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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
61
Ruxolitinib cream is a topical formulation applied as a thin film to affected areas.
Vehicle cream is matching in appearance to ruxolitinib cream and is to be applied in the same manner as ruxolitinib cream.
Cahaba Dermatology
Birmingham, Alabama, United States
Mayo Clinic - Scottsdale
Scottsdale, Arizona, United States
UC Irvine
Irvine, California, United States
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.
Time frame: Baseline; Week 12
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.
Time frame: 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.
Time frame: Baseline; Week 12
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The Centers For Vulvovaginal Disorders
Washington D.C., District of Columbia, United States
New Age Medical Research Corporation
Miami, Florida, United States
Circuit Clinical
West Seneca, New York, United States
Unc Dermatology and Skin Cancer Center At Southern Village
Chapel Hill, North Carolina, United States
Apex Dermatology
Ashtabula, Ohio, United States
Bexley Dermatology
Bexley, Ohio, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
...and 4 more locations
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: from Week 12 to Week 24 plus 30 days