Trial Outcomes & Findings for Efficacy and Safety of APP13007 for Treatment of Inflammation and Pain After Cataract Surgery (NCT NCT04739709)

NCT ID: NCT04739709

Last Updated: 2023-07-20

Results Overview

The number of cells in the anterior chamber of the eye are counted using slit-lamp biomicroscopy at each study visit and the results are reported as number of cells in a 1mm x 1mm area.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 378 participants
• Primary outcome timeframe: Postoperative Day 8 and Postoperative Day 15
• Results posted on: 2023-07-20

Participant Flow

A multi-center US study in which 27 sites recruited subjects between January 2021 and February 2022.

Of 456 enrolled participants, 378 met inclusion criteria and were randomized to treatment.

Participant milestones

Measure	APP13007 0.05% BID 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Overall Study STARTED	181	197
Overall Study COMPLETED	180	197
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Measure	APP13007 0.05% BID 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Overall Study Physician Decision	1	0

Baseline Characteristics

Efficacy and Safety of APP13007 for Treatment of Inflammation and Pain After Cataract Surgery

Baseline characteristics by cohort

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop	Total n=378 Participants Total of all reporting groups
Age, Continuous	68.7 Years STANDARD_DEVIATION 8.9 • n=5 Participants	67.8 Years STANDARD_DEVIATION 8.6 • n=7 Participants	68.3 Years STANDARD_DEVIATION 8.7 • n=5 Participants
Sex: Female, Male Female	106 Participants n=5 Participants	119 Participants n=7 Participants	225 Participants n=5 Participants
Sex: Female, Male Male	75 Participants n=5 Participants	78 Participants n=7 Participants	153 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	35 Participants n=5 Participants	52 Participants n=7 Participants	87 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	146 Participants n=5 Participants	145 Participants n=7 Participants	291 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Asian	11 Participants n=5 Participants	16 Participants n=7 Participants	27 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	18 Participants n=5 Participants	27 Participants n=7 Participants	45 Participants n=5 Participants
Race (NIH/OMB) White	150 Participants n=5 Participants	151 Participants n=7 Participants	301 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	181 participants n=5 Participants	197 participants n=7 Participants	378 participants n=5 Participants

PRIMARY outcome

Timeframe: Postoperative Day 8 and Postoperative Day 15

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

The number of cells in the anterior chamber of the eye are counted using slit-lamp biomicroscopy at each study visit and the results are reported as number of cells in a 1mm x 1mm area.

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants With ACC Count = 0 at All Visits From Postoperative Day 8 Through Postoperative Day 15 Without Rescue Medication	48 Participants	10 Participants

PRIMARY outcome

Timeframe: Postoperative Day 4, Postoperative Day 8 and Postoperative Day 15

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

Ocular pain is measured in each eye using a subject-reported five-point (0-4) ocular pain grade scale (0 = no pain; 1 = minimal; 2 = mild; 3 = moderate; 4 = severe pain).

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants With Ocular Pain Grade = 0 at All Visits From Postoperative Day 4 Through Postoperative Day 15 Without Rescue Medication	123 Participants	46 Participants

PRIMARY outcome

Timeframe: From First dose to Postoperative Day 22

Population: Safety Population

Number of participants with ocular and systemic treatment-emergent AEs.

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants With Treatment-emergent Adverse Events (AEs)	38 Participants	39 Participants

SECONDARY outcome

Timeframe: Postoperative Day 8

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

The number of cells in the anterior chamber of the eye are counted using slit-lamp biomicroscopy at each study visit and the results are reported as number of cells in a 1mm x 1mm area. ACC Grade 0 = 0 cells, ACC grade 1 = 1-5 cells, ACC grade 2 = 6-15 cells, ACC grade 3 = 16-30 cells, ACC grade 4 = >30 cells

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants With Anterior Chamber Cell Grade = 0 at POD8 Without Rescue Medication	59 Participants	23 Participants

