A Study to Evaluate the Safety and Efficacy of NEXAGON® (Lufepirsen Ophthalmic Gel) in Subjects With PCED

Phase 2

Recruiting

• Conditions: Persistent Corneal Epithelial Defect
• Interventions: Drug: Vehicle; Drug: lufepirsen

• Registration Number: NCT05966493
• Lead Sponsor: Amber Ophthalmics, Inc.

Brief Summary

This study is to evaluate the safety and efficacy of NEXAGON® (lufepirsen ophthalmic gel) (NEXAGON) in subjects with persistent corneal epithelial defects (PCED). The objectives of the study are to evaluate the safety and efficacy of NEXAGON in this population.

Detailed Description

This study is a randomized, multicenter, double-masked, vehicle-controlled study to evaluate the safety and efficacy of NEXAGON (lufepirsen ophthalmic gel) in subjects with persistent corneal epithelial defects (PCED). The study will enroll subjects who will complete a Screening Period, Treatment Period (up to 8 weeks) and Follow-up Period (4 weeks). Those subjects whose defect has not re-epithelialized by the completion of the Treatment Period or who achieved re-epithelialization but failed to maintain re-epithelialization for 28 days after treatment completion (durability) are eligible to enter the NEXAGON Open-label Treatment Period for an additional 8 weeks.

Study Timeline

• Study First Submitted: 2023-06-20
• Study First Submitted QC: 2023-07-21
• Study First Posted: 2023-07-28
• Study Start: 2023-08-17
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-30
• Last Update Posted: 2024-08-01
• Primary Completion: 2025-12
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 84

Inclusion Criteria

1. Have a PCED that is at least 2 weeks in duration and refractory to one or more conventional non-surgical standard of care (SOC) treatments
2. Have no clinical evidence of improvement in the PCED within 2 weeks prior to randomization despite the use of non-surgical SOC treatment
3. Subject must provide written informed consent (or assent)
4. Subjects with childbearing potential must be 1-year postmenopausal, surgically sterilized, or have a negative urine pregnancy test

Exclusion Criteria

1. Have a known ocular infection that is deemed to be active requiring therapeutic intervention
2. Present with a corneal surface defect in either eye that is directly attributed to an infectious etiology (bacterial, viral, fungal and/or protozoal) that has not fully resolved and/or treatment has not been completed
3. Present with evidence of corneal ulceration/melting involving the posterior third of the stroma and/or perforation in either eye
4. Have a blepharitis or meibomian gland disease in the study eye that is deemed to be clinically relevant and/or active
5. Have a history of ocular surgery or any ocular procedure(s) not meeting the designated washout time
6. Have any other ocular disease requiring topical ocular medication in the affected eye
7. Have a presence or history of any ocular or systemic disorder or condition that could interfere the safety or efficacy of the study treatment, or the interpretation of the study results
8. Have a known hypersensitivity to one of the components of the study or procedural medications (e.g., NEXAGON, fluorescein)
9. Participated in an interventional clinical drug or device trial within 28 days prior to Day 1
10. Use of the medications presented in the protocol that are prohibited in the study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
NEXAGON Vehicle (ophthalmic gel)	Vehicle	Vehicle applied topically weekly for 4 to 8 weeks.
NEXAGON® (lufepirsen ophthalmic gel) High Dose Concentration	lufepirsen	Lufepirsen (High dose concentration) applied topically weekly for 4 to 8 weeks.

Primary Outcome Measures

Name	Time	Method
Achieve Corneal Re-epithelialization Including Durability (CRC)	End of Study: 28 Days after achieving re-epithelialization	The proportion of subjects achieving corneal re-epithelialization that is maintained for a minimum of 28 days, based on assessment of corneal fluorescein staining images of the PCED by a Central Reading Center (CRC).

Trial Locations

Locations (1)

Principal Investigator; 🇺🇸 Newport Beach, California, United States