An Open-Label, Phase 2, Safety, and Efficacy Study of Ruxolitinib Cream in Participants With Genital Vitiligo

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Incyte Corp.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

8 Nov 2023 → 6 Mar 2025

Decision date (initial)

2023-09-12

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

The objective of this study is to determine if ruxolitinib 1.5% cream BID effects repigmentation of vitiligo in the genital region.

Secondary objectives 2

1. To evaluate the safety and tolerability of ruxolitinib cream
2. To further evaluate the efficacy of ruxolitinib cream.

Conditions and MedDRA coding

Nonsegmental vitiligo with genital involvement

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 11

1. Ability to comprehend and willingness to sign an ICF for the study.
2. Age ≥ 18 years at the time of signing the ICF.
3. Clinical diagnosis of nonsegmental vitiligo with genital involvement; the genital area (approximately 1% BSA) is defined as labia majora, labia minora, and perineum in females, and penis, scrotum, and perineum in males.
4. At least ≥ 0.25% BSA of nonsegmental vitiligo in the genital area.
5. Presence of pigmented hair within the depigmented areas (if the area is hair bearing).
6. At least 1 genital target lesion that is ≥ 0.1% BSA that has a pigmented hair within it.
7. Vitiligo on areas of the body besides the genitals.
8. Total body vitiligo area not exceeding 10% BSA.
9. Willing to have genital photography conducted.
10. Must agree to discontinue all agents used to treat vitiligo from screening through the final safety follow-up visit. Over-the-counter preparations deemed acceptable by the investigator and camouflage makeups are permitted.
11. Male and female participants must be willing to take appropriate contraceptive measures (see Appendix A) to avoid pregnancy or fathering a child for the duration of study participation with the exception of the following: a. Females of non–childbearing potential (ie, or surgically sterile with a hysterectomy and/or bilateral oophorectomy OR postmenopausal, ≥ 12 months of amenorrhea without an alternative medical cause).

