Assessment of the Safety and Efficacy of RGN-259 Ophthalmic Solutions for Dry Eye Syndrome : ARISE-2

• ClinicalTrials.gov Identifier: NCT02974907
• Recruitment Status: Completed
• First Posted: November 29, 2016
• Results First Posted: January 5, 2022
• Last Update Posted: January 5, 2022
• Sponsor: ReGenTree, LLC
• Information provided by (Responsible Party): ReGenTree, LLC

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Dry Eye Syndrome
• Interventions: Drug: RGN-259; Drug: Placebo
• Enrollment: 601

Participant Flow

Recruitment Details	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 28 days. The study comprised of 5 visits over the course of approximately 6 weeks.
Pre-assignment Details

Arm/Group Title	RGN-259	Placebo
Arm/Group Description	Active (0.1% RGN-259 ophthalmic solution)	Placebo (Placebo ophthalmic solution)
Period Title: Overall Study
Started	299	302
Completed	286	292
Not Completed	13	10

Baseline Characteristics

Arm/Group Title		RGN-259	Placebo	Total
Arm/Group Description		Active (0.1% RGN-259 ophthalmic solution)	Placebo (Placebo ophthalmic solution)	Total of all reporting groups
Overall Number of Baseline Participants		299	302	601
Baseline Analysis Population Description		[Not Specified]
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	299 participants	302 participants	601 participants
		61.9 (11.97)	63.0 (11.48)	62.5 (11.73)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	299 participants	302 participants	601 participants
	Female	215 71.9%	223 73.8%	438 72.9%
	Male	84 28.1%	79 26.2%	163 27.1%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	299 participants	302 participants	601 participants
	Hispanic or Latino	23 7.7%	24 7.9%	47 7.8%
	Not Hispanic or Latino	275 92.0%	278 92.1%	553 92.0%
	Unknown or Not Reported	1 0.3%	0 0.0%	1 0.2%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	299 participants	302 participants	601 participants
	American Indian or Alaska Native	0 0.0%	2 0.7%	2 0.3%
	Asian	9 3.0%	10 3.3%	19 3.2%
	Native Hawaiian or Other Pacific Islander	1 0.3%	1 0.3%	2 0.3%
	Black or African American	36 12.0%	41 13.6%	77 12.8%
	White	251 83.9%	246 81.5%	497 82.7%
	More than one race	0 0.0%	1 0.3%	1 0.2%
	Unknown or Not Reported	2 0.7%	1 0.3%	3 0.5%

Outcome Measures

1. Primary Outcome

Title	Ocular Discomfort
Description	Change from Baseline at Day 29 using the Ora Calibra® Ocular Discomfort Scale (6-point scale where 0 = none and 5 = worst)
Time Frame	29 days after first dosing

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	RGN-259	Placebo
Arm/Group Description:	Active (0.1% RGN-259 ophthalmic solution)	Placebo (Placebo ophthalmic solution)
Overall Number of Participants Analyzed	299	302
Mean (95% Confidence Interval); Unit of Measure: score on a scale
	0.07; (-0.05 to 0.19)	-0.04; (-0.15 to 0.08)

2. Primary Outcome

Title	Corneal Fluorescein Staining
Description	Change from Baseline at Day 29 using the Ora Calibra® scale (5-point scale with half (0.5) increments where 0 = none and 4 = severe)
Time Frame	29 days after first dosing

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	RGN-259	Placebo
Arm/Group Description:	Active (0.1% RGN-259 ophthalmic solution)	Placebo (Placebo ophthalmic solution)
Overall Number of Participants Analyzed	111	107
Mean (95% Confidence Interval); Unit of Measure: score on a scale
	0.07; (-0.06 to 0.20)	-0.01; (-0.13 to 0.12)

3. Secondary Outcome

Title	Corneal Fluorescein Staining
Description	Comparing each of active group & Placebo.
Time Frame	8, 15, 29 days after first dosing

Outcome Measure Data Not Reported

4. Secondary Outcome

Title	Unanesthetized Schirmer's Test
Description	Comparing each of active group & Placebo.
Time Frame	29 days after first dosing

Outcome Measure Data Not Reported

5. Secondary Outcome

Title	Ocular Surface Disease Index (OSDI)©
Description	Comparing each of active group & Placebo.
Time Frame	8, 15, 29 days after first dosing

Outcome Measure Data Not Reported

6. Secondary Outcome

Title	Tear Film Break-Up Time
Description	Comparing each of active group & Placebo.
Time Frame	8, 15, 29 days after first dosing

Outcome Measure Data Not Reported

7. Other Pre-specified Outcome

Title	Visual Acuity
Description	Change or shifts from Baseline
Time Frame	1, 8, 15, 29 days

Outcome Measure Data Not Reported

8. Other Pre-specified Outcome

Title	Change in Biomicroscopy Using the Slit-lamp
Description	Change or shifts from Baseline
Time Frame	1, 8, 15, 29 days

Outcome Measure Data Not Reported

9. Other Pre-specified Outcome

Title	Adverse Event Query
Description	Frequencies
Time Frame	1, 8, 15, 29 days

Outcome Measure Data Not Reported

10. Other Pre-specified Outcome

Title	Change in Biomicroscopy Using the Undilated Fundoscopy
Description	Change or shifts from Baseline
Time Frame	1, 29 days

Outcome Measure Data Not Reported

Adverse Events

Time Frame	Adverse events were collected at every visit through study completion, up to Day 29.
Adverse Event Reporting Description	There was no anticipated/unanticipated deaths due to any cause in this study.
Arm/Group Title	RGN-259		Placebo
Arm/Group Description	Active (0.1% RGN-259 ophthalmic solution)		Placebo (Placebo ophthalmic solution)
All-Cause Mortality
	RGN-259		Placebo
	Affected / at Risk (%)		Affected / at Risk (%)
Total	0/299 (0.00%)		0/302 (0.00%)
Serious Adverse Events
	RGN-259		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	3/299 (1.00%)		2/302 (0.66%)
Ear and labyrinth disorders
Vertigo †	1/299 (0.33%)	1	0/302 (0.00%)	0
Infections and infestations
Diverticulitis †	1/299 (0.33%)	1	0/302 (0.00%)	0
Urinary Tract Infection †	0/299 (0.00%)	0	1/302 (0.33%)	1
Metabolism and nutrition disorders
Hyperglycemia †	1/299 (0.33%)	1	0/302 (0.00%)	0
Nervous system disorders
Transient Ischemic Attack †	0/299 (0.00%)	0	1/302 (0.33%)	1
† Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	1%
	RGN-259		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	19/299 (6.35%)		21/302 (6.95%)
Eye disorders
Visual Acuity Reduced †	1/299 (0.33%)	1	10/302 (3.31%)	12
Eye Pain †	3/299 (1.00%)	3	1/302 (0.33%)	1
Infections and infestations
Nasopharyngitis †	9/299 (3.01%)	9	6/302 (1.99%)	6
Sinusitis †	3/299 (1.00%)	3	3/302 (0.99%)	3
Upper Respiratory Tract Infection †	3/299 (1.00%)	3	1/302 (0.33%)	1
† Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Shinwook Kang
• Organization: ReGenTree, LLC
• Phone: 609-649-5505
• EMail: swkang@regentreellc.com

• Responsible Party: ReGenTree, LLC
• ClinicalTrials.gov Identifier: NCT02974907
• Other Study ID Numbers: RGN-259/16-110-0008
• First Submitted: November 23, 2016
• First Posted: November 29, 2016
• Results First Submitted: November 5, 2021
• Results First Posted: January 5, 2022
• Last Update Posted: January 5, 2022