SECONDARY outcome

Timeframe: Postoperative Day 15

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

The number of cells in the anterior chamber of the eye are counted using slit-lamp biomicroscopy at each study visit and the results are reported as number of cells in a 1mm x 1mm area. ACC Grade 0 = 0 cells, ACC grade 1 = 1-5 cells, ACC grade 2 = 6-15 cells, ACC grade 3 = 16-30 cells, ACC grade 4 = >30 cells

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants With Anterior Chamber Cell Grade = 0 at POD15 Without Rescue Medication	106 Participants	31 Participants

SECONDARY outcome

Timeframe: Postoperative Day 4

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

Ocular pain is measured in each eye using a subject-reported five-point (0-4) ocular pain grade scale (0 = no pain; 4 = severe pain).

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants With Ocular Pain Grade = 0 at POD4 Without Rescue Medication	140 Participants	86 Participants

SECONDARY outcome

Timeframe: Postoperative Day 8

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

Ocular pain is measured in each eye using a subject-reported five-point (0-4) ocular pain grade scale (0 = no pain; 4 = severe pain).

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants With Ocular Pain Grade = 0 at POD8 Without Rescue Medication	149 Participants	84 Participants

SECONDARY outcome

Timeframe: Postoperative Day 15

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

Ocular pain is measured in each eye using a subject-reported five-point (0-4) ocular pain grade scale (0 = no pain; 4 = severe pain).

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants With Ocular Pain Grade = 0 at POD15 Without Rescue Medication	164 Participants	83 Participants

SECONDARY outcome

Timeframe: Postoperative Day 4

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

The degree of turbidity (flare) in the anterior chamber of the eye is measured using slit-lamp biomicroscopy and quantified on a five-point (0-4) anterior chamber flare grade scale (0 = none; 4 = Intense).

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants With Anterior Chamber Flare (ACF) = 0 at POD4 Without Rescue Medication	111 Participants	79 Participants

SECONDARY outcome

Timeframe: Postoperative Day 8

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

The degree of turbidity (flare) in the anterior chamber of the eye is measured using slit-lamp biomicroscopy and quantified on a five-point (0-4) anterior chamber flare grade scale (0 = none; 4 = Intense).

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants With Anterior Chamber Flare (ACF) = 0 at POD8 Without Rescue Medication	142 Participants	75 Participants

SECONDARY outcome

Timeframe: Postoperative Day 15

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

The degree of turbidity (flare) in the anterior chamber of the eye is measured using slit-lamp biomicroscopy and quantified on a five-point (0-4) anterior chamber flare grade scale (0 = none; 4 = Intense).

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants With Anterior Chamber Flare (ACF) = 0 at POD15 Without Rescue Medication	159 Participants	65 Participants

SECONDARY outcome

Timeframe: Baseline and Postoperative Day 15

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

The number of cells in the anterior chamber of the eye are counted using slit-lamp biomicroscopy and the results are reported as number of cells in a 1mm x 1mm area using a 5 point grading scale. ACC Grade 0 = 0 cells, ACC grade 1 = 1-5 cells, ACC grade 2 = 6-15 cells, ACC grade 3 = 16-30 cells, ACC grade 4 = >30 cells

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Anterior Chamber Cell Grade - Change From Baseline (POD1 Prior to Dosing) to POD 15	-2.1 grade on a scale Standard Error 0.1	-0.6 grade on a scale Standard Error 0.1

SECONDARY outcome

Timeframe: Baseline and Postoperative Day 15

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

Ocular pain is measured in each eye using a subject-reported five-point (0-4) ocular pain grade scale (0 = no pain; 4 = severe pain).

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Ocular Pain Grade - Change From Baseline (POD1 Prior to Dosing) to POD 15	-1.1 grade on a scale Standard Error 0.1	-0.3 grade on a scale Standard Error 0.1

SECONDARY outcome

Timeframe: Baseline and Postoperative Day 15

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

The degree of turbidity (flare) in the anterior chamber of the eye is measured using slit-lamp biomicroscopy and quantified on a five-point (0-4) anterior chamber flare grade scale (0 = none; 4 = Intense).

