Dry EyePhase 2NCT04391894
A Study to Assess the Safety and Efficacy of ECF843 vs Vehicle in Subjects With Dry Eye Disease
A Phase 2 study testing ECF843 in people with Dry Eye, run by Novartis Pharmaceuticals at 52 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Dry Eye
- What is being tested?
- ECF843
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 718 participants
- Where
- 52 sitesUnited States
- Study dates
- 6 Oct 2020to 13 May 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 14Show fewer
- 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
Trial locations and contacts
52 sites in 1 country.
52 sites in 1 country
United States52 sites
Texan Eye P A
Austin, Texas, 78731
Milton Hom OD
Azusa, California, 91702
Tufts Medical Center
Boston, Massachusetts, 02111
Arizona Eye Center
Chandler, Arizona, 85224
Chattanooga Eye Institute
Chattanooga, Tennessee, 37411
Eye Care Assoc of Greater Cincinnati Inc
Cincinnati, Ohio, 45236
Abrams Eye Center
Cleveland, Ohio, 44115
Vision Institute
Colorado Springs, Colorado, 80907
Scott Christie Eye Care Associates
Cranberry Township, Pennsylvania, 16066
Advanced Research LLC
Deerfield Beach, Florida, 33064
Pinnacle Research Institute
Fort Lauderdale, Florida, 33309
Eye Associates Of Fort Myers
Fort Myers, Florida, 33901
Orange County Opthamology Med Grp
Garden Grove, California, 92843
Dr Kent Small
Glendale, California, 91203
Global Research Management
Glendale, California, 91205
NV Eye Surgery
Henderson, Nevada, 89052
Cornerstone Eye Care
High Point, North Carolina, 27262
Intouch Clinical Research Center
Houston, Texas, 77034
Neuro Eye Clinical Trials Inc
Houston, Texas, 77074
United Medical Research Institute
Inglewood, California, 90301
Silverstein Eye Centers
Kansas City, Missouri, 64133
Moyes Eye Center
Kansas City, Missouri, 64154
Harvard Eye Associates
Laguna Hills, California, 92653
Revolution Research
Lakeway, Texas, 78738
Lee Shettle Eye & Hearing
Largo, Florida, 33773
AdvanceMed Clinical Research
Las Vegas, Nevada, 89123
The Eye Care Institute
Louisville, Kentucky, 40206
Piedmont Eye Center
Lynchburg, Virginia, 24502
University Eye Specialists
Maryville, Tennessee, 37803
Apex Eye Clinical Reserach
Mason, Ohio, 45040
Total Eye Care PA
Memphis, Tennessee, 38119
Carrot Eye Center
Mesa, Arizona, 85202
North Valley Eye Medical Group
Mission Hills, California, 91345
Mid Florida Eye Center Pa
Mt. Dora, Florida, 32757
Toyos Clinic
Nashville, Tennessee, 37215
The Eye Research Foundation
Newport Beach, California, 92663
Virginia Eye Consultants
Norfolk, Virginia, 23502
North Bay Eye Associates Inc
Petaluma, California, 94954
Phoenix Eye Care and Dry Eye Ctr
Phoenix, Arizona, 85032
Kannarr Eye Care
Pittsburg, Kansas, 66762
Martel Eye Medical Group
Rancho Cordova, California, 95670
Stacy R Smith MD PC
Salt Lake City, Utah, 84117
Parkhurst NuVision
San Antonio, Texas, 78229
Sierra Clin Trials Rsch Org
Santa Ana, California, 92705
Periman Eye Institute
Seattle, Washington, 98119
Vance Thompson Vision Hospital
Sioux Falls, South Dakota, 57108
Advancing Vision Research LLC
Smyrna, Tennessee, 37167
Northern New Jersey Eye Institut PA
South Orange, New Jersey, 07079
Tekwani Vision Center
St Louis, Missouri, 63128
Southern Utah Medical Research
St. George, Utah, 84790
Comprehensive Eye Care Limited
Washington, Missouri, 63090
Bucci Laser Vision Institute
Wilkes-Barre Township, Pennsylvania, 18702
About this study
Official title: 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.
Read the 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.
