A Safety and Efficacy Study of 2 Dosing Regimens of Recombinant Human Nerve Growth Factor (rhNGF) Eye Drop Solution Compared With Vehicle in Patients With Dry Eye Disease.

NCT06244316 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: NGF01232023-507561-26-00
• Study Type: interventional
• Target: 317
• Locations: 2 countries
• Sites: 14

Brief Summary

Primary objective

• To evaluate the efficacy of 5 μg/mL and 10 μg/mL concentrations of the new formulation of rhNGF ophthalmic solution versus vehicle, in order to demonstrate superiority of at least 1 of the concentrations over vehicle in the improvement of ocular symptoms of dry eye in participants with dry eye disease (DED) Key secondary objectives
• To evaluate the efficacy of the new formulation of rhNGF ophthalmic solution in increasing the number of participants with improved reflex tear production as compared to vehicle at week 4
• To evaluate the efficacy of the new formulation of rhNGF ophthalmic solution in improving reflex tear production as compared to vehicle at week 4
• To evaluate the efficacy of the new formulation of rhNGF ophthalmic solution in improving ocular surface integrity (corneal epitheliopathy) as compared to vehicle at week 4 Secondary objectives
• To evaluate the efficacy of the new formulation of rhNGF ophthalmic solution in improving reflex tear production as compared to vehicle at week 8
• To evaluate the efficacy of the new formulation of rhNGF ophthalmic solution in improving tear film stability as compared to vehicle at weeks 4 and 8
• To evaluate the efficacy of the new formulation of rhNGF ophthalmic solution in improving ocular surface integrity (corneal and conjunctival epitheliopathy) as compared to vehicle at weeks 4 and 8
• To evaluate the efficacy of the new formulation of rhNGF ophthalmic solution in improving the severity and frequency of dry eye symptoms as compared to vehicle at weeks 4 and 8
• To evaluate the efficacy of the new formulation of rhNGF ophthalmic solution in improving dry eye symptoms as compared to vehicle at week 4
• To evaluate the efficacy of the new formulation of rhNGF ophthalmic solution in improving associated symptoms in DED as compared to vehicle at weeks 4 and 8
• To evaluate the efficacy of the new formulation of rhNGF ophthalmic solution in improving the quality of life in participants with DED as compared to vehicle at weeks 4 and 8
• To evaluate the efficacy of the new formulation of rhNGF ophthalmic solution in improving best corrected visual acuity in DED as compared to vehicle at weeks 4 and 8 Safety objectives
• To evaluate safety/tolerability of the new formulation of rhNGF ophthalmic solution
• To evaluate safety of the new formulation of rhNGF ophthalmic solution
• To evaluate tolerability of the new formulation of rhNGF ophthalmic solution

Conditions

Dry Eye Disease

Keywords

rhNGF; Recombinant Human Nerve Growth Factor; DED; Eye Drop Solution

Outcome Measures

Primary Outcomes (1)

• Mean Change From Baseline to Week 8 in Symptoms of Dry Eye Assessed by Symptom Assessment in Dry Eye (SANDE) Global Score

  SANDE questionnaire included 2 VAS-based questions that assessed: (i) DED symptom frequency (from 0 to 100) (ii) DED symptom severity (from 0 to 100) compiled by the participants. The global SANDE score (from 0 to 100, with 100 representing the most frequent and severe dry eye symptoms) was calculated by multiplying the frequency score by the severity score and obtaining the square root. For SANDE global Score, the lower the score, the better the outcome. Adjusted means are reported. Data here reported are the data after having applied the predefined strategy to handle intercurrent events (i.e. data for SANDE collected in the week after an administration of a prohibited medication are set to missing under the hypothetical strategy while a treatment policy strategy is used for any other intercurrent event). Missing data for SANDE Global Score are imputed employing Multiple Imputation (MI) based on copy-reference approach assuming MNAR.

  Week 8 (V5)

Secondary Outcomes (19)

• Key Secondary Outcome: Percentage of Participants Improving to Schirmer-I Test Without Anesthesia ≥ 10 mm/5 Min in the Study Eye at Week 4

  Week 4 (V4)

• Key Secondary Outcome: Mean Change From Baseline to Week 4 in Schirmer-I Score Without Anesthesia in the Study Eye

  Week 4 (V4)

• Key Secondary Outcome: Mean Change From Baseline to Week 4 in Total Corneal Fluorescein Staining (National Eye Institute NEI Scale) in the Study Eye as Assessed by the Investigator

  Week 4 (V4)

• Mean Change From Baseline to Week 8 in Schirmer-I Score Without Anesthesia in the Study Eye

  Week 8 (V5)

• Percentage of Participants Improving to Schirmer-I Test Without Anesthesia ≥ 10 mm/5min in the Study Eye at Week 8

  Week 8 (V5)

Study Arms (3)

rhNGF 5 μg/mL

EXPERIMENTAL

IMP1

Drug: rhNGF 5 μg/mL

rhNGF 10 μg/mL

EXPERIMENTAL

IMP2

Drug: rhNGF 10 μg/mL

Vehicle IMP

PLACEBO COMPARATOR

Vehicle

Drug: Vehicle (Placebo solution)

Interventions

rhNGF 5 μg/mL DRUG

1 drop of rhNGF ophthalmic solution at 5 μg/mL in each eye TID, at approximately 6-hour intervals, for 4 weeks of treatment.

Also known as: Recombinant Human Nerve Growth Factor

rhNGF 5 μg/mL

rhNGF 10 μg/mL DRUG

1 drop of rhNGF ophthalmic solution at 10 μg/mL in each eye TID, at approximately 6-hour intervals, for 4 weeks of treatment.

Also known as: Recombinant Human Nerve Growth Factor

rhNGF 10 μg/mL

Vehicle (Placebo solution) DRUG

Eye drop solution, containing no rhNGF. 1 drop of vehicle in each eye TID at approximately 6-hour intervals, for 4 weeks of treatment.

Also known as: Placebo

Vehicle IMP

Eligibility Criteria

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

You may qualify if:

• Male or female aged ≥18 years of any race/ethnicity and eye color.
• A diagnosis of dry eye disease at least 6 months before enrollment (current use or recommended use of artificial tears for the treatment of dry eye).
• Moderate-to-severe dry eye characterized by the following clinical features:
• Symptoms Assessment in Dry Eye (SANDE) questionnaire global score ≥50, and
• Schirmer-I test without anesthesia \>2 mm and \<10 mm/5 minutes, and
• Total corneal fluorescein staining grade ≥3 (NEI scale) and/or total conjunctival lissamine green staining score ≥3 assessed by the NEI grading system, and
• Fluorescein tear film break-up time (fTBUT) \< 10 seconds The same eye must have fulfilled all the above criteria.
• Best corrected distance visual acuity (BCDVA) score on ETDRS chart of ≥0.1 decimal units (≤1.0 logarithm of the minimum angle of resolution \[logMAR\]) in each eye at the time of study enrollment
• Negative pregnancy test in females of childbearing potential.
• Only participants who satisfy all informed consent requirements were included in the study; the participant and/or his/her legal representative must have read, signed, and dated the informed consent document before any study-related procedures were performed; the informed consent form signed by participants and/or legal representatives must have been approved by the Institutional Review Board (IRB) for the current study.
• 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 ocular medication during the entire duration of the study.
• History of severe systemic allergy or severe ocular allergy \[including seasonal conjunctivitis, AKC (Atopic KeratoConjunctivitis), VKC (Vernal KeratoConjunctivitis)\] or chronic conjunctivitis and/or keratitis other than dry eye.
• Ocular scarring due to irradiation, alkali burns, Stevens-Johnson syndrome and ocular cicatricial pemphigoid.
• Destruction of conjunctival goblet cells such as in Vitamin A deficiency.
• Severe blepharitis or obvious inflammation of the lid margin.
• Intraocular inflammation defined as Tyndall score \>0.
• Medical history of tumor malignancy in the previous 3 years
• Systemic disease not stabilized within 1 month before the 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.
• History of a serious adverse reaction or significant hypersensitivity to any drug or chemically related compounds or had a clinically significant allergy to drugs, foods, topical anesthetic eye drop or other local anesthetics or other materials, including ocular vital dyes, tropicamide eye drops, commercial artificial tears.
• Known or suspected allergy to sesame and other seeds, tree nuts, and/or peanuts, and/or any other component of the new rhNGF formulation.
• Fertile patients (i.e., not surgically sterilized, or postmenopausal women for at least 1 year) are excluded from participation in the study if they do not practice abstinence from heterosexual intercourse as per usual and customary lifestyle, or are unwilling to use an acceptable form of contraception such as condom with spermicidal cream or jelly for males, or for females if they meet any one of the following conditions:
• Currently pregnant (positive urine pregnancy test at screening or baseline visits) or planning to become pregnant during the duration of the treatment phase of the clinical trial.
• Participant is breastfeeding.

Sponsors & Collaborators

• Dompé Farmaceutici S.p.A: lead

Study Sites (14)

Arizona Eye Center

Chandler, Arizona, 85224, United States

Location

East West Eye Institute

Torrance, California, 90505, United States

Location

Vision Institute - Fontanero St

Colorado Springs, Colorado, 80907, United States

Location

Sibia Eye Institute

Boynton Beach, Florida, 33437, United States

Location

Eye Consultants of Atlanta

Atlanta, Georgia, 30339, United States

Location

New England Eye Center - Boston

Boston, Massachusetts, 02111, United States

Location

Eye Associates of North Jersey

Dover, New Jersey, 07801, United States

Location

The Scheie Eye Institute

Philadelphia, Pennsylvania, 19104, United States

Location

Total Eye Care PA

Memphis, Tennessee, 38119, United States

Location

Toyos Clinic

Nashville, Tennessee, 37215, United States

Location

Azienda Ospedaliera Universitaria Policlinico G Martino

Messina, 98124, Italy

Location

Ospedale S. Giuseppe Multimedica

Milan, 20123, Italy

Location

Fondazione Policlinico Universitario Campus Bio-Medico di Roma

Roma, 00128, Italy

Location

Azienda Ospedaliero Universitario Policlinicol Umberto I

Roma, 00161, Italy

Location

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.

clinicaltrials@dompe.com

+39 02 583831

Study Officials

• Flavio Mantelli, MD, PhD

  Dompé Farmaceutici S.p.A

  STUDY DIRECTOR

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
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 29, 2024
• First Posted: February 6, 2024
• Study Start: January 22, 2024
• Primary Completion: December 4, 2024
• Study Completion: December 4, 2024
• Last Updated: February 13, 2026
• Results First Posted: February 13, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

IT (4); US (10)