Dry EyePhase 2NCT03019627
An 8-week Study to Evaluate Safety and Efficacy of NGF Eye Drops Solution Versus Vehicle in Patients With Dry Eye
A Phase 2 study testing NGF in people with Dry Eye, run by Dompé Farmaceutici S.p.A at 1 site in United States.
Trial summary
The primary objective of this study was to assess the efficacy and safety of rhNGF when administered as eye drops to patients with dry eye The secondary objectives of this study were: To assess change from baseline in Symptom Assessment In Dry Eye (SANDE) scores (without imputation), corneal and conjunctival staining… 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?
- NGF
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Jan 2017to 31 Aug 2017
- 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
- Patients (male or female) must be ≥ 18 years of age.
- Patients must be diagnosed with any type of dry eye (e.g. Meibomian Gland Dysfunction, Blepharitis, Keratoconjunctivitis sicca etc) at least 3 months before enrollment.
- Patients must present dry eye pathology characterized by the following clinical features:
- Corneal and/or conjunctival staining with fluorescein and lissamine green using National Eye Institute (NEI) grading system > 3
- Mean Symptom Assessment in Dry Eye (SANDE) questionnaire ≥30
- Schirmer test without anesthesia < 10 mm/5 minutes and/or tear film break-up time (TFBUT) < 10 seconds in the study eye
Show all 11Show fewer
- The same eye (study eye) must fulfill all the above criteria.
- Patients must have best corrected distance visual acuity (BCDVA) score of ≥ 0.1 decimal units in both eyes at the time of study enrollment.
- Female patients must have negative pregnancy test if at childbirth potential.
- Only patients who satisfy all requirements for informed consent may be included in the study. Written Informed Consent must be obtained before the initiation of any study specific procedures.
- Patients must have the ability and willingness to comply with study procedures.
Who cannot join
- Best corrected distance visual acuity (BCDVA) score of < 0.1 decimal units in either eye.
- Evidence of an active ocular infection in either eye.
- Presence or history of any ocular disorder or condition, including ocular surgery, trauma, or disease that could possibly interfere with the interpretation of study results in the opinion of the Investigator.
- Intraocular inflammation defined as Tyndall score >0.
- Active or recent diagnosis of malignancy (i.e., currently under chemo/radiotherapy).
- Systemic disease not stabilized within 1 month before baseline visit (e.g., uncontrolled diabetes; thyroid malfunction) or judged by the Investigator to be incompatible with the study (e.g., current systemic infections) or with a condition incompatible with the frequent assessment required by the study.
Show all 20Show fewer
- Patients who have had a serious adverse reaction or significant hypersensitivity to any drug or chemically related compounds, or had a clinically significant allergy to drugs, foods, amide local anesthetics, or other materials, including commercial artificial tears containing carboxymethylcellulose (CMC) (in the opinion of the Investigator).
- Use of topical cyclosporine, topical corticosteroids, or any other topical medication for the treatment of dry eye in either eye until the day of study enrollment.
- Contact lenses or punctal plug use during the study (previous use not an exclusion criteria, but must be discontinued at the baseline visit.
- An anticipated need of additional systemic treatments for dry eye during the study (all prior treatment must be continued for the entire duration of the study).
- Females of childbearing potential (those who are not surgically sterilized or postmenopausal for at least 1 year) are excluded from participation in the study if they meet any one of the following conditions:
- are currently pregnant or,
- have a positive result at the urine pregnancy test (Baseline/Day 0) or,
- intend to become pregnant during the study treatment period or,
- are breast-feeding or,
- are not willing to use highly effective birth control measures, such as: hormonal contraceptives - oral, implanted, transdermal, or injected - and/or mechanical barrier methods - spermicide in conjunction with a barrier such as a condom or diaphragm or a intrauterine device (IUD) - during the entire course of and 30 days after the study treatment periods.
- History of drug addiction or alcohol abuse.
