxolitinib Cream. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

New Indication for Topical Ruxolitnib. [2023]

4. England pubmed.ncbi.nlm.nih.gov

Ruxolitinib cream for treatment of vitiligo: a randomised, controlled, phase 2 trial. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Topical ruxolitinib: A new treatment for vitiligo. [2023]

6. United States pubmed.ncbi.nlm.nih.gov

A Review on the Use of Topical Ruxolitinib for the Treatment of Vitiligo. [2023]