Ruxolitinib Cream for Vitiligo

Recruiting at 99 trial locations

Overseen By Incyte Corporation Call Center (ex-US)

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Incyte Corporation

Must not be taking: Depigmentation treatments, JAK inhibitors

Disqualifiers: Other vitiligo, Skin diseases, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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: 24 weeks

180 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a cream called ruxolitinib to determine its safety and effectiveness in treating nonsegmental vitiligo in children. Vitiligo causes patches of skin to lose color. Participants will receive either the ruxolitinib cream or a placebo (a cream with no active ingredient) to compare results. Ideal candidates for this trial have nonsegmental vitiligo, including noticeable patches on the face and other body parts, and are willing to stop other vitiligo treatments during the study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for vitiligo.

Will I have to stop taking my current medications?

The trial requires participants to stop using all treatments for vitiligo from the start of the study until the final safety follow-up visit.

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

Studies have shown that ruxolitinib cream is generally well tolerated, with most people not experiencing serious side effects. Some users might notice mild reactions at the application site, such as redness or itchiness. Importantly, significant side effects affecting the whole body have not been reported.

Research indicates that the cream is safe for treating vitiligo, a condition that causes skin to lose its color. While the safety of ruxolitinib cream for children under 12 with vitiligo is still under investigation, it has been used safely for other skin conditions like atopic dermatitis.

Overall, ruxolitinib cream has demonstrated a good safety profile, meaning it is unlikely to cause harm when used as directed. ^{1 2 3 4 5}

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

Ruxolitinib cream is unique because it targets the Janus kinase (JAK) pathway, which plays a key role in the immune response related to vitiligo. Unlike current treatments like corticosteroids or calcineurin inhibitors, which can have significant side effects with long-term use, ruxolitinib cream may offer a more targeted approach with potentially fewer side effects. Researchers are excited about this treatment because it could provide a more effective and safer option for repigmenting skin affected by vitiligo.

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

Research has shown that ruxolitinib cream can effectively treat vitiligo, a condition where the skin loses its color. In this trial, participants will receive either ruxolitinib 1.5% cream or a vehicle cream without active medication. Two studies found that people using ruxolitinib cream experienced more skin color return than those using a cream without medicine. Specifically, about 30% of people using ruxolitinib cream saw a 75% improvement in their facial vitiligo after six months. This suggests that ruxolitinib cream could be a promising option for restoring skin color in people with vitiligo. ^{6 7 8 9 10}

Who Is on the Research Team?

Incyte Medical Monitor

Principal Investigator

Incyte Corporation

Are You a Good Fit for This Trial?

This trial is for children with a type of skin condition called nonsegmental vitiligo, which causes loss of skin color in patches. They should have it on at least 0.5% of their face or 3% on other body parts but not more than 10% overall. Participants must stop using other vitiligo treatments during the study and if sexually active, avoid pregnancy.

Inclusion Criteria

I agree to stop all vitiligo treatments during the study.

I agree to prevent pregnancy or fathering a child during and 30 days after the study.

I have vitiligo with significant skin depigmentation on my face and body.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ruxolitinib 1.5% cream or vehicle cream applied topically to the affected area

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

28 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ruxolitinib Cream

Trial Overview

The study tests Ruxolitinib cream to see if it's safe and effective for treating pediatric nonsegmental vitiligo compared to a placebo (Vehicle Cream). It will involve applying these creams to see if they can restore skin color in affected areas.

How Is the Trial Designed?

Group I: Ruxolitinib 1.5 % Cream Experimental Treatment 1 Intervention

Participants received ruxolitinib 1.5% cream, applied topically to the affected area as defined in the protocol.

Group II: Vehicle Cream Placebo Group 1 Intervention

Participants received vehicle cream, applied topically to the affected area as defined by the protocol.

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

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 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]

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]

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]

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]

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.

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.

2.

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 ...

3.

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.

4.

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.

5.

sciencedirect.com

sciencedirect.com/science/article/pii/S2405857223000438

Do the benefits of Opzelura (Ruxolitinib) outweigh ...

The 30% of patients who received Opzelura showed 75% betterment in facial vitiligo [1]. In a randomized control phase, 2 trials for Ruxolitinib cream on 157 ...

6.

opzelura.com

opzelura.com/vitiligo/safety-side-effects

Safety & Side Effects

It is not known if OPZELURA is safe and effective in children less than 12 years of age with nonsegmental vitiligo. OPZELURA is for use on the skin only.

7.

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 ...

8.

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.

9.

accessdata.fda.gov

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

FULL PRESCRIBING INFORMATION - accessdata.fda.gov

OPZELURA® (ruxolitinib) cream, for topical use. Initial U.S. ... The safety and effectiveness of OPZELURA for the topical treatment of nonsegmental vitiligo.

10.

healio.com

healio.com/news/dermatology/20240430/safety-data-show-ruxolitinib-cream-well-tolerated-in-atopic-dermatitis-vitiligo-at-1-year

Safety data show ruxolitinib cream well tolerated in atopic ...

Safety data show ruxolitinib cream well tolerated in atopic dermatitis, vitiligo at 1 year. April 30, 2024. 1 min read. ByGabrielle M. Grasso.