An 8-week Study to Evaluate Safety and Efficacy of NGF Eye Drops Solution Versus Vehicle in Patients With Dry Eye

NCT03019627 | Completed Phase 2 Results FDA Drug

• 1 other identifier: NGF0216
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 1

Brief 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 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.

Conditions

Dry Eye

Outcome Measures

Primary Outcomes (1)

• 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.

  week 8

Secondary Outcomes (5)

• SANDE Scores

  Week 4, week 8, week 12

• Cornea Vital Staining

  week 4, week 8, week 12

• Conjunctival Vital Staining

  week 4, week 8, week 12

• Change in Tear Film Break-up Time (TFBUT)

  week 4, week 8, week 12

• Change From Baseline in Wetting Distance

  week 8

Study Arms (2)

rhNGF 20μg/mL

EXPERIMENTAL

Recombinant Human Nerve Growth Factor (rhNGF) at 20 μg/mL eye drops six times daily

Drug: NGF

Vehicle

PLACEBO COMPARATOR

vehicle eye drops six times daily

Other: Vehicle

Interventions

NGF DRUG

Eye Drop 20 μg/mL

Also known as: cenegermin

rhNGF 20μg/mL

Vehicle OTHER

Vehicle Eye Drop

Also known as: Placebo

Vehicle

Eligibility Criteria

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

You may qualify if:

• 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
• 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.

You may not qualify if:

• 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.
• 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.
• 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.

Sponsors & Collaborators

• Dompé Farmaceutici S.p.A: lead

Study Sites (1)

Penn Dry Eye and Ocular Surface Center, University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Dry Eye Syndromes

Interventions

Nerve Growth Factor; cenegermin

Condition Hierarchy (Ancestors)

Lacrimal Apparatus Diseases; Eye Diseases

Intervention Hierarchy (Ancestors)

Nerve Growth Factors; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Nerve Tissue Proteins; Biological Factors

Limitations and Caveats

LImitations and caveats are not specified.

Results Point of Contact

• Title: Valeria Motta, PhD
• Organization: Dompé

info@dompe.it

02583831

Study Officials

• Giacomina Massaro Giordano, MD

  Penn Dry Eye and Oc. Surf. Center, Univ. of Pennsylvania, Scheie Eye Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: The identity of the treatments was concealed from the patient, Investigator, site staff, and Dompé's clinical research personnel until the study was unmasked for the final statistical analysis (after data base lock) except in case of specific events that required unmasking of the patient.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 11, 2017
• First Posted: January 12, 2017
• Study Start: January 20, 2017
• Primary Completion: August 31, 2017
• Study Completion: August 31, 2017
• Last Updated: February 8, 2019
• Results First Posted: February 8, 2019

Record last verified: 2018-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)