Trial Outcomes & Findings for Assessment of the Safety and Efficacy of RGN-259 Ophthalmic Solutions for Dry Eye Syndrome: ARISE-3 (NCT NCT03937882)

NCT ID: NCT03937882

Last Updated: 2026-08-28

Results Overview

Inferior corneal staining was assessed using the Ora Calibra® Corneal and Conjunctival Staining Scale, which is a 0 to 4 scale and where higher numbers indicating more severe staining.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

700 participants

Primary outcome timeframe

Day 15

Results posted on

2026-08-28

Participant Flow

Subjects were screened during a 14-day study run-in period prior to randomization. And after run-in period and confirmation of inclusion and exclusion criteria, all eligible subjects were randomized in a 1:1 ratio to receive 0.1% RGN-259 or placebo ophthalmic solution bilaterally, four times per day (QID) for 14 days. The study comprised of 4 visits over the course of approximately 4 weeks

One participant was enrolled and completed the study twice and received placebo in both periods. This subject was counted once in the ITT population but twice in the safety population. Another participant randomized to the 0.1% RGN-259 arm did not receive treatment and was included only in the ITT population. Consequently, the ITT population included 351 participants in the 0.1% RGN-259 arm and 348 in the placebo arm; the safety population included 350 and 349 participants, respectively.

Participant milestones

Participant milestones
Measure
Experimental: RGN-259
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Overall Study
STARTED
351
348
Overall Study
COMPLETED
346
343
Overall Study
NOT COMPLETED
5
5

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Assessment of the Safety and Efficacy of RGN-259 Ophthalmic Solutions for Dry Eye Syndrome: ARISE-3

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Experimental: RGN-259
n=351 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=348 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Total
n=699 Participants
Total of all reporting groups
Age, Continuous
63.2 Years
STANDARD_DEVIATION 12.31 • n=31 Participants
62.5 Years
STANDARD_DEVIATION 12.36 • n=49 Participants
62.8 Years
STANDARD_DEVIATION 12.33 • n=80 Participants
Sex: Female, Male
Female
267 Participants
n=31 Participants
254 Participants
n=49 Participants
521 Participants
n=80 Participants
Sex: Female, Male
Male
84 Participants
n=31 Participants
94 Participants
n=49 Participants
178 Participants
n=80 Participants
Race (NIH/OMB)
American Indian or Alaska Native
2 Participants
n=31 Participants
2 Participants
n=49 Participants
4 Participants
n=80 Participants
Race (NIH/OMB)
Asian
8 Participants
n=31 Participants
11 Participants
n=49 Participants
19 Participants
n=80 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
1 Participants
n=31 Participants
0 Participants
n=49 Participants
1 Participants
n=80 Participants
Race (NIH/OMB)
Black or African American
44 Participants
n=31 Participants
54 Participants
n=49 Participants
98 Participants
n=80 Participants
Race (NIH/OMB)
White
295 Participants
n=31 Participants
275 Participants
n=49 Participants
570 Participants
n=80 Participants
Race (NIH/OMB)
More than one race
1 Participants
n=31 Participants
6 Participants
n=49 Participants
7 Participants
n=80 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=31 Participants
0 Participants
n=49 Participants
0 Participants
n=80 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
26 Participants
n=31 Participants
22 Participants
n=49 Participants
48 Participants
n=80 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
323 Participants
n=31 Participants
325 Participants
n=49 Participants
648 Participants
n=80 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
2 Participants
n=31 Participants
1 Participants
n=49 Participants
3 Participants
n=80 Participants

PRIMARY outcome

Timeframe: Day 15

Inferior corneal staining was assessed using the Ora Calibra® Corneal and Conjunctival Staining Scale, which is a 0 to 4 scale and where higher numbers indicating more severe staining.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=351 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=348 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Change From Baseline in Inferior Corneal Staining Change From Pre-CAE®(Controlled Adverse Environment) to Post-CAE® at Day 15 (Visit 4) Using the Ora Calibra® Scale
-0.41 Score on a scale
Standard Deviation 0.841
-0.46 Score on a scale
Standard Deviation 0.804

PRIMARY outcome

Timeframe: Day 15

The severity of ocular discomfort was measured using the Ora Calibra® Ocular Discomfort \& 4-Symptom Questionnaire which is a 0 to 5 scale and where higher numbers indicating more severe discomfort.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=351 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=348 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Change From Baseline in Ocular Discomfort at Day 15 (Visit 4) Pre-CAE® Using the Ora Calibra® Ocular Discomfort & 4-Symptom Questionnaire
-0.4 Score on a scale
Standard Deviation 1.12
-0.4 Score on a scale
Standard Deviation 1.14

SECONDARY outcome

Timeframe: Day 8, Day 15

Population: Intent-to-treat population; number of participants analyzed varies by outcome due to missing assessments.