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Anterior Chamber Flare (ACF) - Change From Baseline (POD1 Prior to Dosing) to POD 15	-0.7 grade on a scale Standard Error 0.1	-0.0 grade on a scale Standard Error 0.0

SECONDARY outcome

Timeframe: Baseline and Postoperative Day 4

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

The visual acuity in each eye is corrected using the pinhole method and the acuity is quantified on a logMAR (minimal angle of resolution) scale using the 'Early Treatment Diabetic Retinopathy Study' [ETDRS] charts for right and left eyes.

A logMAR Score is calculated using the letter row on the chart and the number of incorrect letters read by the participant.

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Best Corrected Visual Acuity - Change From Baseline (POD1 Prior to Dosing) to POD4	-0.07 logMAR score Standard Error 0.01	-0.04 logMAR score Standard Error 0.01

SECONDARY outcome

Timeframe: Baseline and Postoperative Day 8

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

The visual acuity in each eye is corrected using the pinhole method and the acuity is quantified on a logMAR (minimal angle of resolution) scale using the 'Early Treatment Diabetic Retinopathy Study' [ETDRS] charts for right and left eyes. A logMAR Score is calculated using the letter row on the chart and the number of incorrect letters read by the participant.

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Best Corrected Visual Acuity - Change From Baseline (POD1 Prior to Dosing) to POD8	-0.10 logMAR score Standard Error 0.01	-0.02 logMAR score Standard Error 0.01

SECONDARY outcome

Timeframe: Baseline and Postoperative Day 15

Population: Intent to Treat Population. Last observation carried forward (LOCF) imputation method.

The visual acuity in each eye is corrected using the pinhole method and the acuity is quantified on a logMAR (minimal angle of resolution) scale using the 'Early Treatment Diabetic Retinopathy Study' [ETDRS] charts for right and left eyes. A logMAR Score is calculated using the letter row on the chart and the number of incorrect letters read by the participant.

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Best Corrected Visual Acuity - Change From Baseline (POD1 Prior to Dosing) to POD15	-0.11 logMAR score Standard Error 0.01	-0.02 logMAR score Standard Error 0.01

SECONDARY outcome

Timeframe: First dose to Postoperative Day 15

Population: Intent to Treat Population.

Participants who do not respond to study treatment after randomization are started on anti-inflammatory medication (referred to as 'Rescue' medication). The number of subjects starting 'Rescue' medication is recorded at each study visit

Outcome measures

Measure	APP13007 0.05% BID n=181 Participants 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 Participants 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Participants Using of Anti-inflammatory 'Rescue' Medication Through End-of-Treatment	10 Participants	100 Participants

Adverse Events

APP13007 0.05% BID

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Matching Vehicle Placebo

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	APP13007 0.05% BID n=181 participants at risk 1 drop APP13007 0.05% twice daily for 14 days to study (operated) eye APP13007, 0.05%: APP13007 eye drop, 0.05%	Matching Vehicle Placebo n=197 participants at risk 1 drop matching vehicle place twice daily for 14 days to study (operated) eye Matching Vehicle Placebo for APP13007, 0.05%: Matching vehicle placebo eye drop
Eye disorders Corneal oedema	1.7% 3/181 • Number of events 3 • All-Cause Mortality data were collected from informed consent through Postoperative Day 22. Serious adverse events were collected from informed consent through Postoperative Day 22. Other (not Including Serious) adverse events were collected from the first dose through Postoperative Day 22.	5.1% 10/197 • Number of events 10 • All-Cause Mortality data were collected from informed consent through Postoperative Day 22. Serious adverse events were collected from informed consent through Postoperative Day 22. Other (not Including Serious) adverse events were collected from the first dose through Postoperative Day 22.

Additional Information

Chief Medical Officer

AimMax Therapeutics Inc.

Phone: 919-797-1146

Email: admin@aimmaxrx.com

Results disclosure agreements

• Principal investigator is a sponsor employee The Institutions and Investigators participating in this trial shall have no right to publish or present the results of this study without the prior written consent of the Sponsor.
• Publication restrictions are in place

Restriction type: OTHER