A Study to Evaluate Safety and Efficacy of rhNGF Eye Solution vs Vehicle in Patients With Moderate to Severe Dry Eye

NCT03982368 | Completed Phase 2 Results FDA Drug

• 1 other identifier: NGF0118
• Study Type: interventional
• Target: 261
• Locations: 1 country
• Sites: 12

Brief Summary

The objective of this study is to assess the efficacy and safety of rhNGF eye drops at 20 μg/ml concentration administered two or three times daily for 4 weeks in patients with moderate to severe dry eye.

Conditions

Dry Eye Syndrome

Outcome Measures

Primary Outcomes (2)

• Change From Baseline in Schirmer I Test (Without Anesthesia) vs Week 4 - Full Analysis Set

  The Schirmer test is used in ophthalmic examination to measure tear production for the diagnosis of conditions such as keratoconjunctivitis sicca and dry eye. Without previously instilling anesthetic drops, the Schirmer strip is inserted into the lower conjunctival sac at the junction of the lateral and middle thirds, avoiding touching the cornea, and the length of wetting strips in millimeters is recorded after 5 minutes. The patients will be instructed to close their eyes gently. After 5 minutes have elapsed, the Schirmer test strip will be removed and the length of the tear absorption on the strip will be measured (millimeters/5 minutes) Cutoff values: \<5 mm - pathologic dry eye 5-10 mm - marginal dry eye \>10 and \<30 mm - normal secretion

  Baseline, week 4

• Change From Baseline in Schirmer I Test (Without Anesthesia) vs Week 4 - Per Protocol Set

  The Schirmer test is used in ophthalmic examination to measure tear production for the diagnosis of conditions such as keratoconjunctivitis sicca and dry eye. Without previously instilling anesthetic drops, the Schirmer strip is inserted into the lower conjunctival sac at the junction of the lateral and middle thirds, avoiding touching the cornea, and the length of wetting strips in millimeters is recorded after 5 minutes. The patients will be instructed to close their eyes gently. After 5 minutes have elapsed, the Schirmer test strip will be removed and the length of the tear absorption on the strip will be measured (millimeters/5 minutes) Cutoff values: \<5 mm - pathologic dry eye 5-10 mm - marginal dry eye \>10 and \<30 mm - normal secretion

  Baseline, week 4

Secondary Outcomes (18)

• Change From Baseline in Symptoms Questionnaire (SANDE) Scores for Severity vs Week 4 - Full Analysis Set

  Baseline, week 4

• Change From Baseline in Symptoms Questionnaire (SANDE) Scores for Severity vs Week 4 - Per Protocol Set

  Baseline, week 4

• Change From Baseline in Symptoms Questionnaire (SANDE) Scores for Frequency vs Week 4 - Full Analysis Set

  Baseline, week 4

• Change From Baseline in Symptoms Questionnaire (SANDE) Scores for Frequency vs Week 4 - Per Protocol Set

  Baseline, week 4

• Change From Baseline in Schirmer II Test (With Anesthesia) vs Week 4 - Full Analysis Set

  Baseline, week 4

Study Arms (3)

rhNGF 20 μg/ml TID

EXPERIMENTAL

One drop of rhNGF 20 μg/ml will be instilled in both eyes three times daily (every 6-8 hours)

Drug: rhNGF 20 μg/ml

rhNGF 20 μg/ml BID + vehicle OD

EXPERIMENTAL

One drop of rhNGF 20 μg/ml will be instilled in both eyes two times daily (BID) plus one drop (40 μL) of vehicle will be instilled in both eyes once daily (OD) (every 6-8 hours)

Drug: rhNGF 20 μg/ml + vehicle

Vehicle TID

PLACEBO COMPARATOR

Vehicle eye one drop will be instilled in both eyes three times daily (every 6-8 hours)

Other: Vehicle

Interventions

rhNGF 20 μg/ml DRUG

one drop of rhNGF 20 μg/ml will be instilled in both eyes three times daily (every 6-8 hours)

Also known as: cenegermin

rhNGF 20 μg/ml TID

rhNGF 20 μg/ml + vehicle DRUG

one drop of rhNGF 20 μg/ml will be instilled in both eyes two times daily plus one drop (40 μL) of vehicle will be instilled in both eyes once daily (every 6-8 hours). rhNGF will be instilled in the morning and in the evening while the vehicle will be instilled in the afternoon.

Also known as: cenegermin + placebo

rhNGF 20 μg/ml BID + vehicle OD

Vehicle OTHER

one drop of vehicle will be instilled in both eyes three times daily (every 6-8 hours)

Also known as: placebo

Vehicle TID

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female aged ≥ 18 years
• Patients with moderate to severe dry eye characterized by the following clinical features:
• Corneal and/or conjunctival staining with fluorescein using National Eye Institute (NEI) grading system \> 3
• SANDE questionnaire \>25 mm
• Schirmer test I (without anaesthesia) \>2mm \<10 mm/5 minutes
• Tear film break-up time (TFBUT) \< 10 seconds in the worse eye
• The same eye (eligible eye) must fulfill all the above criteria
• Patients diagnosed with dry eye at least 6 months before enrolment (current use or recommended use of artificial tears for the treatment of Dry Eye)
• Best corrected distance visual acuity (BCDVA) score of ≥ 0.1 decimal units (20/200 Snellen value) in both eyes at the time of study enrolment
• If a female of childbearing potential, have a negative pregnancy test
• Only patients who satisfy all Informed Consent requirements may be included in the study. The patient and/or his/her legal representative must read, sign and date the Informed Consent document before any study-related procedures are performed. The Informed Consent form signed by patients and/or legal representative must have been approved by the Institutional Review Board (IRB) / Independent Ethics Committee (IEC) for the current study
• Patients must have the ability and willingness to comply with study procedures.

You may not qualify if:

• Inability to speak and understand the local language sufficiently to understand the nature of the study, to provide written informed consent, and to allow the completion of all study assessments;
• Evidence of an active ocular infection, in either eye
• Presence of any other ocular disorder or condition requiring topical medication during the entire duration of study
• History of severe systemic allergy or of ocular allergy (including seasonal conjunctivitis) or chronic conjunctivitis and/or keratitis other than dry eye
• Intraocular inflammation defined as Tyndall score \>0
• History of malignancy in the last 5 years
• Systemic disease not stabilized within 1 month before Screening Visit (e.g. diabetes with glycemia out of range, 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
• Patient 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 anaesthetics or other materials including commercial artificial tears (in the opinion of the investigator)
• Females of childbearing potential (those who are not surgically sterilized or post-menopausal 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 Intra Uterine Device (IUD) - during the entire course of and 30 days after the study treatment periods
• Any concurrent medical condition, that in the judgment of the PI, might interfere with the conduct of the study, confound the interpretation of the study results, or endanger the patient's well-being

Sponsors & Collaborators

• Dompé Farmaceutici S.p.A: lead

Study Sites (12)

Global Research Management

Glendale, California, 91204, United States

Location

Eye Research Foundation

Newport Beach, California, 92663, United States

Location

Martel Eye Medical Group

Rancho Cordova, California, 95670, United States

Location

Sierra Clinical Trials & Research Organization

Santa Ana, California, 92647, United States

Location

Clayton Eye Clinical Research, LLC

Morrow, Georgia, 30260, United States

Location

Kentucky Eye Institute

Lexington, Kentucky, 40517, United States

Location

Tauber Eye Center

Kansas City, Missouri, 64111, United States

Location

Moyes Eye Center

Kansas City, Missouri, 64154, United States

Location

SightMD

Babylon, New York, 11702, United States

Location

Toyos Clinic

Nashville, Tennessee, 37027, United States

Location

Houston Eye Associates HEA - Gramercy Location

Houston, Texas, 77025, United States

Location

Advanced Laser Vision Surgical Institute (Study Site) Intouch Clinical Research Center (SMO)

Houston, Texas, 77034, United States

Location

Related Publications (1)

• Wirta D, Lipsky W, Toyos M, Martel J, Goosey J, Verachtert A, El-Harazi S, Karpecki P, Allegretti M, Goisis G, Pasedis G, Mantelli F. Recombinant human nerve growth factor (cenegermin) for moderate-to-severe dry eye: phase II, randomized, vehicle-controlled, dose-ranging trial. BMC Ophthalmol. 2024 Jul 17;24(1):290. doi: 10.1186/s12886-024-03564-w.

  PMID: 39020305 DERIVED

MeSH Terms

Conditions

Dry Eye Syndromes

Interventions

cenegermin

Condition Hierarchy (Ancestors)

Lacrimal Apparatus Diseases; Eye Diseases

Results Point of Contact

• Title: Clinical Development & Operations
• Organization: Dompé farmaceutici s.p.a.

clinical.trials@dompe.com

+39 02 583831

Study Officials

• Flavio Mantelli, MD, PhD

  Dompé Farmaceutici SpA

  STUDY DIRECTOR

• William Lipsky, MD

  Advanced Laser Vision Surgical Institute (Study Site) Intouch Clinical Research Center (SMO)

  PRINCIPAL INVESTIGATOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double masked
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Parallel groups

Study Record Dates

• First Submitted: May 23, 2019
• First Posted: June 11, 2019
• Study Start: June 10, 2019
• Primary Completion: July 15, 2020
• Study Completion: July 15, 2020
• Last Updated: April 19, 2024
• Results First Posted: February 8, 2022

Record last verified: 2023-12

Locations

US (12)