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

NCT04391894 | Completed Phase 2 Results FDA Drug

• 1 other identifier: CECF843A2201
• Study Type: interventional
• Target: 718
• Locations: 1 country
• Sites: 52

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.

Conditions

Dry Eye

Keywords

Dry Eye; Sjogrens; dry eye syndrome (DES); Keratoconjunctivitis sicca (KCS); keratitis; Xerophthalmia; Sjögren's Syndrome; Sjogren's Syndrome

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (3)

• Part 1: Change From Baseline in Central Corneal Fluorescein Staining

  Up to 28 days (Baseline (BL) to end of randomized treatment)

• Part 1: Change From Baseline in Inferior Corneal Fluorescein 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)

  Up to 28 days (Baseline (BL) to end of randomized treatment)

Study Arms (5)

ECF843 0.45 mg/mL TID or vehicle (Part 1)

EXPERIMENTAL

ECF843 0.45 mg/mL TID or vehicle (Part 1)

Drug: ECF843; Other: ECF843 vehicle

ECF843 0.15 mg/mL TID or vehicle (Part 1)

EXPERIMENTAL

ECF843 0.15 mg/mL TID or vehicle (Part 1)

Drug: ECF843; Other: ECF843 vehicle

ECF843 vehicle TID (Part 1)

PLACEBO COMPARATOR

ECF843 vehicle TID (Part 1)

Other: ECF843 vehicle

ECF843 0.15 mg/mL BID or vehicle (Part 1)

EXPERIMENTAL

ECF843 0.15 mg/mL BID or vehicle (Part 1)

Drug: ECF843; Other: ECF843 vehicle

ECF843 vehicle BID (Part 1)

PLACEBO COMPARATOR

ECF843 vehicle BID (Part 1)

Other: ECF843 vehicle

Interventions

ECF843 DRUG

Topical ocular eye drop

ECF843 0.15 mg/mL BID or vehicle (Part 1); ECF843 0.15 mg/mL TID or vehicle (Part 1); ECF843 0.45 mg/mL TID or vehicle (Part 1)

ECF843 vehicle OTHER

Topical ocular eye drop

ECF843 0.15 mg/mL BID or vehicle (Part 1); ECF843 0.15 mg/mL TID or vehicle (Part 1); ECF843 0.45 mg/mL TID or vehicle (Part 1); ECF843 vehicle BID (Part 1); ECF843 vehicle TID (Part 1)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (52)

Arizona Eye Center

Chandler, Arizona, 85224, United States

Location

Carrot Eye Center

Mesa, Arizona, 85202, United States

Location

Phoenix Eye Care and Dry Eye Ctr

Phoenix, Arizona, 85032, United States

Location

Milton Hom OD

Azusa, California, 91702, United States

Location

Orange County Opthamology Med Grp

Garden Grove, California, 92843, United States

Location

Dr Kent Small

Glendale, California, 91203, United States

Location

Global Research Management

Glendale, California, 91205, United States

Location

United Medical Research Institute

Inglewood, California, 90301, United States

Location

Harvard Eye Associates

Laguna Hills, California, 92653, United States

Location

North Valley Eye Medical Group

Mission Hills, California, 91345, United States

Location

The Eye Research Foundation

Newport Beach, California, 92663, United States

Location

North Bay Eye Associates Inc

Petaluma, California, 94954, United States

Location

Martel Eye Medical Group

Rancho Cordova, California, 95670, United States

Location

Sierra Clin Trials Rsch Org

Santa Ana, California, 92705, United States

Location

Vision Institute

Colorado Springs, Colorado, 80907, United States

Location

Advanced Research LLC

Deerfield Beach, Florida, 33064, United States

Location

Pinnacle Research Institute

Fort Lauderdale, Florida, 33309, United States

Location

Eye Associates Of Fort Myers

Fort Myers, Florida, 33901, United States

Location

Lee Shettle Eye & Hearing

Largo, Florida, 33773, United States

Location

Mid Florida Eye Center Pa

Mt. Dora, Florida, 32757, United States

Location

Kannarr Eye Care

Pittsburg, Kansas, 66762, United States

Location

The Eye Care Institute

Louisville, Kentucky, 40206, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

Silverstein Eye Centers

Kansas City, Missouri, 64133, United States

Location

Moyes Eye Center

Kansas City, Missouri, 64154, United States

Location

Tekwani Vision Center

St Louis, Missouri, 63128, United States

Location

Comprehensive Eye Care Limited

Washington, Missouri, 63090, United States

Location

NV Eye Surgery

Henderson, Nevada, 89052, United States

Location

AdvanceMed Clinical Research

Las Vegas, Nevada, 89123, United States

Location

Northern New Jersey Eye Institut PA

South Orange, New Jersey, 07079, United States

Location

Cornerstone Eye Care

High Point, North Carolina, 27262, United States

Location

Eye Care Assoc of Greater Cincinnati Inc

Cincinnati, Ohio, 45236, United States

Location

Abrams Eye Center

Cleveland, Ohio, 44115, United States

Location

Apex Eye Clinical Reserach

Mason, Ohio, 45040, United States

Location

Scott Christie Eye Care Associates

Cranberry Township, Pennsylvania, 16066, United States

Location

Bucci Laser Vision Institute

Wilkes-Barre Township, Pennsylvania, 18702, United States

Location

Vance Thompson Vision Hospital

Sioux Falls, South Dakota, 57108, United States

Location

Chattanooga Eye Institute

Chattanooga, Tennessee, 37411, United States

Location

University Eye Specialists

Maryville, Tennessee, 37803, United States

Location

Total Eye Care PA

Memphis, Tennessee, 38119, United States

Location

Toyos Clinic

Nashville, Tennessee, 37215, United States

Location

Advancing Vision Research LLC

Smyrna, Tennessee, 37167, United States

Location

Texan Eye P A

Austin, Texas, 78731, United States

Location

Intouch Clinical Research Center

Houston, Texas, 77034, United States

Location

Neuro Eye Clinical Trials Inc

Houston, Texas, 77074, United States

Location

Revolution Research

Lakeway, Texas, 78738, United States

Location

Parkhurst NuVision

San Antonio, Texas, 78229, United States

Location

Stacy R Smith MD PC

Salt Lake City, Utah, 84117, United States

Location

Southern Utah Medical Research

St. George, Utah, 84790, United States

Location

Piedmont Eye Center

Lynchburg, Virginia, 24502, United States

Location

Virginia Eye Consultants

Norfolk, Virginia, 23502, United States

Location

Periman Eye Institute

Seattle, Washington, 98119, United States

Location

MeSH Terms

Conditions

Dry Eye Syndromes; Keratoconjunctivitis Sicca; Keratitis; Xerophthalmia; Sjogren's Syndrome

Condition Hierarchy (Ancestors)

Lacrimal Apparatus Diseases; Eye Diseases; Keratoconjunctivitis; Conjunctivitis; Conjunctival Diseases; Corneal Diseases; Arthritis, Rheumatoid; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Xerostomia; Salivary Gland Diseases; Mouth Diseases; Stomatognathic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@Novartis.com

1 862 778 8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Double-masked
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 13, 2020
• First Posted: May 18, 2020
• Study Start: October 6, 2020
• Primary Completion: May 13, 2021
• Study Completion: May 13, 2021
• Last Updated: January 28, 2025
• Results First Posted: May 26, 2022

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share

Locations

US (52)