Corneal and conjunctival staining were assessed using the Ora Calibra® Corneal and Conjunctival Staining Scale, which is a 0 to 20 scale and where higher numbers indicating more severe staining.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=351 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=348 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Fluorescein Staining (Ora Calibra® Scale) at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre and Post-CAE®)
Visit 4 (Day 15), Post-CAE
10.11 Score on a scale
Standard Deviation 3.052
9.95 Score on a scale
Standard Deviation 3.018
Fluorescein Staining (Ora Calibra® Scale) at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre and Post-CAE®)
Visit 4 (Day 15), Change from Pre-CAE to Post-CAE
1.91 Score on a scale
Standard Deviation 2.319
1.92 Score on a scale
Standard Deviation 2.262
Fluorescein Staining (Ora Calibra® Scale) at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre and Post-CAE®)
Visit 4 (Day 15), Pre-CAE
8.20 Score on a scale
Standard Deviation 2.996
8.06 Score on a scale
Standard Deviation 2.909
Fluorescein Staining (Ora Calibra® Scale) at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre and Post-CAE®)
Visit 3 (Day 8), Pre-CAE
8.46 Score on a scale
Standard Deviation 2.960
8.36 Score on a scale
Standard Deviation 2.885

SECONDARY outcome

Timeframe: Day 8, Day 15

Population: Intent-to-treat population; number of participants analyzed varies by outcome due to missing assessments.

Lissamine green staining were assessed using the Ora Calibra® Corneal and Conjunctival Staining Scale, which is a 0 to 20 scale and where higher numbers indicating more severe staining.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=351 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=348 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Lissamine Green Staining (Ora Calibra® Scale) at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®)
Visit 3 (Day 8), Pre-CAE
5.71 Score on a scale
Standard Deviation 3.192
5.71 Score on a scale
Standard Deviation 3.106
Lissamine Green Staining (Ora Calibra® Scale) at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®)
Visit 4 (Day 15), Post-CAE
7.03 Score on a scale
Standard Deviation 3.406
6.70 Score on a scale
Standard Deviation 3.246
Lissamine Green Staining (Ora Calibra® Scale) at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®)
Visit 4 (Day 15), Change from Pre-CAE to Post-CAE
1.69 Score on a scale
Standard Deviation 2.281
1.54 Score on a scale
Standard Deviation 2.289
Lissamine Green Staining (Ora Calibra® Scale) at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®)
Visit 4 (Day 15), Pre-CAE
5.34 Score on a scale
Standard Deviation 3.149
5.18 Score on a scale
Standard Deviation 2.961

SECONDARY outcome

Timeframe: Day 8, Day 15

Population: Intent-to-treat population; number of participants analyzed varies by outcome due to missing assessments.

TFBUT© measures tear film stability and was assessed by measuring, in seconds, the time from eye opening to the formation of micelles in the tear film. Measurements were obtained using a stopwatch. Longer TFBUT values indicate greater tear film stability and less severe dry eye.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=351 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=348 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Tear Film Break-Up Time (TFBUT©) at Visits 3 and 4 (Pre- and Post-CAE®)
Visit 4 (Day 15), Post-CAE
3.1072 Seconds
Standard Deviation 1.36872
3.1739 Seconds
Standard Deviation 1.43102
Tear Film Break-Up Time (TFBUT©) at Visits 3 and 4 (Pre- and Post-CAE®)
Visit 3 (Day 8), Pre-CAE
3.1782 Seconds
Standard Deviation 1.34370
3.2186 Seconds
Standard Deviation 1.20830
Tear Film Break-Up Time (TFBUT©) at Visits 3 and 4 (Pre- and Post-CAE®)
Visit 4 (Day 15), Pre-CAE
3.1210 Seconds
Standard Deviation 1.48605
3.1237 Seconds
Standard Deviation 1.34341

SECONDARY outcome

Timeframe: Day 8, Day 15

Population: Intent-to-treat population; number of participants analyzed varies by outcome due to missing assessments.

