A Study to Evaluate the Efficacy and Safety of Ruxolitinib Cream in Participants With Lichen Sclerosus

A Phase 2, Randomized, Double-Blind, Vehicle-Controlled, Study of the Efficacy and Safety of Ruxolitinib Cream in Participants With Lichen Sclerosus

The purpose of this study is to evaluate the efficacy and safety of Ruxolitinib cream in participants With Lichen Sclerosus. This is randomized, double-blind, vehicle-controlled (DBVC) study with a DBVC period of 12 weeks followed by an open label period (OLE) period of 12 weeks.

Study Overview

• Status: Completed
• Conditions: Lichen Sclerosus
• Intervention / Treatment: Drug: Ruxolitinib cream; Drug: Vehicle cream

• Study Type: Interventional
• Enrollment (Actual): 61
• Phase: Phase 2

Expanded Access

Approved for sale to the public. See expanded access record.

Contacts and Locations

Study Locations

• Canada
  • Quebec, Canada, G1V 3M7
    • Clinique Rsf
  • Ontario
    • Waterloo, Ontario, Canada, N2J 1C4
      • K. Papp Clinical Research
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35244
      • Cahaba Dermatology
  • Arizona
    • Scottsdale, Arizona, United States, 85259
      • Mayo Clinic - Scottsdale
  • California
    • Irvine, California, United States, 92697
      • UC Irvine
  • District of Columbia
    • Washington, District of Columbia, United States, 20037
      • The Centers For Vulvovaginal Disorders
  • Florida
    • Miami, Florida, United States, 33186
      • New Age Medical Research Corporation
  • New York
    • West Seneca, New York, United States, 14224
      • Circuit Clinical
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27516
      • Unc Dermatology and Skin Cancer Center At Southern Village
  • Ohio
    • Ashtabula, Ohio, United States, 44004
      • Apex Dermatology
    • Bexley, Ohio, United States, 43209
      • Bexley Dermatology
  • Texas
    • Dallas, Texas, United States, 75390
      • University of Texas Southwestern Medical Center
  • Utah
    • Murray, Utah, United States, 84107
      • University of Utah Health Care Midvalley Health Center Dermatology
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Skin and Laser Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Biopsy-proven LS in the anogenital area.
• Baseline IGA score ≥ 2 for LS.
• Baseline Itch NRS score ≥ 4 in anogenital area.
• Willingness to avoid pregnancy.

Exclusion Criteria:

• Participants who do not have LS involving anogenital area.

• Concurrent conditions and history of other diseases:

