Evaluation of Efficacy of 20 µg/ml rhNGF New Formulation (With Anti-oxidant) in Patients With Stage 2 and 3 NK

NCT02227147 | Completed Phase 2 Results

• 1 other identifier: NGF0214
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 12

Brief Summary

The primary objective of this study is to evaluate the efficacy of 20 µg/ml 6 times a day of rhNGF eye drops solution (formulation containing anti-oxidant) compared to vehicle (formulation containing anti-oxidant) given 6 times a day. The evaluation of efficacy is intended as:

• complete healing of stage 2 (persistent epithelial defect) and 3 (corneal ulcer) neurotrophic keratitis (NK) as measured by the central reading center using corneal fluorescein staining,
• assessing the duration of complete healing,
• improvement in visual acuity and improvement in corneal sensitivity.

Conditions

Neurotrophic Keratitis

Keywords

Neurotrophic Keratitis

Outcome Measures

Primary Outcomes (1)

• Complete Healing of the Persistent Epithelial Defect (PED) or Corneal Ulcer Defined by Central Reviewer

  Percentage of patients achieving complete healing of the PED or corneal ulcer determined by corneal fluorescein staining at 8 weeks as defined by the central reading center on clinical pictures.

  Week 8

Secondary Outcomes (7)

• Complete Healing of the Persistent Epithelial Defect (PED) or Corneal Ulcer Defined by the Investigator

  Week 8

• Complete Healing of the Persistent Epithelial Defect (PED) or Corneal Ulcer Defined by Central Reading Center and Investigator.

  At weeks 4 and 6

• Percentage of Patients With Complete Corneal Clearing

  at weeks 4, 6, and 8

• Mean Change From Baseline in Best Corrected Distance Visual Acuity (BCDVA)

  Baseline, Week 8

• Percentage of Patients That Achieve a 15 Letter Gain in BCDVA

  Weeks 4, week 6 and week 8

Study Arms (2)

rhNGF 20 µg/ml

EXPERIMENTAL

rhNGF 20 µg/ml eye drops solution, formulation containing anti-oxidant

Drug: rhNGF 20µg/ml

Placebo

PLACEBO COMPARATOR

Vehicle: formulation containing anti-oxidant

Other: Placebo

Interventions

rhNGF 20µg/ml DRUG

Also known as: cenegermin

rhNGF 20 µg/ml

Placebo OTHER

Formulation containing antioxidant

Also known as: Vehicle

Placebo

Eligibility Criteria

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

You may qualify if:

• Patients 18 years of age or older.
• Patients with stage 2 (persistent epithelial defect, PED) or stage 3 (corneal ulcer) neurotrophic keratitis (NK).
• PED or corneal ulceration of at least 2 weeks duration refractory to one or more conventional non-surgical treatments for neurotrophic keratitis.
• Evidence of decreased corneal sensitivity (≤ 4 cm using the Cochet-Bonnet aesthesiometer) within the area of the PED or corneal ulcer and outside of the area of the defect in at least one corneal quadrant.
• Best corrected distance visual acuity (BCDVA) score ≤ 75 ETDRS (Early Treatment Diabetic Retinopathy Study)letters, (≥ 0.2 LogMAR, ≤ 20/32 Snellen or ≤ 0.625 decimal fraction) in the affected eye(s).
• No objective clinical evidence of improvement in the PED or corneal ulceration within the 2 weeks prior to study enrolment.
• 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 IRB (Institutional Review Board) for the current study.
• Patients must have the ability and willingness to comply with study procedures.

You may not qualify if:

• Any active ocular infection or active ocular inflammation not related to NK in the affected eye(s).
• Any other ocular disease requiring topical ocular treatment during the course of the study treatment period. No topical treatments other than the study medications provided by the study sponsor and allowed by the study protocol can be administered in the affected eye(s) during the course of the study treatment periods.
• Patients with severe vision loss with no potential for visual improvement in the opinion of the investigator as a result of the study treatment.
• Schirmer test without anesthesia ≤3 mm/5 minutes.
• Patients with severe blepharitis and/or severe meibomian gland disease.
• History of any ocular surgery (including laser or refractive surgical procedures) within the three months before study enrolment. (An exception to the preceding statement will be allowed if the ocular surgery is considered to be the cause of the stage 2 or 3 NK). Ocular surgery will not be allowed during the study treatment period and elective ocular surgery procedures should not be planned during the duration of the follow-up period.
• Prior surgical procedure(s) for the treatment of NK with the exception of amniotic membrane transplantation. Patients previously treated with amniotic membrane transplantation may only be enrolled two weeks after the membrane has disappeared within the area of the PED or corneal ulcer or at least six weeks after the date of the amniotic membrane transplantation procedure.
• Patients previously treated with Botox injections used to induce pharmacologic blepharoptosis are eligible for enrolment only if the last injection was given at least 90 days prior to enrolment in the study.
• Anticipated need to use therapeutic contact lenses or contact lens wear for refractive correction during the study treatment period in the eye(s) with NK.
• Anticipated need for punctual occlusion during the study treatment period. Patients with punctual occlusion or punctual plugs inserted prior to the study are eligible for enrolment provided that the punctual occlusion is maintained during the study.
• Evidence of corneal ulceration involving the posterior third of the corneal stroma, corneal melting or perforation.
• Presence or history of any ocular or systemic disorder or condition that might hinder the efficacy of the study treatment or its evaluation, could possibly interfere with the interpretation of study results, or could be judged by the investigator to be incompatible with the study visit schedule or conduct.
• Any need for or anticipated change in the dose of systemic medications known to impair the function of the trigeminal nerve. These treatments are allowed during the study if initiated prior study enrolment provided they remain stable throughout the course of the study treatment periods.
• Known hypersensitivity to one of the components of the study or procedural medications (e.g. fluorescein).
• History of drug, medication or alcohol abuse or addiction.

Sponsors & Collaborators

• Dompé Farmaceutici S.p.A: lead

Study Sites (12)

Loma Linda University Ophthalmology

Loma Linda, California, 92354, United States

Location

Jules Stein Eye Institute

Los Angeles, California, 90095, United States

Location

Shiley Eye Center University of California

San Diego, California, 92093, United States

Location

Bascom Palmer Eye Institute University of Miami

Miami, Florida, 33324, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

Massachusetts Eye & Ear Infirmary

Boston, Massachusetts, 02114, United States

Location

Massachusetts Eye Research and Surgery Institution

Cambridge, Massachusetts, 02451, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

New York Eye and Ear Infirmary

New York, New York, 10003, United States

Location

Penn Eye Care Scheie Eye Institute

Philadelphia, Pennsylvania, 19104, United States

Location

UPMC eye center, department of ophthalmology, University of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

MeSH Terms

Interventions

cenegermin

Limitations and Caveats

LImitations and caveats non specified

Results Point of Contact

• Title: Flavio Mantelli, MD, PhD
• Organization: Dompé

info@dompe.com

02 583831

Study Officials

• Flavio Mantelli, MD, PhD

  Dompé farmaceutici S.p.A., Milan

  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: August 26, 2014
• First Posted: August 27, 2014
• Study Start: February 1, 2015
• Primary Completion: June 1, 2016
• Study Completion: September 1, 2016
• Last Updated: December 8, 2022
• Results First Posted: June 14, 2019

Record last verified: 2022-12

Locations

US (12)