The Unanesthetized Schirmer's Test measures tear production. A sterile Schirmer test strip was placed in the lower temporal lid margin of each eye, and after 5 minutes, the length of the moistened area on the strip was recorded in millimeters (mm). Higher values indicate greater tear production and a better outcome.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=351 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=348 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Unanesthetized Schirmer's Test at Visit 3, Visit 4 (Pre-CAE®)
Visit 3 (Day 8), Pre-CAE
5.3 mm
Standard Deviation 4.36
5.9 mm
Standard Deviation 5.05
Unanesthetized Schirmer's Test at Visit 3, Visit 4 (Pre-CAE®)
Visit 4 (Day 15), Pre-CAE
5.6 mm
Standard Deviation 4.75
6.0 mm
Standard Deviation 6.13

SECONDARY outcome

Timeframe: Day 1

Population: Intent-to-treat population; number of participants analyzed varies by outcome due to missing assessments.

Drop comfort for each eye was assessed using subject-reported drop comfort scale, which is a 0 to 10 scale and where higher numbers indicating more uncomfortable.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=349 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=348 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Drop Comfort Assessment After Randomization at Visit 2
2.2 Score on a scale
Standard Deviation 2.31
2.0 Score on a scale
Standard Deviation 2.23

SECONDARY outcome

Timeframe: Day 8, Day 15

Population: Intent-to-treat population; number of participants analyzed varies by outcome due to missing assessments.

The severity of dry eye disease and its impact on vision-related function were measured using the Ocular Surface Disease Index. Scores range from 0 to 100, with higher scores indicating greater disability.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=351 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=348 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Ocular Surface Disease Index (OSDI)© at Visits 3 and 4 (Pre-CAE®)
Total OSDI Score - Visit 3 (Day 8)
32.2 Score on a scale
Standard Deviation 19.74
33.7 Score on a scale
Standard Deviation 19.64
Ocular Surface Disease Index (OSDI)© at Visits 3 and 4 (Pre-CAE®)
Total OSDI Score - Visit 4 (Day 15)
31.3 Score on a scale
Standard Deviation 19.79
32.3 Score on a scale
Standard Deviation 19.87

SECONDARY outcome

Timeframe: Day 8, Day 15

Population: Intent-to-treat population; number of participants analyzed varies by outcome due to missing assessments.

The severity of 5 symptoms was measured using the Ora Calibra® Ocular Discomfort \& 4-Symptom Questionnaire which is a 0 to 5 scale and where higher numbers indicating more severe discomfort.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=351 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=348 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Ocular Discomfort & 4-Symptom Questionnaire at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®), Excluding the Hierarchical Efficacy Measure
Ocular Discomfort - Visit 3 (Day 8), Pre-CAE
2.4 Score
Standard Deviation 1.19
2.4 Score
Standard Deviation 1.15
Ocular Discomfort & 4-Symptom Questionnaire at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®), Excluding the Hierarchical Efficacy Measure
Ocular Discomfort - Visit 4 (Day 15), Pre-CAE
2.3 Score
Standard Deviation 1.20
2.3 Score
Standard Deviation 1.21
Ocular Discomfort & 4-Symptom Questionnaire at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®), Excluding the Hierarchical Efficacy Measure
Ocular Discomfort - Visit 4 (Day 15), Post-CAE
3.3 Score
Standard Deviation 0.98
3.4 Score
Standard Deviation 0.96
Ocular Discomfort & 4-Symptom Questionnaire at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®), Excluding the Hierarchical Efficacy Measure
Ocular Discomfort - Visit 4 (Day 15), Change from Pre-CAE to Post-CAE
1.1 Score
Standard Deviation 1.28
1.1 Score
Standard Deviation 1.24

SECONDARY outcome

Timeframe: Day 8, Day 15

Population: Intent-to-treat population; number of participants analyzed varies by outcome due to missing assessments.

