Trial Outcomes & Findings for A Phase 2 Study Evaluating the Safety and Efficacy of Netarsudil Ophthalmic Solution in Patients With Corneal Edema Due to Fuchs Corneal Dystrophy (NCT NCT04498169)

NCT ID: NCT04498169

Last Updated: 2022-09-15

Results Overview

Mean change from baseline in CCT by ultrasound pachymetry

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 40 participants
• Primary outcome timeframe: Baseline & 4 weeks
• Results posted on: 2022-09-15

Participant Flow

Recruitment took place at 12 clinic sites in the United States between September 2020 and June 2021.

All participants underwent a washout of their pre-study treatment for Fuchs Corneal Dystrophy for a period of 24 hours prior to the baseline visit.

Participant milestones

Measure	Once Daily Netarsudil Ophthalmic Solution One drop of study artificial tear in the study eye in the morning, and one drop of Netarsudil 0.02% ophthalmic solution in the study eye in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution	Twice Daily Netarsudil Ophthalmic Solution One drop of Netarsudil 0.02% ophthalmic solution in the study eye in the morning and in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution
Modified Intent-to-Treat Population STARTED	20	20
Modified Intent-to-Treat Population COMPLETED	19	19
Modified Intent-to-Treat Population NOT COMPLETED	1	1
Safety Population STARTED	20	20
Safety Population COMPLETED	19	19
Safety Population NOT COMPLETED	1	1
Per-Protocol Population STARTED	18	16
Per-Protocol Population COMPLETED	18	15
Per-Protocol Population NOT COMPLETED	0	1
Study Completion STARTED	20	20
Study Completion COMPLETED	19	19
Study Completion NOT COMPLETED	1	1

Reasons for withdrawal

Measure	Once Daily Netarsudil Ophthalmic Solution One drop of study artificial tear in the study eye in the morning, and one drop of Netarsudil 0.02% ophthalmic solution in the study eye in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution	Twice Daily Netarsudil Ophthalmic Solution One drop of Netarsudil 0.02% ophthalmic solution in the study eye in the morning and in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution
Study Completion Adverse Event	1	1

Baseline Characteristics

A Phase 2 Study Evaluating the Safety and Efficacy of Netarsudil Ophthalmic Solution in Patients With Corneal Edema Due to Fuchs Corneal Dystrophy

Baseline characteristics by cohort

Measure	Once Daily Netarsudil Ophthalmic Solution n=20 Participants One drop of study artificial tear in the study eye in the morning, and one drop of Netarsudil 0.02% ophthalmic solution in the study eye in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution	Twice Daily Netarsudil Ophthalmic Solution n=20 Participants One drop of Netarsudil 0.02% ophthalmic solution in the study eye in the morning and in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution	Total n=40 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=93 Participants	9 Participants n=4 Participants	18 Participants n=27 Participants
Age, Categorical >=65 years	11 Participants n=93 Participants	11 Participants n=4 Participants	22 Participants n=27 Participants
Sex: Female, Male Female	9 Participants n=93 Participants	12 Participants n=4 Participants	21 Participants n=27 Participants
Sex: Female, Male Male	11 Participants n=93 Participants	8 Participants n=4 Participants	19 Participants n=27 Participants
Race/Ethnicity, Customized Black/African American	0 Participants n=93 Participants	2 Participants n=4 Participants	2 Participants n=27 Participants
Race/Ethnicity, Customized White	20 Participants n=93 Participants	18 Participants n=4 Participants	38 Participants n=27 Participants
Region of Enrollment United States	20 participants n=93 Participants	20 participants n=4 Participants	40 participants n=27 Participants

PRIMARY outcome

Timeframe: Baseline & 4 weeks

Population: Modified Intent-to-Treat Population

Mean change from baseline in CCT by ultrasound pachymetry

Outcome measures

Measure	Once Daily Netarsudil Ophthalmic Solution n=20 Participants One drop of study artificial tear in the study eye in the morning, and one drop of Netarsudil 0.02% ophthalmic solution in the study eye in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution	Twice Daily Netarsudil Ophthalmic Solution n=20 Participants One drop of Netarsudil 0.02% ophthalmic solution in the study eye in the morning and in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution
Central Corneal Thickness (CCT)	-28.4 µm Standard Deviation 35.72	-20.1 µm Standard Deviation 39.12

Adverse Events

Once Daily Netarsudil Ophthalmic Solution

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Twice Daily Netarsudil Ophthalmic Solution

