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

NCT ID: NCT03019627

Last Updated: 2019-02-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 150 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-01-20
• Study Completion Date: 2017-08-31

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.

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.

Conditions

Dry Eye

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

rhNGF 20μg/mL

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

Group Type: EXPERIMENTAL

NGF

Intervention Type: DRUG

Eye Drop 20 μg/mL

Vehicle

vehicle eye drops six times daily

Group Type: PLACEBO_COMPARATOR

Vehicle

Intervention Type: OTHER

Vehicle Eye Drop

Interventions

NGF

Eye Drop 20 μg/mL

Intervention Type: DRUG

Vehicle

Vehicle Eye Drop

Intervention Type: OTHER

Other Intervention Names

cenegermin; Placebo

Eligibility Criteria

Inclusion Criteria

1. Patients (male or female) must be ≥ 18 years of age.

2. 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.

3. Patients must present dry eye pathology characterized by the following clinical features:

1. Corneal and/or conjunctival staining with fluorescein and lissamine green using National Eye Institute (NEI) grading system > 3

2. Mean Symptom Assessment in Dry Eye (SANDE) questionnaire ≥30

3. Schirmer test without anesthesia < 10 mm/5 minutes and/or tear film break-up time (TFBUT) < 10 seconds in the study eye

4. The same eye (study eye) must fulfill all the above criteria.

5. Patients must have best corrected distance visual acuity (BCDVA) score of ≥ 0.1 decimal units in both eyes at the time of study enrollment.

6. Female patients must have negative pregnancy test if at childbirth potential.

7. 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.

8. Patients must have the ability and willingness to comply with study procedures.

Exclusion Criteria

1. Best corrected distance visual acuity (BCDVA) score of < 0.1 decimal units in either eye.

2. Evidence of an active ocular infection in either eye.

3. 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.

4. Intraocular inflammation defined as Tyndall score >0.

5. Active or recent diagnosis of malignancy (i.e., currently under chemo/radiotherapy).

6. 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.

7. 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).

8. 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.

10. 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).

11. 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:

1. are currently pregnant or,

2. have a positive result at the urine pregnancy test (Baseline/Day 0) or,

3. intend to become pregnant during the study treatment period or,

4. are breast-feeding or,

5. 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.

12. History of drug addiction or alcohol abuse.

13. Any prior ocular surgery (including refractive palpebral and cataract surgery) if within 90 days before the screening visit.

14. Participation in a clinical trial with a new active substance during the past 30 days.

15. Participation in another clinical trial study at the same time as the present study.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Dompé Farmaceutici S.p.A

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Giacomina Massaro Giordano, MD

Role: PRINCIPAL_INVESTIGATOR

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

Locations

Penn Dry Eye and Ocular Surface Center, University of Pennsylvania

Philadelphia, Pennsylvania, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

NGF0216

Identifier Type: -

Identifier Source: org_study_id