Subjects' perceived severity of ocular discomfort was measured using the Ocular Discomfort Scale which is a 0 to 4 scale and where higher numbers indicating more severe discomfort.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=351 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=348 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Ocular Discomfort Outside CAE® at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®)
Visit 3 (Day 8), Pre-CAE
2.3 Score on a scale
Standard Deviation 1.16
2.2 Score on a scale
Standard Deviation 1.12
Ocular Discomfort Outside CAE® at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®)
Visit 4 (Day 15), Change from Pre-CAE to Post-CAE
1.2 Score on a scale
Standard Deviation 1.27
1.2 Score on a scale
Standard Deviation 1.36
Ocular Discomfort Outside CAE® at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®)
Visit 4 (Day 15), Pre-CAE
2.1 Score on a scale
Standard Deviation 1.13
2.1 Score on a scale
Standard Deviation 1.19
Ocular Discomfort Outside CAE® at Visits 3 and 4 (Change From Pre-CAE® to Post-CAE®, Pre- and Post-CAE®)
Visit 4 (Day 15), Post-CAE
3.3 Score on a scale
Standard Deviation 0.92
3.3 Score on a scale
Standard Deviation 0.88

SECONDARY outcome

Timeframe: Day 15

Population: Intent-to-treat population; number of participants analyzed varies by outcome due to missing assessments.

Subjects' perceived severity of ocular discomfort during CAE® exposure was measured using the Ocular Discomfort Scale which is a 0 to 4 scale and where higher numbers indicating more severe discomfort.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=344 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=332 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Ocular Discomfort During CAE® at Visit 4
1.5 Score on a scale
Standard Deviation 1.11
1.7 Score on a scale
Standard Deviation 1.06

OTHER_PRE_SPECIFIED outcome

Timeframe: Day -14 ± 1 day, Day 1, Day 8, Day 15

Population: Visual acuity is a safety assessment and all safety assessments were analyzed using the safety population, which differed from the intent-to-treat (ITT) population that included all randomized subjects, due to one participant in placebo arm who was consented, enrolled twice, and another participant in 0.1% RGN-259 arm who was randomized, but not treated.

LogMAR Visual acuity was measured using ETDRS chart at Visits 1, 2, 3 and 4 and represented in LogMAR.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=350 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=349 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Visual Acuity (Using an ETDRS (Early Treatment of Diabetic Retinopathy Study) Chart) at Visits 1, 2, 3 and 4
BCVA (Better Corrected Visual Acuity) in study eye at Visit 1 (Day -14)
0.135 logMAR
Standard Deviation 0.1568
0.128 logMAR
Standard Deviation 0.1424
Visual Acuity (Using an ETDRS (Early Treatment of Diabetic Retinopathy Study) Chart) at Visits 1, 2, 3 and 4
BCVA in study eye at Visit 2 (Day 1)
0.134 logMAR
Standard Deviation 0.1658
0.120 logMAR
Standard Deviation 0.1504
Visual Acuity (Using an ETDRS (Early Treatment of Diabetic Retinopathy Study) Chart) at Visits 1, 2, 3 and 4
BCVA in study eye at Visit 3 (Day 8)
0.124 logMAR
Standard Deviation 0.1637
0.112 logMAR
Standard Deviation 0.1499
Visual Acuity (Using an ETDRS (Early Treatment of Diabetic Retinopathy Study) Chart) at Visits 1, 2, 3 and 4
BCVA in study eye at Visit 4 (Day 15)
0.118 logMAR
Standard Deviation 0.1629
0.106 logMAR
Standard Deviation 0.1466

OTHER_PRE_SPECIFIED outcome

Timeframe: Day -14 ± 1 day, Day 1, Day 8, Day 15

Population: Slit-lamp biomicroscopy is a safety assessment and all safety assessments were analyzed using the safety population, which differed from the intent-to-treat (ITT) population that included all randomized subjects, due to one participant in placebo arm who was consented, enrolled twice, and another participant in 0.1% RGN-259 arm who was randomized, but not treated.