Exclusion criteria 19

1. Other forms of vitiligo (eg, segmental) or other differential diagnosis of vitiligo or other skin depigmentation disorders (eg, balanitis xerotica obliterans, piebaldism, pityriasis alba, leprosy, postinflammatory hypopigmentation, progressive macule hypomelanosis, nevus anemicus, chemical leukoderma, and tinea versicolor).
2. Prior or current use of depigmentation treatments (eg, monobenzone).
3. Active or recurrent genital warts or herpes.
4. Male participants with partners with known current/active cervical intraepithelial neoplasia or anal intraepithelial neoplasia.
5. An active sexually transmitted disease, sexually transmitted infection, or other skin disorder affecting the genital area (eg, scabies, fungal infection, molluscum).
6. Had ≥ 3 laser hair removal treatments in an area to be treated for vitiligo.
7. No venous access outside of areas to be treated.
8. Concurrent conditions and history of other diseases as follows: a. Any other skin disorder that, in the opinion of the investigator, would interfere with the study medication application or study assessments. b. Immunocompromised (eg, lymphoma, acquired immunodeficiency syndrome, or Wiskott-Aldrich syndrome). c. Chronic or acute infection requiring treatment with systemic antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungals within 2 weeks before baseline. d. Active acute bacterial, fungal, or viral skin infection (eg, herpes simplex, herpes zoster, chicken pox, clinically infected atopic dermatitis, or impetigo) within 1 week before baseline. e. Unstable asthma or COPD requiring systemic treatment (such as intravenous steroids) or hospital admission or emergency room treatment within 6 months from baseline; or stable asthma or COPD requiring more than 880 μg/day of inhaled budesonide or equivalent high dose of other inhaled corticosteroids. f. Current or history of hepatitis B or C virus infection. g. Current or history of HIV infection.
9. Any serious illness or medical, physical, or psychiatric condition(s) that, in the investigator's opinion, would interfere with full participation in the study, including application of study cream and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data. For example: a. Clinically significant or uncontrolled cardiovascular disease, including unstable angina, acute myocardial infarction, or stroke within 6 months from Day 1 of study cream application, New York Heart Association Class III or IV congestive heart failure, and arrhythmia requiring therapy or uncontrolled hypertension (blood pressure > 150/90 mmHg) unless approved by medical monitor/sponsor. b. Participants with or a history of malignancy in the 5 years preceding the baseline visit, except for adequately treated, nonmetastatic, nonmelanoma, skin cancer. c. Current and/or history of arterial or venous thrombosis, including deep venous thrombosis and pulmonary embolism. d. Current and/or history of active tuberculosis; or current and/or history of latent tuberculosis unless adequately treated. e. History of severe anemia, severe thrombocytopenia, or severe neutropenia.
10. Any of the following clinical laboratory test results at screening: a. Hemoglobin < 100 g/L (ie, 10 g/dL) b. Absolute neutrophil count < 1.5 × 109/L (ie, 1500/μL) c. Platelet count < 1 × 1011/L (ie, 100,000/μL) d. AST or ALT ≥ 2.5 × ULN e. Total bilirubin > 1.5 × ULN unless Gilbert's syndrome f. Estimated glomerular filtration rate < 60 mL/min/1.73 m2 (using the Chronic Kidney Disease Epidemiology Collaboration equation) g. Any other clinically significant laboratory result that, in the opinion of the investigator, poses a significant risk to the participant.
11. Use of any of the following treatments within the indicated washout period before baseline: a. 1 week: Topical drugs when used on the vitiligo areas (eg, corticosteroids, calcineurin, bimatoprost, phosphodiesterase type 4 inhibitors, retinoids). b. 4 weeks: − Melanocyte-stimulating agents (eg, afamelanotide). − Immunomodulating systemic medications (eg, corticosteroids, methotrexate, cyclosporine). − Live vaccines. Note: Live-attenuated vaccines are prohibited during the course of the study and within 4 weeks after the EOT visit. c. 12 weeks: − JAK inhibitors, systemic or topical. − Laser or any kind of phototherapy, including tanning bed or intentional UV exposure. d. 5 half-lives or 12 weeks, whichever is longer: Biologic agents, investigational or experimental therapy or procedures for vitiligo. Investigational biologics should be discussed with the sponsor to determine whether a longer period of discontinuation is required.
12. Body mass index < 17 or > 40 kg/m2.
13. Pregnant or lactating participants, or those considering pregnancy during the period of their study participation.
14. History of alcoholism or drug addiction within 1 year before screening or current alcohol or drug use that, in the opinion of the investigator, will interfere with the participant's ability to comply with the application schedule and study assessments.
15. Current treatment or treatment within 30 days or 5 half-lives (whichever is longer) before the baseline visit with another investigational medication or current enrollment in another investigational drug study.
16. Committed to a mental health institution by virtue of an order issued either by the judicial or the administrative authorities.
17. In the opinion of the investigator, unable or unlikely to comply with the application schedule and study evaluations.
18. Employees of the sponsor or investigator or are otherwise dependents of them.
19. Known allergy or reaction to any component of the study drug formulation.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint is the proportion of participants achieving a genital VNS of "4 – A lot less noticeable" or "5 – No longer noticeable" at Week 48.

Secondary endpoints 6

1. Occurrence of AEs and changes in vital signs and laboratory data.
2. Proportion of participants achieving genital PhGVA of 0 or 1 at Week 48.
3. Change from baseline in affected BSA in the genital region at Weeks 24 and 48.
4. Proportion of participants achieving T-VASI50/75/90 at Weeks 24 and 48.
5. Proportion of participants achieving a genital VNS of "4 – A lot less noticeable" or "5 – No longer noticeable" at Week 24.
6. Proportion of participants in each category of the color-matching question at Weeks 24 and 48.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Incyte Corp.

Sponsor organisation

Incyte Corp.

Address

1801 Augustine Cut Off

City

Wilmington

Postcode

19803-4404

Country

United States

Scientific contact point

Organisation

Incyte Corp.

Contact name

Clinical Trial Information Desk

Public contact point

Organisation

Incyte Corp.

Contact name

Clinical Trial Information Desk

Third parties 2

Locations

1 EU/EEA country · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 8 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications