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

Persistent Corneal Epithelial Defect Clinical Trial

— NEXPEDE-1  

Official title:

A Randomized, Multicenter, Double-Masked, Vehicle-Controlled Phase 2/3 Study to Evaluate the Safety and Efficacy of NEXAGON® (Lufepirsen Ophthalmic Gel) in Subjects With Persistent Corneal Epithelial Defects (NEXPEDE-1)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05966493
• Other study ID #: AMB-01-006
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: July 2023
• Est. completion date: October 2024

Study information

• Verified date: July 2023
• Source: Amber Ophthalmics, Inc.
• Contact: President and CSO, PharmD
• Phone: 858-663-1500
• Email: clinical[@]amberophthalmics.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 120
• Est. completion date: October 2024
• Est. primary completion date: October 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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. PCED measures at least 2 mm along the largest diameter

4. Subject must provide written informed consent (or assent)

5. 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 in the opinion of the Investigator is deemed to be clinically relevant and/or active

5. Have a history of a full thickness keratoplasty, > 1 Descemet membrane endothelial keratoplasty (DMEK) or Descemet's stripping endothelial keratoplasty (DSEK) procedure

6. Have a history of ocular surgery or any ocular procedure(s) not meeting the designated washout time

7. Have any other ocular disease requiring topical ocular medication in the affected eye

8. A Schirmer I test result (without anesthesia) of = 3 mm/5 minutes in the study eye

9. Have a presence or history of any ocular or systemic disorder or condition that, in the judgement of the Investigator, might hinder the efficacy of the study treatment or its evaluation, could possibly interfere with the interpretation of study results, or could be incompatible with the study visit schedule or conduct

10. Have a known hypersensitivity to one of the components of the study or procedural medications (e.g., NEXAGON, fluorescein)

11. Participated in an interventional clinical drug or device trial within 28 days prior to Day 1

12. Use of the medications presented in the protocol that are prohibited in the study.

13. Use of Oxervate within 30 days of study enrollment.

Related Conditions & MeSH terms

• Persistent Corneal Epithelial Defect

Intervention

Drug:
• lufepirsen
Lufepirsen is an unmodified connexin43 antisense oligonucleotide.
• Vehicle
Matching vehicle without lufepirsen.

Locations

Country	Name	City	State
United States	Principal Investigator	Atlanta	Georgia
United States	Principal Investigator	Grand Junction	Colorado
United States	Principal Investigator	Nashville	Tennessee
United States	Principal Investigator	Pasadena	California
United States	Principal Investigator	San Antonio	Texas
United States	Principal Investigator	Torrance	California

Sponsors (1)

Lead Sponsor	Collaborator
Amber Ophthalmics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Achieve Corneal Re-epithelialization Including Durability (CRC)	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).	End of Study: 28 Days after achieving re-epithelialization
Secondary	Achieve Corneal Re-epithelialization Including Durability (Investigator)	The proportion of subjects achieving corneal re-epithelialization that is maintained for a minimum of 28 days, based on assessment of corneal fluorescein staining of the PCED by the Investigator	End of Study: 28 Days after achieving re-epithelialization
Secondary	Achieve Corneal Re-epithelialization (CRC)	The proportion of subjects achieving corneal re-epithelialization, based on assessment of corneal fluorescein staining images of the PCED by a CRC.	Weeks 1, 2, 3, 4, 5, 6, 7, 8
Secondary	Achieve Corneal Re-epithelialization (Investigator)	The proportion of subjects achieving corneal re-epithelialization based on assessment of corneal fluorescein staining images of the PCED by Investigator.	Weeks 1, 2, 3, 4, 5, 6, 7, 8
Secondary	Number of Dose Administrations Required to Achieve Corneal Re-epithelialization (CRC)	The number of dose administrations subjects required to achieve corneal re-epithelialization that is maintained for a minimum of 28 days after completing treatment, based on assessment of corneal fluorescein staining images of the PCED by a CRC.	Weeks 1, 2, 3, 4, 5, 6, 7, 8
Secondary	Number of Dose Administrations Required to Achieve Corneal Re-epithelialization (Investigator)	The number of dose administrations subjects required to achieve corneal re-epithelialization that is maintained for a minimum of 28 days after completing treatment, based on assessment of corneal fluorescein staining images of the PCED by Investigator.	Weeks 4, 5, 6, 7, 8
Secondary	Time to Corneal Re-epithelialization (CRC)	The time (in days) to corneal re-epithelialization, defined as the time from randomization to the time of corneal re-epithelialization based on assessment of corneal fluorescein staining images of the PCED by a CRC.	Weeks 1, 2, 3, 4, 5, 6, 7, 8
Secondary	Time to Corneal Re-epithelialization (Investigator)	The time (in days) to corneal re-epithelialization, defined as the time from randomization to the time of corneal re-epithelialization based on assessment of corneal fluorescein staining images of the PCED by Investigator.	Weeks 1, 2, 3, 4, 5, 6, 7, 8
Secondary	Ocular Pain Assessment Survey	The mean change from baseline (CFB) in ocular pain based on Ocular Pain Assessment Survey (OPAS).	End of Study: Week 9, 10, 11, or 12
Secondary	Assessment of visual health status indices by the administration of the NEI Visual Function Questionnaire (NEI-VFQ-25).	Assess the change from baseline in the subject's visual health status using the NEI-VFQ-25 (0-100, higher scores representing a better status).	End of Study: Week 9, 10, 11, or 12
Secondary	Visual Acuity Assessment	The mean CFB in Best Corrected Distance Visual Acuity (BCDVA).	End of Study: Week 9, 10, 11, or 12
Secondary	Visual Acuity Improvement	The proportion of subjects who achieve a 15 letter (ETDRS) gain in BCDVA.	End of Study: Week 9, 10, 11, or 12
Secondary	Open-label Treatment Period	The proportion of subjects requiring open-label treatment during the Treatment Period.	Open-label Day 1
Secondary	Achieve Corneal Re-epithelialization in Open-label Treatment Period	The proportion of subjects in the Open-Label Treatment Period that achieve re-epithelialization of the corneal epithelial defect that remains durable for a minimum of 28 days based on the CRC assessment on images.	End of Study: 28 Days after achieving re-epithelialization