Slit-lamp biomicroscopy at Visits 1, 2, 3 and 4

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=350 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=349 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Slit-lamp Biomicroscopy at Visits 1, 2, 3 and 4
Abnormal findings at Visit 1 (Day -14), Pre-CAE
1 Participants
0 Participants
Slit-lamp Biomicroscopy at Visits 1, 2, 3 and 4
Abnormal findings at Visit 1 (Day -14), Post-CAE
1 Participants
0 Participants
Slit-lamp Biomicroscopy at Visits 1, 2, 3 and 4
Abnormal findings at Visit 2 (Day 1), Pre-CAE
1 Participants
0 Participants
Slit-lamp Biomicroscopy at Visits 1, 2, 3 and 4
Abnormal findings at Visit 2 (Day 1), Post-CAE
1 Participants
0 Participants
Slit-lamp Biomicroscopy at Visits 1, 2, 3 and 4
Abnormal findings at Visit 3 (Day 8), Pre-CAE
1 Participants
0 Participants
Slit-lamp Biomicroscopy at Visits 1, 2, 3 and 4
Abnormal findings at Visit 4 (Day 15), Pre-CAE
1 Participants
0 Participants
Slit-lamp Biomicroscopy at Visits 1, 2, 3 and 4
Abnormal findings at Visit 4 (Day 15), Post-CAE
1 Participants
0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Day -14 ± 1 day, Day 15

Population: Corneal Sensitivity is a safety assessment and all safety assessments were analyzed using the safety population, which differed from the intent-to-treat (ITT) population that included all randomized subjects, due to one participant in placebo arm who was consented, enrolled twice, and another participant in 0.1% RGN-259 arm who was randomized, but not treated.

Corneal sensitivity was measured using a Cochet-Bonnet esthesiometer by applying gentle pressure to the central corneal surface and recording the filament length (mm) at which the subject perceived the sensation. Higher values (longer filament lengths) indicate greater corneal sensitivity and a better outcome.

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=350 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=349 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Corneal Sensitivity at Visits 1 and 4 (Pre-CAE®)
Corneal sensitivity in study eye at Visit 1 (Day -14)
57.8 mm
Standard Deviation 5.89
58.2 mm
Standard Deviation 4.70
Corneal Sensitivity at Visits 1 and 4 (Pre-CAE®)
Corneal sensitivity in study eye at Visit 4 (Day 15)
57.5 mm
Standard Deviation 7.11
58.4 mm
Standard Deviation 4.32

OTHER_PRE_SPECIFIED outcome

Timeframe: Day -14 ± 1 day, Day 15

Population: Undilated Fundoscopy is a safety assessment and all safety assessments were analyzed using the safety population, which differed from the intent-to-treat (ITT) population that included all randomized subjects, due to one participant in placebo arm who was consented, enrolled twice, and another participant in 0.1% RGN-259 arm who was randomized, but not treated.

Undilated Fundoscopy at Visits 1 and 4

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=350 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=349 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Undilated Fundoscopy at Visits 1 and 4
Abnormal findings in study eye at baseline Visit 1 (Day -14)
0 Participants
0 Participants
Undilated Fundoscopy at Visits 1 and 4
Abnormal findings in study eye at Visit 4 (Day 15)
0 Participants
0 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Day -14 ± 1 day, Day 15

Population: Intraocular Pressure is a safety assessment and all safety assessments were analyzed using the safety population, which differed from the intent-to-treat (ITT) population that included all randomized subjects, due to one participant in placebo arm who was consented, enrolled twice, and another participant in 0.1% RGN-259 arm who was randomized, but not treated.

Intraocular Pressure (mmHg) at Visits 1 and 4

Outcome measures

Outcome measures
Measure
Experimental: RGN-259
n=350 Participants
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=349 Participants
It is composed of the same excipients as RGN-259 but does not contain Tβ4
Intraocular Pressure at Visits 1 and 4
Intraocular Pressure in study eye at Visit 1 (Day -14)
14.5 mmHg
Standard Deviation 2.65
14.8 mmHg
Standard Deviation 2.83
Intraocular Pressure at Visits 1 and 4
Intraocular Pressure in study eye at Visit 4 (Day 15)
14.5 mmHg
Standard Deviation 2.79
14.8 mmHg
Standard Deviation 2.96

