Clinical trial NCT04391894: A Study to Assess the Safety and Efficacy of ECF843 vs Vehicle in Subjects With Dry Eye Disease
Official title: A Randomized, Double-masked, Multicenter Study to Evaluate the Safety and Efficacy of ECF843 vs Vehicle in Subjects With Dry Eye Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Dry Eye
Keywords: Dry Eye
Sjogrens
dry eye syndrome (DES)
Keratoconjunctivitis sicca (KCS)
keratitis
Xerophthalmia
Sjögren's Syndrome
Sjogren's Syndrome
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2020-05-18
Enrollment: 718
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The study was planned to be conducted in 2 parts: Part 1 to determine the efficacy and safety of ECF843 vs vehicle, followed by Part 2 with additional exploratory assessments of ECF843 vs Vehicle. Both parts of the study included a double-masked study design, with randomization stratified for subjects with Sjogren's Syndrome.

Detailed description: Part 1 of the study was a double-masked, randomized, parallel design in which participants were assigned to one of the following five treatment arms/groups in a ratio of 1:1:1:1:1.

* ECF843 0.45 mg/mL three times daily (TID) or vehicle
* ECF843 0.15 mg/mL TID or vehicle
* ECF843 vehicle TID
* ECF843 0.15 mg/mL twice daily (BID) or vehicle
* ECF843 vehicle BID The planned duration of double-masked treatment during Part 1 was 56 days. For subjects randomized to ECF843, the maximum drug exposure was up to 28 days. At some point during Part 1, all participants received vehicle.

The study was terminated after completion of Part 1 and Part 2 of the study was not therefore initiated.

Eligibility criteria: Inclusion Criteria:

* Written informed consent must be obtained before any assessment
* Adult male or female subjects 18 years of age or older
* At least 6 months history of dry eye disease in both eyes
* Must use, or feel the need to use, artificial tears/gels/lubricants on a regular basis
* Composite corneal fluorescein staining score \>= 4 (modified National Eye Institute (NEI) scale) in at least one eye
* Schirmer score \>= 1 and =\< 10 mm after 5 minutes in at least one eye
* Patients with Sjögren's Syndrome must have dry eye

Exclusion Criteria:

* Ocular infection in either eye within 30 days prior to Screening
* Use of artificial tears, gels, lubricants within 4 hours of the Screening Visit
* Use of contact lenses in either eye within 14 days of Screening
* Uncontrolled ocular rosacea
* Clinically significant conjunctivochalasis in either eye
* Other Corneal conditions affecting the corneal structure
* Severe ocular conditions such as herpes, graft versus host disease, Stephen's Johnson Syndrome, sarcoidosis
* Currently active, or history of ocular allergies during the time of year the patient will be participating in the study
* Patients with current punctal plugs or punctal cauterization or occlusion
* Chronic medications (both over the counter and prescription) that have not been stable for at least 30 days prior to Screening.
* Use of Restasis®, Cequa®, or Xiidra® within 30 days prior to Screening
* Use of ocular, nasal, inhaled, or systemic corticosteroids within 30 days of Screening
* History of malignancy of any organ system within the past five years
* Pregnant or nursing women

Primary outcomes:
measure: Part 1: Change From Baseline in Symptom Assessment in Dry Eye (SANDE) Score
timeFrame: Up to 28 days (Baseline (BL) to end of randomized treatment)
measure: Part 1: Change From Baseline in Composite Corneal Fluorescein Staining Score
timeFrame: Up to 28 days (Baseline (BL) to end of randomized treatment)
Secondary outcomes:
measure: Part 1: Change From Baseline in Central Corneal Fluorescein Staining
timeFrame: Up to 28 days (Baseline (BL) to end of randomized treatment)
measure: Part 1: Change From Baseline in Inferior Corneal Fluorescein Staining
timeFrame: Up to 28 days (Baseline (BL) to end of randomized treatment)
measure: Part 1: Percentage of Participants With Ocular and Non-ocular Treatment Emergent Adverse Events (AEs)
timeFrame: Up to 28 days (Baseline (BL) to end of randomized treatment)
Other outcomes:
Locations:
city: Chandler
country: United States
facility: Arizona Eye Center
state: Arizona
city: Mesa
country: United States
facility: Carrot Eye Center
state: Arizona
city: Phoenix
country: United States
facility: Phoenix Eye Care and Dry Eye Ctr
state: Arizona
city: Azusa
country: United States
facility: Milton Hom OD
state: California
city: Garden Grove
country: United States
facility: Orange County Opthamology Med Grp
state: California
city: Glendale
country: United States
facility: Dr Kent Small
state: California
city: Glendale
country: United States
facility: Global Research Management
state: California
city: Inglewood
country: United States
facility: United Medical Research Institute
state: California
city: Laguna Hills
country: United States
facility: Harvard Eye Associates
state: California
city: Mission Hills
country: United States
facility: North Valley Eye Medical Group
state: California
city: Newport Beach
country: United States
facility: The Eye Research Foundation
state: California
city: Petaluma
country: United States
facility: North Bay Eye Associates Inc
state: California
city: Rancho Cordova
country: United States
facility: Martel Eye Medical Group
state: California
city: Santa Ana
country: United States
facility: Sierra Clin Trials Rsch Org
state: California
city: Colorado Springs
country: United States
facility: Vision Institute
state: Colorado
city: Deerfield Beach
country: United States
facility: Advanced Research LLC
state: Florida
city: Fort Lauderdale
country: United States
facility: Pinnacle Research Institute
state: Florida
city: Fort Myers
country: United States
facility: Eye Associates Of Fort Myers
state: Florida
city: Largo
country: United States
facility: Lee Shettle Eye & Hearing
state: Florida
city: Mount Dora
country: United States
facility: Mid Florida Eye Center Pa
state: Florida
city: Pittsburg
country: United States
facility: Kannarr Eye Care
state: Kansas
city: Louisville
country: United States
facility: The Eye Care Institute
state: Kentucky
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Kansas City
country: United States
facility: Silverstein Eye Centers
state: Missouri
city: Kansas City
country: United States
facility: Moyes Eye Center
state: Missouri
city: Saint Louis
country: United States
facility: Tekwani Vision Center
state: Missouri
city: Washington
country: United States
facility: Comprehensive Eye Care Limited
state: Missouri
city: Henderson
country: United States
facility: NV Eye Surgery
state: Nevada
city: Las Vegas
country: United States
facility: AdvanceMed Clinical Research
state: Nevada
city: South Orange
country: United States
facility: Northern New Jersey Eye Institut PA
state: New Jersey
city: High Point
country: United States
facility: Cornerstone Eye Care
state: North Carolina
city: Cincinnati
country: United States
facility: Eye Care Assoc of Greater Cincinnati Inc
state: Ohio
city: Cleveland
country: United States
facility: Abrams Eye Center
state: Ohio
city: Mason
country: United States
facility: Apex Eye Clinical Reserach
state: Ohio
city: Cranberry Township
country: United States
facility: Scott Christie Eye Care Associates
state: Pennsylvania
city: Wilkes-Barre Township
country: United States
facility: Bucci Laser Vision Institute
state: Pennsylvania
city: Sioux Falls
country: United States
facility: Vance Thompson Vision Hospital
state: South Dakota
city: Chattanooga
country: United States
facility: Chattanooga Eye Institute
state: Tennessee
city: Maryville
country: United States
facility: University Eye Specialists
state: Tennessee
city: Memphis
country: United States
facility: Total Eye Care PA
state: Tennessee
city: Nashville
country: United States
facility: Toyos Clinic
state: Tennessee
city: Smyrna
country: United States
facility: Advancing Vision Research LLC
state: Tennessee
city: Austin
country: United States
facility: Texan Eye P A
state: Texas
city: Houston
country: United States
facility: Intouch Clinical Research Center
state: Texas
city: Houston
country: United States
facility: Neuro Eye Clinical Trials Inc
state: Texas
city: Lakeway
country: United States
facility: Revolution Research
state: Texas
city: San Antonio
country: United States
facility: Parkhurst NuVision
state: Texas
city: Saint George
country: United States
facility: Southern Utah Medical Research
state: Utah
city: Salt Lake City
country: United States
facility: Stacy R Smith MD PC
state: Utah
city: Lynchburg
country: United States
facility: Piedmont Eye Center
state: Virginia
city: Norfolk
country: United States
facility: Virginia Eye Consultants
state: Virginia
city: Seattle
country: United States
facility: Periman Eye Institute
state: Washington
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: