Ruxolitinib Cream for Vitiligo

Recruiting at 93 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?

The purpose of this study is to to evaluate the safety and efficacy of ruxolitinib cream in pediatric participants with nonsegmental 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 Ruxolitinib Cream safe for humans?

Ruxolitinib cream, also known as Opzelura, has been approved for use in treating vitiligo in people aged 12 and older. Common side effects include acne, itching, cold symptoms, headache, urinary tract infection, redness at the application site, and fever. ^{1 2 3 4 5}

How is ruxolitinib cream different from other vitiligo treatments?

Ruxolitinib cream is unique because it is a topical treatment that works by inhibiting Janus kinase 1 and 2 (proteins involved in inflammation), which helps in repigmenting the skin in people with vitiligo. Unlike some other treatments, it is applied directly to the skin, making it a non-invasive option. ^{1 5 6 7 8}

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

Research shows that Ruxolitinib Cream, a topical treatment, has been effective in repigmenting skin in people with vitiligo, as demonstrated in clinical trials. It has been approved for use in patients aged 12 and older, showing promising results in improving skin color. ^{1 2 5 7 9}

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 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]

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]

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 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]

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]

Citations

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

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

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

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

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

New Indication for Topical Ruxolitnib. [2023]

6. England pubmed.ncbi.nlm.nih.gov

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

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

Off-label Studies on the Use of Ruxolitinib in Dermatology. [2021]

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

Two Phase 3, Randomized, Controlled Trials of Ruxolitinib Cream for Vitiligo. [2023]

9. England pubmed.ncbi.nlm.nih.gov

Rapid pruritus reduction with ruxolitinib cream treatment in patients with atopic dermatitis. [2023]