A Study to Assess the Safety and Efficacy of ECF843 vs Vehicle in Subjects With Dry Eye Disease

A Randomized, Double-masked, Multicenter Study to Evaluate the Safety and Efficacy of ECF843 vs Vehicle in Subjects With Dry Eye Disease

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.

Study Overview

• Status: Completed
• Conditions: Dry Eye
• Intervention / Treatment: Drug: ECF843; Other: ECF843 vehicle

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.

• Study Type: Interventional
• Enrollment (Actual): 718
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Chandler, Arizona, United States, 85224
      • Arizona Eye Center
    • Mesa, Arizona, United States, 85202
      • Carrot Eye Center
    • Phoenix, Arizona, United States, 85032
      • Phoenix Eye Care and Dry Eye Ctr
  • California
    • Azusa, California, United States, 91702
      • Milton Hom OD
    • Garden Grove, California, United States, 92843
      • Orange County Opthamology Med Grp
    • Glendale, California, United States, 91205
      • Global Research Management
    • Glendale, California, United States, 91203
      • Dr Kent Small
    • Inglewood, California, United States, 90301
      • United Medical Research Institute
    • Laguna Hills, California, United States, 92653
      • Harvard Eye Associates
    • Mission Hills, California, United States, 91345
      • North Valley Eye Medical Group
    • Newport Beach, California, United States, 92663
      • The Eye Research Foundation
    • Petaluma, California, United States, 94954
      • North Bay Eye Associates INC
    • Rancho Cordova, California, United States, 95670
      • Martel Eye Medical Group
    • Santa Ana, California, United States, 92705
      • Sierra Clin Trials Rsch Org
  • Colorado
    • Colorado Springs, Colorado, United States, 80907
      • Vision Institute
  • Florida
    • Deerfield Beach, Florida, United States, 33064
      • Advanced Research LLC
    • Fort Lauderdale, Florida, United States, 33309
      • Pinnacle Research Institute
    • Fort Myers, Florida, United States, 33901
      • Eye Associates of Fort Myers
    • Largo, Florida, United States, 33773
      • Lee Shettle Eye & Hearing
    • Mount Dora, Florida, United States, 32757
      • Mid Florida Eye Center Pa
  • Kansas
    • Pittsburg, Kansas, United States, 66762
      • Kannarr Eye Care
  • Kentucky
    • Louisville, Kentucky, United States, 40206
      • The Eye Care Institute
  • Massachusetts
    • Boston, Massachusetts, United States, 02111
      • Tufts Medical Center
  • Missouri
    • Kansas City, Missouri, United States, 64154
      • Moyes Eye Center
    • Kansas City, Missouri, United States, 64133
      • Silverstein Eye Centers
    • Saint Louis, Missouri, United States, 63128
      • Tekwani Vision Center
    • Washington, Missouri, United States, 63090
      • Comprehensive Eye Care Limited
  • Nevada
    • Henderson, Nevada, United States, 89052
      • NV Eye Surgery
    • Las Vegas, Nevada, United States, 89123
      • AdvanceMed Clinical Research
  • New Jersey
    • South Orange, New Jersey, United States, 07079
      • Northern New Jersey Eye Institut PA
  • North Carolina
    • High Point, North Carolina, United States, 27262
      • Cornerstone Eye Care
  • Ohio
    • Cincinnati, Ohio, United States, 45236
      • Eye Care Assoc of Greater Cincinnati Inc
    • Cleveland, Ohio, United States, 44115
      • Abrams Eye Center
    • Mason, Ohio, United States, 45040
      • Apex Eye Clinical Reserach
  • Pennsylvania
    • Cranberry Township, Pennsylvania, United States, 16066
      • Scott Christie Eye Care Associates
    • Wilkes-Barre Township, Pennsylvania, United States, 18702
      • Bucci Laser Vision Institute
  • South Dakota
    • Sioux Falls, South Dakota, United States, 57108
      • Vance Thompson Vision Hospital
  • Tennessee
    • Chattanooga, Tennessee, United States, 37411
      • Chattanooga Eye Institute
    • Maryville, Tennessee, United States, 37803
      • University Eye Specialists
    • Memphis, Tennessee, United States, 38119
      • Total Eye Care PA
    • Nashville, Tennessee, United States, 37215
      • Toyos Clinic
    • Smyrna, Tennessee, United States, 37167
      • Advancing Vision Research LLC
  • Texas
    • Austin, Texas, United States, 78731
      • Texan Eye P A
    • Houston, Texas, United States, 77034
      • Intouch Clinical Research Center
    • Houston, Texas, United States, 77074
      • Neuro Eye Clinical Trials Inc
    • Lakeway, Texas, United States, 78738
      • Revolution Research
    • San Antonio, Texas, United States, 78229
      • Parkhurst NuVision
  • Utah
    • Saint George, Utah, United States, 84790
      • Southern Utah Medical Research
    • Salt Lake City, Utah, United States, 84117
      • Stacy R Smith MD PC
  • Virginia
    • Lynchburg, Virginia, United States, 24502
      • Piedmont Eye Center
    • Norfolk, Virginia, United States, 23502
      • Virginia Eye Consultants
  • Washington
    • Seattle, Washington, United States, 98119
      • Periman Eye Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ECF843 0.45 mg/mL TID or vehicle (Part 1)	Drug: ECF843; Topical ocular eye drop; Other: ECF843 vehicle; Topical ocular eye drop
Experimental: ECF843 0.15 mg/mL TID or vehicle (Part 1)	Drug: ECF843; Topical ocular eye drop; Other: ECF843 vehicle; Topical ocular eye drop
Placebo Comparator: ECF843 vehicle TID (Part 1)	Other: ECF843 vehicle; Topical ocular eye drop
Experimental: ECF843 0.15 mg/mL BID or vehicle (Part 1)	Drug: ECF843; Topical ocular eye drop; Other: ECF843 vehicle; Topical ocular eye drop
Placebo Comparator: ECF843 vehicle BID (Part 1)	Other: ECF843 vehicle; Topical ocular eye drop

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Part 1: Change From Baseline in Symptom Assessment in Dry Eye (SANDE) Score	The SANDE uses a 100 mm visual analog scale (VAS) and asks the subject to score frequency and severity of their ocular discomfort over the past 24 hours by putting a vertical mark on two separate horizontal scoring lines. The frequency scoring line utilizes the anchors of 'Rarely' to 'All the Time', while the severity scoring line utilizes the anchors of 'Very Mildly' to 'Very Severely uncomfortable'. The SANDE questionnaire was completed through an electronic diary by the subject at the Screening Visit(s) of Part 1, and thereafter every evening before bedtime during the study. The overall SANDE score was calculated by taking the square root of the product of the frequency of symptoms score and the severity of symptoms score. The SANDE scale ranged from 0 to 100 with 100 being the maximal amount of dry eye symptoms and 0 being the minimal amount of dry eye symptoms. Negative change from baseline indicates improvement.	Up to 28 days (Baseline (BL) to end of randomized treatment)
Part 1: Change From Baseline in Composite Corneal Fluorescein Staining Score	The degree of staining was based on the Corneal Fluorescein Modified NEI Scale. Each of the five regions (central (C), superior (S), inferior (I), temporal (T), and nasal (N)) were graded based on a scale of 0 to 4, with higher scores suggestive of higher degrees of corneal staining. After entry of the scores per region, the total or composite (sum) score for each eye was automatically calculated (maximum score = 20/eye). A (+1) was added to the sum score for any eye with the presence of filaments.	Up to 28 days (Baseline (BL) to end of randomized treatment)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Part 1: Change From Baseline in Central Corneal Fluorescein Staining	The degree of staining was based on the Corneal Fluorescein Modified NEI Scale. Central region was graded based on a scale of 0 to 4, with higher scores suggestive of higher degrees of corneal staining.	Up to 28 days (Baseline (BL) to end of randomized treatment)
Part 1: Change From Baseline in Inferior Corneal Fluorescein Staining	The degree of staining was based on the Corneal Fluorescein Modified NEI Scale. Inferior region was graded based on a scale of 0 to 4, with higher scores suggestive of higher degrees of corneal staining.	Up to 28 days (Baseline (BL) to end of randomized treatment)
Part 1: Percentage of Participants With Ocular and Non-ocular Treatment Emergent Adverse Events (AEs)	The number of treatment emergent ocular and non-ocular adverse events was reported categorically: Mild, Moderate, Severe. Treatment emergent adverse events (TEAEs) are adverse events started after the first administration of randomized study treatment or events present prior to start of the randomized treatment but increased in severity based on preferred term.	Up to 28 days (Baseline (BL) to end of randomized treatment)

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Publications and helpful links

Helpful Links

• A Plain Language Trial Summary is available on novctrd.com

Study record dates

Study Major Dates

• Study Start (Actual): October 6, 2020
• Primary Completion (Actual): May 13, 2021
• Study Completion (Actual): May 13, 2021

Study Registration Dates

• First Submitted: May 13, 2020
• First Submitted That Met QC Criteria: May 13, 2020
• First Posted (Actual): May 18, 2020

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 6, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: Sjogren's Syndrome; Dry Eye; Sjögren's Syndrome; keratitis; Xerophthalmia; Sjogrens; dry eye syndrome (DES); Keratoconjunctivitis sicca (KCS)
• Additional Relevant MeSH Terms: Corneal Diseases; Lacrimal Apparatus Diseases; Keratoconjunctivitis; Conjunctivitis; Conjunctival Diseases; Keratitis; Eye Diseases; Dry Eye Syndromes; Keratoconjunctivitis Sicca
• Other Study ID Numbers: CECF843A2201

Plan for Individual participant data (IPD)

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

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent 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.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

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