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

Primary Purpose

Dry Eye

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

ECF843

ECF843 vehicle

About this trial

This is an interventional treatment trial for Dry Eye focused on measuring Dry Eye, Sjogrens, dry eye syndrome (DES), Keratoconjunctivitis sicca (KCS), keratitis, Xerophthalmia, Sjögren's Syndrome, Sjogren's Syndrome

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Placebo Comparator

Experimental

Placebo Comparator

Arm Label

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

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

ECF843 vehicle TID (Part 1)

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

ECF843 vehicle BID (Part 1)

Arm Description

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

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

ECF843 vehicle TID (Part 1)

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

ECF843 vehicle BID (Part 1)

Outcomes

Primary Outcome Measures

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.

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.

Secondary Outcome Measures

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.

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.

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.

Full Information

NCT ID

NCT04391894

First Posted

May 13, 2020

Last Updated

January 27, 2023

Sponsor

Novartis Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT04391894

Brief Title

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

2. Study Status

Record Verification Date

January 2023

Overall Recruitment Status

Completed

Study Start Date

October 6, 2020 (Actual)

Primary Completion Date

May 13, 2021 (Actual)

Study Completion Date

May 13, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Dry Eye

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigator

Masking Description

Double-masked

Allocation

Randomized

Enrollment

558 (Actual)

8. Arms, Groups, and Interventions

Arm Title

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

Arm Type

Experimental

Arm Description

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

Arm Title

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

Arm Type

Experimental

Arm Description

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

Arm Title

ECF843 vehicle TID (Part 1)

Arm Type

Placebo Comparator

Arm Description

ECF843 vehicle TID (Part 1)

Arm Title

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

Arm Type

Experimental

Arm Description

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

Arm Title

ECF843 vehicle BID (Part 1)

Arm Type

Placebo Comparator

Arm Description

ECF843 vehicle BID (Part 1)

Intervention Type

Drug

Intervention Name(s)

ECF843

Intervention Description

Topical ocular eye drop

Intervention Type

Other

Intervention Name(s)

ECF843 vehicle

Intervention Description

Topical ocular eye drop

Primary Outcome Measure Information:

Title

Part 1: Change From Baseline in Symptom Assessment in Dry Eye (SANDE) Score

Description

Time Frame