- Conditions
- Dry Eye
- Being tested
- ECF843, ECF843 vehicle
- Keywords
- Dry Eye, Sjogrens, dry eye syndrome (DES), Keratoconjunctivitis sicca (KCS), keratitis, Xerophthalmia, Sjögren's Syndrome, Sjogren's Syndrome
- Medical subject headings
- Dry Eye Syndromes, Keratoconjunctivitis Sicca, Keratitis, Xerophthalmia, Sjogren's Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ECF843Drug
Topical ocular eye drop
ECF843 vehicleOther
Topical ocular eye drop
| Study group | Role | What they receive |
|---|---|---|
| ECF843 0.45 mg/mL TID or vehicle (Part 1) | Experimental | ECF843 0.45 mg/mL TID or vehicle (Part 1) Receives: 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) Receives: Drug: ECF843; Other: ECF843 vehicle |
| ECF843 vehicle TID (Part 1) | Placebo comparator | ECF843 vehicle TID (Part 1) Receives: Other: ECF843 vehicle |
| ECF843 0.15 mg/mL BID or vehicle (Part 1) | Experimental | ECF843 0.15 mg/mL BID or vehicle (Part 1) Receives: Drug: ECF843; Other: ECF843 vehicle |
| ECF843 vehicle BID (Part 1) | Placebo comparator | ECF843 vehicle BID (Part 1) Receives: Other: ECF843 vehicle |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Up to 28 days (Baseline (BL) to end of randomized treatment)
Secondary outcomes (3)
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.
Measured: 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.
Measured: 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.
Measured: Up to 28 days (Baseline (BL) to end of randomized treatment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2020
- Study start6 Oct 2020
- Primary completion13 May 2021
- Study completion13 May 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Dry EyeStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 718 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04391894
- Sponsor's protocol ID
- CECF843A2201
- Status last verified
- Jan 2025
- First submitted
- 13 May 2020
- Last updated
- 28 Jan 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 28 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-masked, Multicenter Study to Evaluate the Safety and Efficacy of ECF843 vs Vehicle in Subjects With Dry Eye Disease
- ClinicalTrials.gov ID
- NCT04391894
- Acronym / sponsor ID
- CECF843A2201
- Conditions
- Dry Eye
- Keywords
- Dry Eye, Sjogrens, dry eye syndrome (DES), Keratoconjunctivitis sicca (KCS), keratitis, Xerophthalmia, Sjögren's Syndrome, Sjogren's Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 718 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 6 Oct 2020 (actual)
- Primary completion
- 13 May 2021 (actual)
- Study completion
- 13 May 2021 (actual)
- First posted
- 18 May 2020 (actual)
- Results
- Posted 26 May 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| ECF843 0.45 mg/mL TID or vehicle (Part 1) | Experimental | Drug: ECF843, Other: ECF843 vehicleECF843 0.45 mg/mL TID or vehicle (Part 1) |
| ECF843 0.15 mg/mL TID or vehicle (Part 1) | Experimental | Drug: ECF843, Other: ECF843 vehicleECF843 0.15 mg/mL TID or vehicle (Part 1) |
| ECF843 vehicle TID (Part 1) | Placebo comparator | Other: ECF843 vehicleECF843 vehicle TID (Part 1) |
| ECF843 0.15 mg/mL BID or vehicle (Part 1) | Experimental | Drug: ECF843, Other: ECF843 vehicleECF843 0.15 mg/mL BID or vehicle (Part 1) |
| ECF843 vehicle BID (Part 1) | Placebo comparator | Other: ECF843 vehicleECF843 vehicle BID (Part 1) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ECF843Topical ocular eye drop | Drug | 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 vehicleTopical ocular eye drop | Other | 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) |
Primary outcome
Part 1: Change From Baseline in Symptom Assessment in Dry Eye (SANDE) Score
Time frame: Up to 28 days (Baseline (BL) to end of randomized treatment)
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
Time frame: Up to 28 days (Baseline (BL) to end of randomized treatment)
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.
3 secondary outcomes
Part 1: Change From Baseline in Central Corneal Fluorescein Staining
Time frame: Up to 28 days (Baseline (BL) to end of randomized treatment)
Part 1: Change From Baseline in Inferior Corneal Fluorescein Staining
Time frame: 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)
Time frame: Up to 28 days (Baseline (BL) to end of randomized treatment)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jan 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
52 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ECF843 in people with Dry Eye, run by Novartis Pharmaceuticals at 52 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 52 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 May 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part 1: Change From Baseline in Symptom Assessment in Dry Eye (SANDE) Score (time frame: Up to 28 days (Baseline (BL) to end of randomized treatment)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jan 2025.