Trial Outcomes & Findings for NEXAGON for the Treatment of Corneal Persistent Epithelial Defects Following Severe Ocular Injuries (NCT NCT04081103)
NCT ID: NCT04081103
Last Updated: 2025-07-14
Results Overview
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.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
35 participants
Primary outcome timeframe
Up to 56 days.
Results posted on
2025-07-14
Participant Flow
Unit of analysis: Eyes
Participant milestones
| Measure |
Nexagon® (Lufepirsen) High Dose Concentration
Nexagon® (lufepirsen) High Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Nexagon® (Lufepirsen) Low Dose Concentration
Nexagon® (lufepirsen) Low Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Vehicle
Vehicle: Vehicle is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
|---|---|---|---|
|
Overall Study
STARTED
|
12 12
|
12 12
|
11 11
|
|
Overall Study
COMPLETED
|
11 11
|
11 11
|
9 9
|
|
Overall Study
NOT COMPLETED
|
1 1
|
1 1
|
2 2
|
Reasons for withdrawal
| Measure |
Nexagon® (Lufepirsen) High Dose Concentration
Nexagon® (lufepirsen) High Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Nexagon® (Lufepirsen) Low Dose Concentration
Nexagon® (lufepirsen) Low Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Vehicle
Vehicle: Vehicle is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
1
|
1
|
1
|
|
Overall Study
Physician Decision
|
0
|
0
|
1
|
Baseline Characteristics
NEXAGON for the Treatment of Corneal Persistent Epithelial Defects Following Severe Ocular Injuries
Baseline characteristics by cohort
| Measure |
Nexagon® (Lufepirsen) High Dose Concentration
n=12 Participants
Nexagon® (lufepirsen) High Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Nexagon® (Lufepirsen) Low Dose Concentration
n=12 Participants
Nexagon® (lufepirsen) Low Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Vehicle
n=11 Participants
Vehicle: Vehicle is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Total
n=35 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
25.9 years
STANDARD_DEVIATION 20.42 • n=99 Participants
|
30.3 years
STANDARD_DEVIATION 16.38 • n=107 Participants
|
30.9 years
STANDARD_DEVIATION 19.03 • n=206 Participants
|
29.0 years
STANDARD_DEVIATION 18.26 • n=7 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
7 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
28 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
11 Participants
n=206 Participants
|
34 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Asian
|
11 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
11 Participants
n=206 Participants
|
33 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=99 Participants
|
1 participants
n=107 Participants
|
0 participants
n=206 Participants
|
2 participants
n=7 Participants
|
|
Region of Enrollment
India
|
11 participants
n=99 Participants
|
11 participants
n=107 Participants
|
11 participants
n=206 Participants
|
33 participants
n=7 Participants
|
|
Subjects with of Persistent Epithelial Defect
|
12 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
11 Participants
n=206 Participants
|
35 Participants
n=7 Participants
|
PRIMARY outcome
Timeframe: Up to 56 days.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.
Outcome measures
| Measure |
Nexagon® (Lufepirsen) High Dose Concentration
n=12 Participants
Nexagon® (lufepirsen) High Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Nexagon® (Lufepirsen) Low Dose Concentration
n=12 Participants
Nexagon® (lufepirsen) Low Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Vehicle
n=11 Participants
Vehicle: Vehicle is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
|---|---|---|---|
|
The Proportion of Subjects Achieving Corneal Epithelial Recovery, as Assessed by Slit Lamp Examination.
|
8 Participants
|
8 Participants
|
3 Participants
|
PRIMARY outcome
Timeframe: Up to 30 days after last application of interventionIncidence of Treatment Emergent Adverse Events as assessed by CTCAE v 5.0.
