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NCT05593445

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

Completed Phase 2 Results posted Last updated 24 September 2024
What this trial tests

Phase 2 trial testing Ruxolitinib cream in Lichen Sclerosus in 61 participants. Completed in 21 December 2023.

Timeline
18 November 2022
Primary endpoint
1 September 2023
21 December 2023

Quick facts

Lead sponsorIncyte Corporation
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingtriple
Primary purposetreatment
Enrollment61
Start date18 November 2022
Primary completion1 September 2023
Estimated completion21 December 2023
Sites14 locations across Canada, United States

Drugs / interventions tested

Conditions studied

Sponsor

Incyte Corporation — full company profile →

Who can join

18 and older, female only, with Lichen Sclerosus. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percentage of Participants With ITCH4 at Week 12 Primary · Baseline; 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.

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID35.718.6 – 55.9
DBVC Period: Vehicle Cream BID40.022.7 – 59.4
Change From Baseline in the Clinical Lichen Sclerosus Score (CLISSCO) at Week 12 Secondary · Baseline; 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 sc

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID-5.79± 0.80
DBVC Period: Vehicle Cream BID-3.03± 0.82
Change From Baseline in the Skin Pain NRS Score at Week 12 Secondary · Baseline; 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.

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID-3.22± 0.50
DBVC Period: Vehicle Cream BID-2.70± 0.52
Time to Achieve ITCH4 Secondary · up to 99.0 days

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.

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID35.07.0 – 57.0
DBVC Period: Vehicle Cream BID28.06.0 – NA
Number of Participants With Any Treatment-emergent Adverse Event (TEAE) During the Double-blind, Vehicle-controlled Period Secondary · from Baseline to Week 12 plus 30 days

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.

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID14
DBVC Period: Vehicle Cream BID12
Number of Participants With Any ≥Grade 3 TEAE During the Double-blind, Vehicle-controlled Period Secondary · from Baseline to Week 12 plus 30 days

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 treatmen

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID1
DBVC Period: Vehicle Cream BID0
Number of Participants With Any TEAE During the Open-label Extension Period Secondary · from Week 12 to Week 24 plus 30 days

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.

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID13
DBVC Period: Vehicle Cream BID11
Number of Participants With Any ≥Grade 3 TEAE During the Open-label Extension Period Secondary · from Week 12 to Week 24 plus 30 days

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 livin

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID1
DBVC Period: Vehicle Cream BID0
Number of Participants With Any Clinically Meaningful Changes Over Time in Clinical Laboratory Test Results During the Double-blind, Vehicle-controlled Period Secondary · from Baseline to Week 12 plus 30 days

The investigator determined if a clinical laboratory test value was clinically meaningful.

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID0
DBVC Period: Vehicle Cream BID0
Number of Participants With Any Clinically Meaningful Changes Over Time in Vital Sign Values During the Double-blind, Vehicle-controlled Period Secondary · from Baseline to Week 12 plus 30 days

The investigator determined if a clinical laboratory test value was clinically meaningful.

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID0
DBVC Period: Vehicle Cream BID0
Number of Participants With Any Clinically Meaningful Changes Over Time in Clinical Laboratory Test Results During the Open-label Extension Period Secondary · from Week 12 to Week 24 plus 30 days

The investigator determined if a clinical laboratory test value was clinically meaningful.

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID0
DBVC Period: Vehicle Cream BID0
Number of Participants With Any Clinically Meaningful Changes Over Time in Vital Sign Values During the Open-label Extension Period Secondary · from Week 12 to Week 24 plus 30 days

The investigator determined if a clinical laboratory test value was clinically meaningful.

GroupValue95% CI
DBVC Period: Ruxolitinib Cream 1.5% BID0
DBVC Period: Vehicle Cream BID0

Adverse events — posted to ClinicalTrials.gov

Time frame: up to approximately 32 weeks. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Ruxolitinib Cream 1.5% BID
Serious: 0/58 (0%)
Deaths: 0/58
Vehicle Cream BID
Serious: 1/30 (3%)
Deaths: 0/30

Serious adverse events (1 terms)

ReactionSystemRuxolitinib Cream 1.5% BIDVehicle Cream BID
COVID-19 pneumoniaInfections and infestations
Other adverse events (5 terms — click to expand)

ReactionSystemRuxolitinib Cream 1.5% BIDVehicle Cream BID
Urinary tract infectionInfections and infestations
NasopharyngitisInfections and infestations
Vulvovaginal mycotic infectionInfections and infestations
COVID-19Infections and infestations
Upper respiratory tract infectionInfections and infestations

Most-reported serious reactions: COVID-19 pneumonia.

Data from ClinicalTrials.gov NCT05593445 adverse events section.

Sponsor's own description

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.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

  1. JAK/STAT pathway: Extracellular signals, diseases, immunity, and therapeutic regimens.
    Hu Q, Bian Q, Rong D, Wang L, et al · · 2023 · cited 190× · PMID 36911202 · DOI 10.3389/fbioe.2023.1110765
  2. Lichen sclerosus: The 2023 update.
    De Luca DA, Papara C, Vorobyev A, Staiger H, et al · · 2023 · cited 94× · PMID 36873861 · DOI 10.3389/fmed.2023.1106318
  3. Clinical, mechanistic, and therapeutic landscape of cutaneous fibrosis.
    Li DJ, Berry CE, Wan DC, Longaker MT. · · 2024 · cited 14× · PMID 39321265 · DOI 10.1126/scitranslmed.adn7871
  4. Efficacy and safety of ruxolitinib cream in patients with lichen sclerosus: Results from a phase 2, randomized, double-blind, vehicle-controlled study.
    Goldstein AT, Lai Z, Rong R, Nawaz H, et al · · 2026 · PMID 42103188 · DOI 10.1016/j.jaad.2026.04.1993

Verify or expand the search:

Other trials of Ruxolitinib cream

Trials testing the same drug.

Other recruiting trials for Lichen Sclerosus

Currently open trials in the same condition.

Other Incyte Corporation trials

Trials by the same sponsor.

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing