NEXAGON for the Treatment of Corneal Persistent Epithelial Defects Following Severe Ocular Injuries

NCT04081103 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: NEX-PED-005
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 1

Brief Summary

This study will enroll participants with a non-infected, corneal persistent epithelial defect (PED) resulting from an ocular chemical and/or thermal ocular injury which is non-responsive or refractory to current standard of care for at least 14 days. It will assess the efficacy and safety of Nexagon® (lufepirsen) plus standard of care versus NEXAGON-vehicle (placebo) plus standard of care. The recovery of the corneal epithelium will be the primary outcome measure, defined as a cornea that re-epithelializes by Day 28 of treatment and remains re-epithelialized for at least a further 28 days.

Conditions

Corneal Persistent Epithelial Defect

Outcome Measures

Primary Outcomes (2)

• The Proportion of Subjects Achieving Corneal Epithelial Recovery, as Assessed by Slit Lamp Examination.

  Corneal epithelial recovery, defined as corneal reepithelialization and maintenance of a durable epithelium for at least 28 days, will be assessed by slit lamp examination.

  Up to 56 days.

• Incidence of Treatment Emergent Adverse Events as Assessed by CTCAE v 5.0

  Incidence of Treatment Emergent Adverse Events as assessed by CTCAE v 5.0.

  Up to 30 days after last application of intervention

Study Arms (3)

Nexagon® (lufepirsen) High Dose Concentration

EXPERIMENTAL

Drug: Nexagon® (lufepirsen) High Dose Concentration; Drug: Open-label Nexagon® (lufepirsen)

Nexagon® (lufepirsen) Low Dose Concentration

EXPERIMENTAL

Drug: Nexagon® (lufepirsen) Low Dose Concentration; Drug: Open-label Nexagon® (lufepirsen)

Vehicle

PLACEBO COMPARATOR

Drug: Vehicle; Drug: Open-label Nexagon® (lufepirsen)

Interventions

Nexagon® (lufepirsen) High Dose Concentration DRUG

Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.

Nexagon® (lufepirsen) High Dose Concentration

Nexagon® (lufepirsen) Low Dose Concentration DRUG

Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.

Nexagon® (lufepirsen) Low Dose Concentration

Vehicle DRUG

Vehicle is administered topically in the affected eye three (3) times over 28 days.

Also known as: Placebo

Vehicle

Open-label Nexagon® (lufepirsen) DRUG

Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.

Nexagon® (lufepirsen) High Dose Concentration; Nexagon® (lufepirsen) Low Dose Concentration; Vehicle

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female of any age.
• The presence of a non-infected, corneal persistent epithelial defect (PED) which has resulted from a severe chemical and/or thermal (burn) injury to one or both eyes.
• The PED is non-responsive to current standard of care for at least 14 days from injury.
• The PED measures at least 2 mm along the largest diameter at Day 1 of the Treatment Period.
• Providing written informed consent and ability to comply with the visit and dosing schedule.

You may not qualify if:

• Have active ocular infection.
• Subjects with corneal perforation or impending corneal perforation.
• Subjects with any other past or present ophthalmic disease or medical condition that, in the Investigator's opinion, may affect the safety of the subject or the outcome of the study.
• Subjects with severe lid abnormalities or ocular conditions that contribute to the persistence of the epithelial defect.
• Female subjects of childbearing potential who are pregnant, nursing, planning a pregnancy or not using an adequate and medically acceptable form of birth control.
• Subjects who have participated in an interventional clinical trial within 30 days prior to Day 1.

Sponsors & Collaborators

• Glaukos Corporation: lead
• Amber Ophthalmics, Inc.: collaborator

Study Sites (1)

Jules Stein Eye Institute

Los Angeles, California, 90095, United States

Location

Limitations and Caveats

Early termination leading to small numbers of subjects analyzed

Results Point of Contact

• Title: Director of Clinical Trials
• Organization: Amber Ophthalmics

clinical@Amberophthalmics.com

858-539-3418

Study Officials

• Maria Feldman

  Amber Ophthalmics

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 29, 2019
• First Posted: September 9, 2019
• Study Start: June 30, 2020
• Primary Completion: February 7, 2022
• Study Completion: February 7, 2022
• Last Updated: July 14, 2025
• Results First Posted: October 23, 2024

Record last verified: 2025-07

Locations

US (1)