- Any prior ocular surgery (including refractive palpebral and cataract surgery) if within 90 days before the screening visit.
- Participation in a clinical trial with a new active substance during the past 30 days.
- Participation in another clinical trial study at the same time as the present study.
Trial locations and contacts
1 site in 1 country.
United States1 site
Penn Dry Eye and Ocular Surface Center, University of Pennsylvania
Philadelphia, Pennsylvania, 19104
About this study
Official title: An 8-week, Phase II, Single-center, Randomized, Double-masked, Vehicle-controlled, Parallel-group Study With 4 Weeks of Follow-up to Evaluate Safety and Efficacy of rhNGF Eye Drops Solution vs Vehicle in Patients With Dry Eye
The primary objective of this study was to assess the efficacy and safety of rhNGF when administered as eye drops to patients with dry eye
The secondary objectives of this study were:
- To assess change from baseline in Symptom Assessment In Dry Eye (SANDE) scores (without imputation), corneal and conjunctival staining according to National Eye Institute (NEI) scale, and in Tear Film Break-up Time (TFBUT) and Schirmer test I, following 4 and 8 weeks of treatment.
- To assess change in levels of inflammatory biomarker matrix metallopeptidase 9 (MMP-9) in tears following 8 weeks of treatment.
- To assess the incidence and frequency of treatment-emergent adverse events (TEAEs) following 8 weeks of treatment.
Read the detailed description
The proposed phase II study is a single-center, randomized, double-masked, parallel-arm, vehicle-controlled trial, designed to evaluate the safety and efficacy of Recombinant Human Nerve Growth Factor (rhNGF) eye drops at 20 μg/ml concentration administered six times daily for 8 weeks in patients with dry eye. After confirmation of inclusion and exclusion criteria all eligible patients will be randomized at 2:1 ratio to rhNGF or vehicle control treatment with 8 weeks of study treatments administration with 4 weeks Follow-up.
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NGFDrug
Also called cenegermin
Eye Drop 20 μg/mL
VehicleOther
Also called Placebo
Vehicle Eye Drop
| Study group | Role | What they receive |
|---|---|---|
| rhNGF 20μg/mL | Experimental | Recombinant Human Nerve Growth Factor (rhNGF) at 20 μg/mL eye drops six times daily Receives: Drug: NGF |
| Vehicle | Placebo comparator | vehicle eye drops six times daily Receives: Other: Vehicle |
What is being measured
The primary outcome is the main question the study is designed to answer.
Symptom Assessment in Dry Eye (SANDE) Scores
Change from baseline with Last Observation Carried Forward (LOCF) imputation. LOCF is a method of imputing missing data in longitudinal studies and tests for difference in mean rates of change in controlled repeated measurements designs with dropouts.
The SANDE score is calculated by taking the square root of the product of the frequency of symptoms score and the severity of symptoms score. The SANDE scale ranges from 0 to 100 with 100 being the maximal amount of dry eye symptoms and 0 being the minimal amount of dry eye symptoms.
Measured: week 8
Secondary outcomes (5)
SANDE Scores
Change from baseline with Last Observation Carried Forward (LOCF) imputation. LOCF is a method of imputing missing data in longitudinal studies and tests for difference in mean rates of change in controlled repeated measurements designs with dropouts.
The SANDE score is calculated by taking the square root of the product of the frequency of symptoms score and the severity of symptoms score. The SANDE scale ranges from 0 to 100 with 100 being the maximal amount of dry eye symptoms and 0 being the minimal amount of dry eye symptoms.
Measured: Week 4, week 8, week 12
Cornea Vital Staining
Changes from baseline on National Eye Institute (NEI) scale. The NEI/Industry Workshop guidelines were used for grading the scale of corneal and conjunctival damage. The cornea was divided into five sectors (central, superior, inferior, nasal and temporal), each of which was scored on a scale of 0-3, with a maximal total corneal staining score of 15.
Both nasally and temporally, the conjunctiva was divided into a superior paralimbal area, an inferior paralimbal area, and a peripheral area with a grading scale of 0-3 and with a maximal total score of 9 for the nasal and temporal conjunctiva (overall the total score ranged from 0-18).
Briefly, grade 0 reflected normal/healthy situation, whereas grade 3 reflected a severe damage in the considered sector.
Measured: week 4, week 8, week 12
Conjunctival Vital Staining
Changes from baseline on National Eye Institute (NEI) scale. The NEI/Industry Workshop guidelines were used for grading the scale of corneal and conjunctival damage. The cornea was divided into five sectors (central, superior, inferior, nasal and temporal), each of which was scored on a scale of 0-3, with a maximal total corneal staining score of 15.
Both nasally and temporally, the conjunctiva was divided into a superior paralimbal area, an inferior paralimbal area, and a peripheral area with a grading scale of 0-3 and with a maximal total score of 9 for the nasal and temporal conjunctiva (overall the total score ranged from 0-18).
Briefly, grade 0 reflected normal/healthy situation, whereas grade 3 reflected a severe damage in the considered sector.
Measured: week 4, week 8, week 12
Change in Tear Film Break-up Time (TFBUT)
TFBUT was measured by determining the time to tear break-up. The TFBUT was performed after instillation of 5 μL of 2% preservative-free sodium fluorescein solution into the inferior conjunctival cul-de-sac of each eye. The patient was instructed to blink several times to thoroughly mix the fluorescein with the tear film.
Measured: week 4, week 8, week 12
Change From Baseline in Wetting Distance
The Schirmer test without anesthesia was performed to measure aqueous tear secretion prior to the instillation of any dilating or eye drops
Measured: week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2017 (estimated)
- Study start20 Jan 2017
- Primary completion31 Aug 2017
- Study completion31 Aug 2017
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.
- 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
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 150 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dompé Farmaceutici S.p.A
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Giacomina Massaro Giordano, MD, Principal investigator, Penn Dry Eye and Oc. Surf. Center, Univ. of Pennsylvania, Scheie Eye Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03019627
- Sponsor's protocol ID
- NGF0216
- Status last verified
- Oct 2018
- First submitted
- 11 Jan 2017
- Last updated
- 8 Feb 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An 8-week, Phase II, Single-center, Randomized, Double-masked, Vehicle-controlled, Parallel-group Study With 4 Weeks of Follow-up to Evaluate Safety and Efficacy of rhNGF Eye Drops Solution vs Vehicle in Patients With Dry Eye
- ClinicalTrials.gov ID
- NCT03019627
- Acronym / sponsor ID
- NGF0216
- Conditions
- Dry Eye
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 150 participants (actual)
- Lead sponsor
- Dompé Farmaceutici S.p.AIndustry (pharma, biotech, devices)
- Study start
- 20 Jan 2017 (actual)
- Primary completion
- 31 Aug 2017 (actual)
- Study completion
- 31 Aug 2017 (actual)
- First posted
- 12 Jan 2017 (estimated)
- Results
- Posted 8 Feb 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study was to assess the efficacy and safety of rhNGF when administered as eye drops to patients with dry eye
The secondary objectives of this study were:
- To assess change from baseline in Symptom Assessment In Dry Eye (SANDE) scores (without imputation), corneal and conjunctival staining according to National Eye Institute (NEI) scale, and in Tear Film Break-up Time (TFBUT) and Schirmer test I, following 4 and 8 weeks of treatment.
- To assess change in levels of inflammatory biomarker matrix metallopeptidase 9 (MMP-9) in tears following 8 weeks of treatment.
- To assess the incidence and frequency of treatment-emergent adverse events (TEAEs) following 8 weeks of treatment.
Detailed description
The proposed phase II study is a single-center, randomized, double-masked, parallel-arm, vehicle-controlled trial, designed to evaluate the safety and efficacy of Recombinant Human Nerve Growth Factor (rhNGF) eye drops at 20 μg/ml concentration administered six times daily for 8 weeks in patients with dry eye. After confirmation of inclusion and exclusion criteria all eligible patients will be randomized at 2:1 ratio to rhNGF or vehicle control treatment with 8 weeks of study treatments administration with 4 weeks Follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| rhNGF 20μg/mL | Experimental | Drug: NGFRecombinant Human Nerve Growth Factor (rhNGF) at 20 μg/mL eye drops six times daily |
| Vehicle | Placebo comparator | Other: Vehiclevehicle eye drops six times daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Patients (male or female) must be ≥ 18 years of age.
- Patients must be diagnosed with any type of dry eye (e.g. Meibomian Gland Dysfunction, Blepharitis, Keratoconjunctivitis sicca etc) at least 3 months before enrollment.
- Patients must present dry eye pathology characterized by the following clinical features:
- The same eye (study eye) must fulfill all the above criteria.
- Patients must have best corrected distance visual acuity (BCDVA) score of ≥ 0.1 decimal units in both eyes at the time of study enrollment.
Key exclusion criteria (20 listed)
- Best corrected distance visual acuity (BCDVA) score of < 0.1 decimal units in either eye.
- Evidence of an active ocular infection in either eye.
- Presence or history of any ocular disorder or condition, including ocular surgery, trauma, or disease that could possibly interfere with the interpretation of study results in the opinion of the Investigator.
- Intraocular inflammation defined as Tyndall score >0.
- Active or recent diagnosis of malignancy (i.e., currently under chemo/radiotherapy).
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 |
|---|---|---|
| NGFAlso: cenegerminEye Drop 20 μg/mL | Drug | rhNGF 20μg/mL |
| VehicleAlso: PlaceboVehicle Eye Drop | Other | Vehicle |
Primary outcome
Symptom Assessment in Dry Eye (SANDE) Scores
Time frame: week 8
Change from baseline with Last Observation Carried Forward (LOCF) imputation. LOCF is a method of imputing missing data in longitudinal studies and tests for difference in mean rates of change in controlled repeated measurements designs with dropouts. The SANDE score is calculated by taking the square root of the product of the frequency of symptoms score and the severity of symptoms score. The SANDE scale ranges from 0 to 100 with 100 being the maximal amount of dry eye symptoms and 0 being the minimal amount of dry eye symptoms.
5 secondary outcomes
SANDE Scores
Time frame: Week 4, week 8, week 12
Cornea Vital Staining
Time frame: week 4, week 8, week 12
Conjunctival Vital Staining
Time frame: week 4, week 8, week 12
Change in Tear Film Break-up Time (TFBUT)
Time frame: week 4, week 8, week 12
Change From Baseline in Wetting Distance
Time frame: week 8
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Dry Eye
Not yet recruiting
TearCare in Young Adults
Meibomian Gland Dysfunction (Disorder)
RecruitingPhase 1/2
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Dry Age-Related Macular Degeneration, Geographic Atrophy
Recruiting
Efficacy and Safety of BUFY01 Versus SVS20 in the Treatment of Dry Eye Disease
Dry Eye Disease
Not yet recruitingPhase 1
A Clinical Study to Evaluate the Safety and Effectiveness of MSCohi-O Lens in the Treatment of Immune-related Ocular Surface Diseases.
Keratitis, Autoimmunity
Recruiting
Treating Contact Lens Discomfort With Cyclosporine 0.1% Ophthalmic Solution
Dry Eye Disease (DED), Contact Lens Discomfort (CLD)
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.
Giacomina Massaro Giordano, MD principal investigator
Penn Dry Eye and Oc. Surf. Center, Univ. of Pennsylvania, Scheie Eye Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NGF in people with Dry Eye, run by Dompé Farmaceutici S.p.A at 1 site 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 11 inclusion and 20 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 8 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 8 Feb 2019). 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?
Symptom Assessment in Dry Eye (SANDE) Scores (time frame: week 8). 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 8 Feb 2019.