Adverse Events

Experimental: RGN-259

Serious events: 0 serious events
Other events: 47 other events
Deaths: 0 deaths

Placebo Comparator: Placebo

Serious events: 0 serious events
Other events: 31 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Experimental: RGN-259
n=350 participants at risk
It is a preservative-free, sterile eye drop solution containing Tβ4 for direct instillation into both eyes, four times per day for 14 days
Placebo Comparator: Placebo
n=349 participants at risk
It is composed of the same excipients as RGN-259 but does not contain Tβ4
General disorders
Instillation site pain
6.6%
23/350 • Adverse events were collected at every visit through study completion, approximately 4 weeks.
The safety population included 699 subjects. One participant completed the study twice and received placebo in both periods; this subject was counted once in the ITT population but twice in the safety population. Another participant randomized to the 0.1% RGN-259 arm was not treated and was included only in the ITT population. Accordingly, the ITT population comprised 351 in the 0.1% RGN-259 arm and 348 in the placebo arm, while the safety population comprised 350 and 349, respectively.
4.6%
16/349 • Adverse events were collected at every visit through study completion, approximately 4 weeks.
The safety population included 699 subjects. One participant completed the study twice and received placebo in both periods; this subject was counted once in the ITT population but twice in the safety population. Another participant randomized to the 0.1% RGN-259 arm was not treated and was included only in the ITT population. Accordingly, the ITT population comprised 351 in the 0.1% RGN-259 arm and 348 in the placebo arm, while the safety population comprised 350 and 349, respectively.
General disorders
Instillation site irritation
1.4%
5/350 • Adverse events were collected at every visit through study completion, approximately 4 weeks.
The safety population included 699 subjects. One participant completed the study twice and received placebo in both periods; this subject was counted once in the ITT population but twice in the safety population. Another participant randomized to the 0.1% RGN-259 arm was not treated and was included only in the ITT population. Accordingly, the ITT population comprised 351 in the 0.1% RGN-259 arm and 348 in the placebo arm, while the safety population comprised 350 and 349, respectively.
0.57%
2/349 • Adverse events were collected at every visit through study completion, approximately 4 weeks.
The safety population included 699 subjects. One participant completed the study twice and received placebo in both periods; this subject was counted once in the ITT population but twice in the safety population. Another participant randomized to the 0.1% RGN-259 arm was not treated and was included only in the ITT population. Accordingly, the ITT population comprised 351 in the 0.1% RGN-259 arm and 348 in the placebo arm, while the safety population comprised 350 and 349, respectively.
Eye disorders
Visual acuity reduced
3.4%
12/350 • Adverse events were collected at every visit through study completion, approximately 4 weeks.
The safety population included 699 subjects. One participant completed the study twice and received placebo in both periods; this subject was counted once in the ITT population but twice in the safety population. Another participant randomized to the 0.1% RGN-259 arm was not treated and was included only in the ITT population. Accordingly, the ITT population comprised 351 in the 0.1% RGN-259 arm and 348 in the placebo arm, while the safety population comprised 350 and 349, respectively.
2.0%
7/349 • Adverse events were collected at every visit through study completion, approximately 4 weeks.
The safety population included 699 subjects. One participant completed the study twice and received placebo in both periods; this subject was counted once in the ITT population but twice in the safety population. Another participant randomized to the 0.1% RGN-259 arm was not treated and was included only in the ITT population. Accordingly, the ITT population comprised 351 in the 0.1% RGN-259 arm and 348 in the placebo arm, while the safety population comprised 350 and 349, respectively.
Infections and infestations
Nasopharyngitis
2.0%
7/350 • Adverse events were collected at every visit through study completion, approximately 4 weeks.
The safety population included 699 subjects. One participant completed the study twice and received placebo in both periods; this subject was counted once in the ITT population but twice in the safety population. Another participant randomized to the 0.1% RGN-259 arm was not treated and was included only in the ITT population. Accordingly, the ITT population comprised 351 in the 0.1% RGN-259 arm and 348 in the placebo arm, while the safety population comprised 350 and 349, respectively.
1.7%
6/349 • Adverse events were collected at every visit through study completion, approximately 4 weeks.
The safety population included 699 subjects. One participant completed the study twice and received placebo in both periods; this subject was counted once in the ITT population but twice in the safety population. Another participant randomized to the 0.1% RGN-259 arm was not treated and was included only in the ITT population. Accordingly, the ITT population comprised 351 in the 0.1% RGN-259 arm and 348 in the placebo arm, while the safety population comprised 350 and 349, respectively.

Additional Information

Director of Clinical Operation

ReGenTree, LLC

Phone: TBD

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place