Ruxolitinib Cream for Lichen Sclerosus

Recruiting at 14 trial locations

Overseen By Incyte Corporation Call Center (ex-US)

Age: 18+

Sex: Female

Trial Phase: Phase 2

Sponsor: Incyte Corporation

Must not be taking: Antibiotics, Antivirals, Antifungals

Disqualifiers: Pregnancy, Vaginal infections, others

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

61 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a cream called Ruxolitinib on people with Lichen Sclerosus, a chronic skin condition. The cream works by blocking enzymes that cause inflammation and immune responses. The study will last for several months. Ruxolitinib cream has been tested in various conditions such as atopic dermatitis and vitiligo, showing anti-inflammatory and repigmentation effects.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot have taken systemic antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungals within 2 weeks before starting the trial.

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

Ruxolitinib cream has shown effectiveness in treating skin conditions like atopic dermatitis and lichen planus by reducing symptoms such as lesion count and severity. Its use in these conditions suggests it may also be beneficial for Lichen Sclerosus, as it works by blocking certain proteins involved in inflammation. ^{1 2 3 4 5}

Is Ruxolitinib Cream safe for use in humans?

Ruxolitinib cream has been generally well tolerated in studies for skin conditions like atopic dermatitis, with mild to moderate side effects such as occasional stinging or burning at the application site. Serious skin reactions are rare, and the cream has a favorable safety profile compared to oral forms of the medication. ^{1 2 5 6 7}

How is Ruxolitinib Cream different from other drugs for lichen sclerosus?

Ruxolitinib Cream is unique because it is a topical formulation of a Janus kinase (JAK) inhibitor, which works by blocking specific pathways involved in inflammation, unlike the standard treatment of potent corticosteroids that primarily reduce inflammation through a different mechanism. This offers a novel approach for managing lichen sclerosus, especially for patients who may not respond well to or cannot use steroids. ^{8 9 10 11 12}

Research Team

Haq Nawaz, MD

Principal Investigator

Incyte Corporation

Eligibility Criteria

This trial is for individuals with Lichen Sclerosus, specifically those who have a confirmed diagnosis in the anogenital area and are experiencing itching. Participants must not be pregnant or planning pregnancy and should not have other infections or conditions that could explain their symptoms.

Inclusion Criteria

Willingness to avoid pregnancy

Your initial IGA score is 2 or higher for LS.

You have a score of 4 or higher for itching in the anogenital area before starting the study.

Exclusion Criteria

Other exclusion criteria may apply

You do not have a specific condition affecting the anal or genital area.

You are suspected of having other conditions that cause vaginal symptoms such as yeast infection, chlamydia, trichomoniasis, gonorrhea, bacterial vaginosis, or herpes.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double-blind, Vehicle-controlled Treatment

Participants receive either Ruxolitinib cream or vehicle cream for 12 weeks

12 weeks

Open-label Extension

Participants receive Ruxolitinib cream for an additional 12 weeks

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Ruxolitinib Cream
• Vehicle Cream

Trial Overview The study is testing Ruxolitinib cream's effectiveness and safety against a placebo (vehicle cream) over a 12-week period, followed by another 12 weeks where everyone receives Ruxolitinib to further assess its effects.

Participant Groups

Group I: Ruxolitinib cream Experimental Treatment 2 Interventions

Ruxolitinib 1.5% cream BID for 12 weeks followed by ruxolitinb 1.5% cream BID (or QD) for 12-weeks in an open-label extension.

Group II: Vehicle Cream Placebo Group 1 Intervention

Vehicle cream BID for 12 weeks followed by ruxolitinb 1.5% cream BID (or QD) for 12-weeks in an open-label extension.

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

Ruxolitinib cream 1.5% significantly improved disease severity, itching, and sleep disturbance in patients aged 12 and older with mild to moderate atopic dermatitis after 8 weeks of treatment, and continued to control symptoms for an additional 44 weeks when applied as needed.

The cream was well tolerated, with a safety profile similar to that of a placebo, and infrequent local side effects, making it a promising alternative to traditional treatments like corticosteroids for managing atopic dermatitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ruxolitinib Cream 1.5%: A Review in Mild to Moderate Atopic Dermatitis. Hoy, SM.[2023]

In a phase II study involving 12 patients, topical ruxolitinib significantly reduced the total lesion count in cutaneous lichen planus by a median of 50 lesions after 8 weeks of treatment, indicating its efficacy.

Transcriptomic analysis revealed that cutaneous lichen planus is driven by interferon pathways, and the treatment with ruxolitinib led to downregulation of interferon-stimulated genes, supporting its mechanism of action in targeting this disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ruxolitinib Cream in the Treatment of Cutaneous Lichen Planus: A Prospective, Open-Label Study. Brumfiel, CM., Patel, MH., Severson, KJ., et al.[2022]

In two phase 3 studies involving 1249 patients with atopic dermatitis, ruxolitinib cream significantly improved treatment outcomes compared to a vehicle cream, with 93.4% of patients using the 0.75% formulation and 90.9% using the 1.5% formulation achieving clinically meaningful responses by week 8.

