Ruxolitinib Cream for Vitiligo

No longer recruiting at 15 trial locations

Overseen By Incyte Corporation Call Center (ex-US)

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Incyte Corporation

Must not be taking: Depigmentation treatments

Disqualifiers: Other vitiligo, Genital warts, STDs, 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: Unknown

Duration: 48 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a cream called ruxolitinib, a topical treatment, to determine its effectiveness for people with non-segmental vitiligo, a condition causing skin color loss, particularly in the genital area. Participants will apply the cream to all affected skin spots twice daily for up to 48 weeks, even if repigmentation occurs. Ideal candidates have vitiligo on the genitals and other body parts but cover no more than 10% of their body. Participants should also have some pigmented hair in the affected genital areas and must stop other vitiligo treatments during the study. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

You will need to stop using any treatments specifically for vitiligo during the trial, but over-the-counter products approved by the study doctor and camouflage makeup are allowed.

Is there any evidence suggesting that ruxolitinib cream is likely to be safe for humans?

Research has shown that ruxolitinib cream is generally safe for treating skin conditions like vitiligo. Studies have found it to be well-tolerated, with few serious side effects. Mild skin reactions at the application site occur in about 1% of users. Importantly, no major side effects affecting the entire body have been reported, indicating the cream does not impact other areas. Overall, the safety profile appears favorable for those considering this treatment. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for vitiligo?

Ruxolitinib cream is unique because it offers a new approach to treating vitiligo by targeting the JAK-STAT pathway, which plays a role in the immune system's attack on skin pigment cells. Unlike standard treatments, such as corticosteroids and calcineurin inhibitors, which aim to suppress the immune response more broadly, ruxolitinib specifically inhibits the signaling pathways that lead to depigmentation. Researchers are excited about this cream because it has the potential to be more effective and safer for long-term use, particularly for sensitive areas like the genitals, by minimizing the risk of skin thinning associated with traditional treatments.

What evidence suggests that ruxolitinib cream might be an effective treatment for vitiligo?

Research has shown that ruxolitinib cream effectively treats non-segmental vitiligo. In clinical trials, about 30% of participants using the cream saw a 75% improvement in affected facial areas within six months. After one year, nearly 75% experienced at least a 50% improvement, with about 30% achieving a 90% improvement. The cream helps restore skin color and reduces the visibility of patches. This treatment offers promising results for those seeking to improve the appearance of vitiligo. ^{6 7 8 9 10}

Are You a Good Fit for This Trial?

This trial is for individuals with non-segmental vitiligo affecting the genital area, covering no more than 10% of their body. Participants must have at least one target lesion in the genital area with pigmented hair and be willing to undergo genital photography. Pregnant or breastfeeding individuals, those considering pregnancy, or people with other skin conditions are excluded.

Inclusion Criteria

I have vitiligo on parts of my body other than the genitals.

I agree to stop all vitiligo treatments except for certain over-the-counter products and makeup.

Less than 10% of my body is affected by vitiligo.

Exclusion Criteria

I do not have any active STDs or skin disorders affecting my genital area.

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

I currently have or recently had genital warts or herpes.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants apply ruxolitinib 1.5% cream twice a day to depigmented areas for up to 48 weeks

48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ruxolitinib Cream

Trial Overview The study tests Ruxolitinib Cream applied twice daily to depigmented areas including genitals for up to 48 weeks. The goal is to assess its safety and effectiveness in restoring pigment. It's an open-label trial where all participants know they're using this specific cream.

How Is the Trial Designed?

Group I: Ruxolitinib Cream Experimental Treatment 1 Intervention

Participants with non-segmental vitiligo with genital involvement will receive ruxolitinib 1.5% cream BID for up to 48 weeks

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]

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 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]

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]

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]

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]

Ruxolitinib, a Janus kinase inhibitor approved for myelofibrosis and polycythemia vera, shows promising efficacy in treating dermatologic conditions like atopic dermatitis, psoriasis, and vitiligo, based on data from clinical trials.