  1. Are suspected clinically (or confirmed diagnostically) of having alternative causes of vaginal symptoms including: candidiasis, chlamydia trachomatis, trichomonas vaginalis, neisseria gonorrhoeae, bacterial vaginosis, or herpes simplex.
  2. Have active genital/vulvar lesions at screening and Day 1, not related to LS
  3. Chronic or acute infection requiring treatment with systemic antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungals within 2 weeks before baseline.
• Laboratory values outside of the protocol-defined criteria
• Pregnant or lactating participants or those considering pregnancy during the period of their study participation..
• Other exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ruxolitinib cream; 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.	Drug: Ruxolitinib cream; Ruxolitinib cream is a topical formulation applied as a thin film to affected areas.; Other Names: INCB018424 cream; Drug: Vehicle cream; Vehicle cream is matching in appearance to ruxolitinib cream and is to be applied in the same manner as ruxolitinib cream.
Placebo Comparator: Vehicle Cream; Vehicle cream BID for 12 weeks followed by ruxolitinb 1.5% cream BID (or QD) for 12-weeks in an open-label extension.	Drug: Vehicle cream; Vehicle cream is matching in appearance to ruxolitinib cream and is to be applied in the same manner as ruxolitinib cream.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants With ITCH4 at Week 12	ITCH4 response was defined as a ≥4-point improvement from Baseline in by-visit Itch Numeric Rating Scale (NRS) score. The Itch NRS is a daily participant-reported measure (24-hour recall) of the worst level of itch intensity. Participants rated itch severity of their lichen sclerosus by selecting a number from 0 (no itch) to 10 (worst imaginable itch) that best described the worst level of itch they experienced in the past 24 hours.	Baseline; Week 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in the Clinical Lichen Sclerosus Score (CLISSCO) at Week 12	The CLISSCO is a validated tool to assess disease severity in vulvar lichen sclerosus. The Clinical Lichen Sclerosus Score consists of 12 items divided into 3 sections: symptoms (3 items; likely reversible [i.e., itch, pain, dysuria]); signs (3 items; possibly reversible [i.e., whitening, petechiae/ecchymosis, fissures]); and architectural changes (6 items; irreversible [i.e., skin fusion, perianal involvement, etc.]). All symptoms, signs, and architectural changes were rated on a 4-point Likert scale: 0 (absent), 1 (mild), 2 (moderate), and 3 (severe). The investigator documented the score of each of the 12 items; the CLISSCO was calculated by summing the score of each question, with a maximum score of 36 and a minimum score of 0. The higher the score, the more severe the disease. Additionally, the total score for each of the 3 sections (symptoms, signs, and architectural changes) was summarized by summing the scores of the questions in each section.	Baseline; Week 12
Change From Baseline in the Skin Pain NRS Score at Week 12	Participants were instructed to complete and record the Skin Pain NRS in a diary each evening beginning on the day of screening through Week 12 or treatment discontinuation. Participants rated their pain, which included all types of pain (e.g., burning, tearing, pulling, stabbing, etc.) severity of lichen sclerosus by selecting a number from 0 (no pain) to 10 (worst imaginable pain) that best described the worst level of pain they experienced in the past 24 hours.	Baseline; Week 12
Time to Achieve ITCH4	ITCH4 response was defined as a ≥4-point improvement from Baseline in by-visit Itch Numeric Rating Scale (NRS) score. The Itch NRS is a daily participant-reported measure (24-hour recall) of the worst level of itch intensity. Participants rated itch severity of their lichen sclerosus by selecting a number from 0 (no itch) to 10 (worst imaginable itch) that best described the worst level of itch they experienced in the past 24 hours.	up to 99.0 days
Number of Participants With Any Treatment-emergent Adverse Event (TEAE) During the Double-blind, Vehicle-controlled Period	An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered drug related. An AE could therefore have been any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. A TEAE was defined as an AE either reported for the first time or the worsening of a pre-existing event after the first application of study drug.	from Baseline to Week 12 plus 30 days
Number of Participants With Any ≥Grade 3 TEAE During the Double-blind, Vehicle-controlled Period	A TEAE was defined as an AE either reported for the first time or the worsening of a pre-existing event after the first application of study drug. The severity of AEs was assessed using the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 (v5.0) Grades 1 through 5. The investigator made an assessment of intensity for each AE and SAE reported during the study and assigned it to 1 of the following categories: Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal.	from Baseline to Week 12 plus 30 days
Number of Participants With Any TEAE During the Open-label Extension Period	An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered drug related. An AE could therefore have been any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. A TEAE was defined as an AE either reported for the first time or the worsening of a pre-existing event after the first application of study drug.	from Week 12 to Week 24 plus 30 days
Number of Participants With Any ≥Grade 3 TEAE During the Open-label Extension Period	A TEAE was defined as an AE either reported for the first time or the worsening of a pre-existing event after the first application of study drug. The severity of AEs was assessed using the CTCAE v5.0 Grades 1 through 5. The investigator made an assessment of intensity for each AE and SAE reported during the study and assigned it to 1 of the following categories: Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal.	from Week 12 to Week 24 plus 30 days
Number of Participants With Any Clinically Meaningful Changes Over Time in Clinical Laboratory Test Results During the Double-blind, Vehicle-controlled Period	The investigator determined if a clinical laboratory test value was clinically meaningful.	from Baseline to Week 12 plus 30 days
Number of Participants With Any Clinically Meaningful Changes Over Time in Vital Sign Values During the Double-blind, Vehicle-controlled Period	The investigator determined if a clinical laboratory test value was clinically meaningful.	from Baseline to Week 12 plus 30 days
Number of Participants With Any Clinically Meaningful Changes Over Time in Clinical Laboratory Test Results During the Open-label Extension Period	The investigator determined if a clinical laboratory test value was clinically meaningful.	from Week 12 to Week 24 plus 30 days
Number of Participants With Any Clinically Meaningful Changes Over Time in Vital Sign Values During the Open-label Extension Period	The investigator determined if a clinical laboratory test value was clinically meaningful.	from Week 12 to Week 24 plus 30 days

Collaborators and Investigators

• Sponsor: Incyte Corporation
• Investigators: Study Director: Haq Nawaz, md, Incyte Corporation

Study record dates

Study Major Dates

• Study Start (Actual): November 18, 2022
• Primary Completion (Actual): September 1, 2023
• Study Completion (Actual): December 21, 2023

Study Registration Dates

• First Submitted: October 21, 2022
• First Submitted That Met QC Criteria: October 21, 2022
• First Posted (Actual): October 25, 2022

Study Record Updates

• Last Update Posted (Actual): September 24, 2024
• Last Update Submitted That Met QC Criteria: August 29, 2024
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Keywords: Ruxolitinib; Lichen Sclerosus; Skin Diseases; 18424; topical cream; vulvar disease
• Additional Relevant MeSH Terms: Skin Diseases; Skin Diseases, Papulosquamous; Lichenoid Eruptions; Lichen Sclerosus et Atrophicus
• Other Study ID Numbers: INCB 18424-220

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency

• IPD Sharing Time Frame: Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
• IPD Sharing Access Criteria: Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No