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Measure	Once Daily Netarsudil Ophthalmic Solution n=20 participants at risk One drop of study artificial tear in the study eye in the morning, and one drop of Netarsudil 0.02% ophthalmic solution in the study eye in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution	Twice Daily Netarsudil Ophthalmic Solution n=20 participants at risk One drop of Netarsudil 0.02% ophthalmic solution in the study eye in the morning and in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution
Gastrointestinal disorders Viral gastroenteritis	0.00% 0/20 • Adverse events were documented from the time of signing the informed consent until the last study visit for each participant. This was a period of approximately 8 weeks (56 days) for participants who completed the full study duration. For AEs ongoing at the time of study completion, the ongoing AE was followed-up and the participant was provided appropriate medical care until the event resolved or stabilized, the participant was lost to follow-up, or there was other resolution to the event. Adverse events information was collected at all study visits.	5.0% 1/20 • Number of events 1 • Adverse events were documented from the time of signing the informed consent until the last study visit for each participant. This was a period of approximately 8 weeks (56 days) for participants who completed the full study duration. For AEs ongoing at the time of study completion, the ongoing AE was followed-up and the participant was provided appropriate medical care until the event resolved or stabilized, the participant was lost to follow-up, or there was other resolution to the event. Adverse events information was collected at all study visits.

Other adverse events

Measure	Once Daily Netarsudil Ophthalmic Solution n=20 participants at risk One drop of study artificial tear in the study eye in the morning, and one drop of Netarsudil 0.02% ophthalmic solution in the study eye in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution	Twice Daily Netarsudil Ophthalmic Solution n=20 participants at risk One drop of Netarsudil 0.02% ophthalmic solution in the study eye in the morning and in the evening for an 8 week period in up to 20 subjects. Netarsudil Ophthalmic: Netarsudil Ophthalmic Solution 0.02% Topical Sterile Ophthalmic Solution
Eye disorders Corneal verticillata	15.0% 3/20 • Number of events 3 • Adverse events were documented from the time of signing the informed consent until the last study visit for each participant. This was a period of approximately 8 weeks (56 days) for participants who completed the full study duration. For AEs ongoing at the time of study completion, the ongoing AE was followed-up and the participant was provided appropriate medical care until the event resolved or stabilized, the participant was lost to follow-up, or there was other resolution to the event. Adverse events information was collected at all study visits.	10.0% 2/20 • Number of events 2 • Adverse events were documented from the time of signing the informed consent until the last study visit for each participant. This was a period of approximately 8 weeks (56 days) for participants who completed the full study duration. For AEs ongoing at the time of study completion, the ongoing AE was followed-up and the participant was provided appropriate medical care until the event resolved or stabilized, the participant was lost to follow-up, or there was other resolution to the event. Adverse events information was collected at all study visits.
Eye disorders Conjunctival hypaeremia	10.0% 2/20 • Number of events 4 • Adverse events were documented from the time of signing the informed consent until the last study visit for each participant. This was a period of approximately 8 weeks (56 days) for participants who completed the full study duration. For AEs ongoing at the time of study completion, the ongoing AE was followed-up and the participant was provided appropriate medical care until the event resolved or stabilized, the participant was lost to follow-up, or there was other resolution to the event. Adverse events information was collected at all study visits.	20.0% 4/20 • Number of events 4 • Adverse events were documented from the time of signing the informed consent until the last study visit for each participant. This was a period of approximately 8 weeks (56 days) for participants who completed the full study duration. For AEs ongoing at the time of study completion, the ongoing AE was followed-up and the participant was provided appropriate medical care until the event resolved or stabilized, the participant was lost to follow-up, or there was other resolution to the event. Adverse events information was collected at all study visits.
Eye disorders Erythema of eyelid	0.00% 0/20 • Adverse events were documented from the time of signing the informed consent until the last study visit for each participant. This was a period of approximately 8 weeks (56 days) for participants who completed the full study duration. For AEs ongoing at the time of study completion, the ongoing AE was followed-up and the participant was provided appropriate medical care until the event resolved or stabilized, the participant was lost to follow-up, or there was other resolution to the event. Adverse events information was collected at all study visits.	10.0% 2/20 • Number of events 2 • Adverse events were documented from the time of signing the informed consent until the last study visit for each participant. This was a period of approximately 8 weeks (56 days) for participants who completed the full study duration. For AEs ongoing at the time of study completion, the ongoing AE was followed-up and the participant was provided appropriate medical care until the event resolved or stabilized, the participant was lost to follow-up, or there was other resolution to the event. Adverse events information was collected at all study visits.

Additional Information

Michelle Senchyna, PhD. VP, Clinical Development and Medical Affairs.

Aerie Pharmaceuticals Inc.

Phone: +1 908-947-3551

Email: msenchyna@aeriepharma.com

Results disclosure agreements

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