Patients who continued using ruxolitinib cream beyond 8 weeks showed progressive improvements, with over half achieving treatment success by week 52, indicating that extended therapy can provide additional benefits and that the cream was well tolerated throughout the study.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinically relevant improvements in adults and adolescents with atopic dermatitis who did not achieve Investigator's Global Assessment treatment success following 8 weeks of ruxolitinib cream monotherapy. Simpson, EL., Kircik, L., Blauvelt, A., et al.[2023]

Ruxolitinib cream, a topical treatment for atopic dermatitis, was found to be generally well tolerated in a study of 41 patients aged 12 to 65, with only 31.7% experiencing treatment-emergent adverse events, and only 9.8% reporting treatment-related adverse events.

The cream demonstrated high efficacy, with 94.6% of patients achieving at least a 75% improvement in their eczema severity after 56 days of treatment, while the mean plasma concentration of the drug remained well below levels that could cause harmful effects on bone marrow.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Maximum-Use Trial of Ruxolitinib Cream in Adolescents and Adults with Atopic Dermatitis. Bissonnette, R., Call, RS., Raoof, T., et al.[2022]

Topical ruxolitinib has been shown to improve various dermatological conditions, including atopic dermatitis, vitiligo, psoriasis, and lichen planus, based on a review of 24 studies involving 2618 patients.

The safety profile of topical ruxolitinib is favorable, with minimal bioavailability and low rates of mild-to-moderate side effects, making it a more tolerable option compared to systemic Janus kinase inhibitors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Utilization of Topical Ruxolitinib in Dermatology: A Review. Kashetsky, N., Turchin, I.[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]

Ruxolitinib, a JAK-2 inhibitor used for treating myeloproliferative neoplasms, is generally associated with very rare skin toxicity, but an unusual case of erythematous skin eruption with necrotic centers was observed in a patient with primary myelofibrosis.

This case highlights the need for increased awareness of potential skin side effects from ruxolitinib, especially as JAK-2 inhibitors may be used more widely in dermatological treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Erythematous skin lesions with necrotic centers on lower extremities due to the use of ruxolitinib for primary myelofibrosis. Dasanu, CA.[2021]

In a multicenter phase II trial involving 84 patients, topical tacrolimus ointment 0.1% demonstrated safety and efficacy in treating long-standing active lichen sclerosus, with 43% of patients achieving complete clearance of the condition after 24 weeks.

The treatment significantly reduced the lesional area and symptoms such as itching, with no serious adverse events reported, indicating it is a promising alternative to conventional corticosteroid therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Multicentre, phase II trial on the safety and efficacy of topical tacrolimus ointment for the treatment of lichen sclerosus. Hengge, UR., Krause, W., Hofmann, H., et al.[2013]

In a preliminary study involving 20 female patients with vulvar lichen sclerosus, injections of Hybrid Cooperative Complexes of Hyaluronic Acid (HCC) significantly reduced symptoms like itching, pain, and burning sensation after 1 and 6 months of treatment.

Patients also reported improved quality of life, with a notable decrease in Dermatology Life Quality Index (DLQI) scores from an average of 5.89 to 3.42, indicating that HCC treatment not only alleviated symptoms but also enhanced overall well-being.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of injecting hybrid cooperative complexes of hyaluronic acid for the treatment of vulvar lichen sclerosus: A preliminary study. Tedesco, M., Garelli, V., Elia, F., et al.[2023]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Ruxolitinib Cream 1.5%: A Review in Mild to Moderate Atopic Dermatitis. [2023]

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

Ruxolitinib Cream in the Treatment of Cutaneous Lichen Planus: A Prospective, Open-Label Study. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Clinically relevant improvements in adults and adolescents with atopic dermatitis who did not achieve Investigator's Global Assessment treatment success following 8 weeks of ruxolitinib cream monotherapy. [2023]

4. New Zealand pubmed.ncbi.nlm.nih.gov

A Maximum-Use Trial of Ruxolitinib Cream in Adolescents and Adults with Atopic Dermatitis. [2022]

5. Canada pubmed.ncbi.nlm.nih.gov

Utilization of Topical Ruxolitinib in Dermatology: A Review. [2023]

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

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

7. England pubmed.ncbi.nlm.nih.gov

Erythematous skin lesions with necrotic centers on lower extremities due to the use of ruxolitinib for primary myelofibrosis. [2021]

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

Vulvar lichen sclerosus: effect of long-term topical application of a potent steroid on the course of the disease. [2019]

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

Short-term effects of topical testosterone in vulvar lichen sclerosus. [2013]

10. Denmark pubmed.ncbi.nlm.nih.gov

[Lichen sclerosus--a neglected disease]. [2011]

11. England pubmed.ncbi.nlm.nih.gov

Multicentre, phase II trial on the safety and efficacy of topical tacrolimus ointment for the treatment of lichen sclerosus. [2013]

12. England pubmed.ncbi.nlm.nih.gov

Efficacy of injecting hybrid cooperative complexes of hyaluronic acid for the treatment of vulvar lichen sclerosus: A preliminary study. [2023]