The evidence for ruxolitinib's effectiveness in alopecia areata is mixed, varying between topical and oral formulations, indicating the need for further research to clarify its role in treating various skin disorders.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Off-label Studies on the Use of Ruxolitinib in Dermatology. Tegtmeyer, K., Ravi, M., Zhao, J., et al.[2021]

In two phase 3 trials involving 1249 patients with atopic dermatitis, ruxolitinib cream showed rapid and significant reductions in itch, with over 16% of patients experiencing a 2-point reduction within 12 hours compared to only 6.9% with the vehicle cream.

By Day 2, a greater percentage of patients using ruxolitinib cream achieved a 4-point reduction in itch severity, with sustained improvements observed over the 8-week treatment period, indicating both efficacy and a quick onset of action.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rapid pruritus reduction with ruxolitinib cream treatment in patients with atopic dermatitis. Blauvelt, A., Kircik, L., Papp, KA., et al.[2023]

In two phase 3 trials involving 674 patients with nonsegmental vitiligo, ruxolitinib cream demonstrated significant efficacy in repigmenting skin lesions, with 29.8% and 30.9% of patients achieving at least a 75% improvement in facial depigmentation compared to 7.4% and 11.4% in the vehicle control groups, respectively.

While ruxolitinib cream was effective, it was associated with common adverse effects such as application-site acne and pruritus, occurring in over half of the patients, indicating the need for further studies to assess long-term safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Two Phase 3, Randomized, Controlled Trials of Ruxolitinib Cream for Vitiligo. Rosmarin, D., Passeron, T., Pandya, AG., et al.[2023]

Citations

1. 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 ...

2. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK618261/

Ruxolitinib cream for treating non-segmental vitiligo in ... - NCBI Clinical trial evidence shows that ruxolitinib cream increases repigmentation and reduces how noticeable vitiligo patches are compared with a ...

3. ajmc.com ajmc.com/view/efficacy-of-ruxolitinib-cream-in-nonsegmental-vitiligo-confirmed-in-real-world-study

Efficacy of Ruxolitinib Cream in Nonsegmental Vitiligo ... Ruxolitinib cream 15 mg/g provided rapid, meaningful repigmentation and improved quality of life for adults with nonsegmental vitiligo.

4. nejm.org nejm.org/doi/full/10.1056/NEJMoa2118828

Two Phase 3 Trials of Ruxolitinib Cream for Vitiligo In two phase 3 trials, application of ruxolitinib cream resulted in greater repigmentation of vitiligo lesions than vehicle control through 52 weeks.

5. investor.incyte.com investor.incyte.com/news-releases/news-release-details/incyte-announces-52-week-data-phase-3-true-v-program-evaluating

Incyte: 52-Wk Data from TRuE-V for Ruxolitinib Cream in Vitiligo At Week 52, approximately 75% of patients achieved ≥50% improvement in F-VASI (F-VASI50), and nearly one-third (approximately 30%) achieved ≥90% improvement in ...

6. opzelurahcp.com opzelurahcp.com/vitiligo/adverse-event-profile

safety data through 2 years 1 - Opzelura HCP Efficacy and safety of ruxolitinib cream for the treatment of vitiligo: week 24 pooled analysis of the TRuE-V phase 3 studies. Presented at the American ...

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

Real-World Use of Ruxolitinib Cream: Safety Analysis at 1 ... Ruxolitinib cream is generally well tolerated, without significant systemic AEs, and with a low incidence of application site reactions.

8. opzelura.com opzelura.com/vitiligo/safety-side-effects

Safety & Side Effects - Vitiligo Two Years of Safety Data. At six months, the most common side effects (occurring in ≥1% or more of patients) observed in patients who used OPZELURA were: ...

9. incytemi.com incytemi.com/document/Poster/Fall%20Clinical%202025_Bunick%20CG_Rux%20Cream%20Integrated%20Safety%20Analysis.pdf

Integrated Safety Analysis of Ruxolitinib Cream A comparison of the risk of major cardiocascular events, venous thromboembolism, serious infections, and malignancies among patients with ...

10. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/full/10.1002/ski2.404

Safety and efficacy of ruxolitinib cream for the treatment ... Safety and efficacy of ruxolitinib cream for the treatment of vitiligo: A randomised controlled trial secondary analysis at 3 years