Outcome measures
| Measure |
Nexagon® (Lufepirsen) High Dose Concentration
n=12 Participants
Nexagon® (lufepirsen) High Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Nexagon® (Lufepirsen) Low Dose Concentration
n=12 Participants
Nexagon® (lufepirsen) Low Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Vehicle
n=11 Participants
Vehicle: Vehicle is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
|---|---|---|---|
|
Incidence of Treatment Emergent Adverse Events as Assessed by CTCAE v 5.0
|
7 Participants
|
8 Participants
|
7 Participants
|
Adverse Events
Nexagon® (Lufepirsen) High Dose Concentration
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Nexagon® (Lufepirsen) Low Dose Concentration
Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths
Vehicle
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Nexagon® (Lufepirsen) High Dose Concentration
n=12 participants at risk
Nexagon® (lufepirsen) High Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Nexagon® (Lufepirsen) Low Dose Concentration
n=12 participants at risk
Nexagon® (lufepirsen) Low Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Vehicle
n=11 participants at risk
Vehicle: Vehicle is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
|---|---|---|---|
|
Eye disorders
Cicatricial ectropion with lateral lagophthalmos and exposed lacrimal gland
|
0.00%
0/12 • 2 months
|
8.3%
1/12 • Number of events 1 • 2 months
|
0.00%
0/11 • 2 months
|
Other adverse events
| Measure |
Nexagon® (Lufepirsen) High Dose Concentration
n=12 participants at risk
Nexagon® (lufepirsen) High Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Nexagon® (Lufepirsen) Low Dose Concentration
n=12 participants at risk
Nexagon® (lufepirsen) Low Dose Concentration: Nexagon® (lufepirsen) is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
Vehicle
n=11 participants at risk
Vehicle: Vehicle is administered topically in the affected eye three (3) times over 28 days.
Open-label Nexagon® (lufepirsen): Open-label Nexagon® (lufepirsen) for participants who do not heal (re-epithelialize) at the end of the 28-day treatment phase.
|
|---|---|---|---|
|
Eye disorders
Symblepharon
|
8.3%
1/12 • 2 months
|
16.7%
2/12 • 2 months
|
27.3%
3/11 • 2 months
|
|
Eye disorders
Corneal epithelium defect
|
8.3%
1/12 • 2 months
|
0.00%
0/12 • 2 months
|
27.3%
3/11 • 2 months
|
|
Eye disorders
Eye pain
|
8.3%
1/12 • 2 months
|
8.3%
1/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Eye disorders
Blepharitis
|
8.3%
1/12 • 2 months
|
0.00%
0/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Eye disorders
Cataract
|
8.3%
1/12 • 2 months
|
0.00%
0/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Eye disorders
Conjunctival hyperaemia
|
0.00%
0/12 • 2 months
|
0.00%
0/12 • 2 months
|
9.1%
1/11 • 2 months
|
|
Eye disorders
Corneal neovascularisation
|
0.00%
0/12 • 2 months
|
8.3%
1/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Eye disorders
Corneal thinning
|
8.3%
1/12 • 2 months
|
0.00%
0/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Eye disorders
Dry eye
|
0.00%
0/12 • 2 months
|
8.3%
1/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Eye disorders
Ectropion
|
0.00%
0/12 • 2 months
|
8.3%
1/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Eye disorders
Eye irritation
|
0.00%
0/12 • 2 months
|
8.3%
1/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Eye disorders
Eye pruritus
|
0.00%
0/12 • 2 months
|
8.3%
1/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Eye disorders
Lacrimation increased
|
0.00%
0/12 • 2 months
|
8.3%
1/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Eye disorders
Ocular hyperaemia
|
8.3%
1/12 • 2 months
|
0.00%
0/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Eye disorders
Visual impairment
|
0.00%
0/12 • 2 months
|
8.3%
1/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
General disorders
Hyperplasia
|
0.00%
0/12 • 2 months
|
0.00%
0/12 • 2 months
|
9.1%
1/11 • 2 months
|
|
Immune system disorders
Seasonal allergy
|
8.3%
1/12 • 2 months
|
0.00%
0/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Infections and infestations
Hypopyon
|
8.3%
1/12 • 2 months
|
8.3%
1/12 • 2 months
|
9.1%
1/11 • 2 months
|
|
Infections and infestations
Periorbital cellulitis
|
0.00%
0/12 • 2 months
|
8.3%
1/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Injury, poisoning and procedural complications
Eyelid scar
|
8.3%
1/12 • 2 months
|
0.00%
0/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Infections and infestations
COVID-19
|
0.00%
0/12 • 2 months
|
8.3%
1/12 • 2 months
|
0.00%
0/11 • 2 months
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/12 • 2 months
|
8.3%
1/12 • 2 months
|
0.00%
0/11 • 2 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place