Trial Outcomes & Findings for Safety and Efficacy of TOP1630 for Dry Eye Syndrome (NCT NCT03088605)

NCT ID: NCT03088605

Last Updated: 2024-02-14

Results Overview

Visual Acuity will be measured using the EDTRS chart to assess changes from baseline

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 69 participants
• Primary outcome timeframe: Part 1: 12 days time frame; Part 2: 35 days time frame
• Results posted on: 2024-02-14

Participant Flow

Participant milestones

Measure	Active TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Overall Study STARTED	37	32
Overall Study COMPLETED	36	32
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Measure	Active TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Overall Study Withdrawal by Subject	1	0

Baseline Characteristics

Safety and Efficacy of TOP1630 for Dry Eye Syndrome

Baseline characteristics by cohort

Measure	Active n=31 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=30 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Total n=61 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	17 Participants n=93 Participants	16 Participants n=4 Participants	33 Participants n=27 Participants
Age, Categorical >=65 years	14 Participants n=93 Participants	14 Participants n=4 Participants	28 Participants n=27 Participants
Age, Continuous	62.5 years STANDARD_DEVIATION 11.42 • n=93 Participants	65.3 years STANDARD_DEVIATION 11.92 • n=4 Participants	63.9 years STANDARD_DEVIATION 11.67 • n=27 Participants
Sex: Female, Male Female	20 Participants n=93 Participants	16 Participants n=4 Participants	36 Participants n=27 Participants
Sex: Female, Male Male	11 Participants n=93 Participants	14 Participants n=4 Participants	25 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) White	31 Participants n=93 Participants	28 Participants n=4 Participants	59 Participants n=27 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	2 Participants n=4 Participants	2 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Region of Enrollment United States	31 participants n=93 Participants	30 participants n=4 Participants	61 participants n=27 Participants

PRIMARY outcome

Timeframe: Part 1: 12 days time frame; Part 2: 35 days time frame

Population: Number analyzed represents the sum of numbers analyzed Part 1 and Part 2

Visual Acuity will be measured using the EDTRS chart to assess changes from baseline

Outcome measures

Measure	Active n=37 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=32 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Visual Acuity Part 1	0.05 logMAR Standard Deviation 0.143	-0.09 logMAR Standard Deviation 0.156
Visual Acuity Part 2	0.017 logMAR Standard Deviation 0.1163	0.075 logMAR Standard Deviation 0.1242

PRIMARY outcome

Timeframe: Part 1: 12 days time frame; Part 2: 35 days time frame

Slit lamp biomicroscopy exams will be performed to assess any changes from baseline; outcome measure is number of patients with no abnormalities

Outcome measures

Measure	Active n=37 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=32 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Slit-lamp Biomicroscopy Part 1	6 participants	2 participants
Slit-lamp Biomicroscopy Part 2	31 participants	30 participants

PRIMARY outcome

Timeframe: Part 1: 12 days time frame

The comfort of the eye drop will be performed to assess changes from baseline. Drop Comfort Assessment Scale; 0-10; 0 being very comfortable and 10 being very uncomfortable

Outcome measures

Measure	Active n=6 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=2 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Drop Comfort Assessment	0.71 score on a scale Standard Deviation 0.846	1.38 score on a scale Standard Deviation 0.495

PRIMARY outcome

Timeframe: Part 1: 12 days time frame; Part 2: 35 days time frame

Population: Number analyzed comes from Part 1 and Part 2

a non-contact tonometer will be used to perform IOP to assess changes from baseline.

Outcome measures

Measure	Active n=37 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=32 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Intraocular Pressure Part 1	12.3 unit of measure "mmHg" Standard Deviation 1.03	15.5 unit of measure "mmHg" Standard Deviation 0.71
Intraocular Pressure Part 2	12.4 unit of measure "mmHg" Standard Deviation 1.75	12.8 unit of measure "mmHg" Standard Deviation 2.54

PRIMARY outcome

Timeframe: Part 1: 12 days time frame; Part 2: 35 days time frame

Population: Number analyzed comes from Part 2

The aesthesiometer will be used to perform corneal sensitivity to assess changes from baseline. Corneal Sensitivity Scale. Scale of 0-6

Outcome measures

Measure	Active n=31 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=30 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Corneal Sensitivity	59.8 unit of measure "mm" Standard Deviation 0.91	59.4 unit of measure "mm" Standard Deviation 1.74

PRIMARY outcome

Timeframe: Part 2: 35 days time frame

Non-Contact undilated fundoscopy exam will be performed to assess changes from baseline; outcome measure is number of patients with no abnormalities

Outcome measures

Measure	Active n=31 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=30 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Undilated Fundoscopy	31 Participants	30 Participants

PRIMARY outcome

Timeframe: Part 1: 12 days time frame; Part 2: 35 days time frame

Population: Number analyzed comes from Part 1 and Part 2

Changes in vital signs is performed to assess changes from baseline

Outcome measures

Measure	Active n=37 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=32 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Vital Signs - Pulse Part 1	64.3 beats per minute Standard Deviation 9.09	75.0 beats per minute Standard Deviation 4.24
Vital Signs - Pulse Part 2	68.8 beats per minute Standard Deviation 10.55	70.5 beats per minute Standard Deviation 15.66

PRIMARY outcome

Timeframe: Part 1: 12 days time frame; Part 2: 35 days time frame

Population: Number analyzed comes from Part 1 and Part 2

Changes in vital signs is performed to assess changes from baseline

Outcome measures

Measure	Active n=37 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=32 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Vital Signs - O2 Saturation Part 1	95.8 percentage of 02 saturation Standard Deviation 1.94	97.0 percentage of 02 saturation Standard Deviation 1.41
Vital Signs - O2 Saturation Part 2	96.3 percentage of 02 saturation Standard Deviation 1.75	95.7 percentage of 02 saturation Standard Deviation 1.66

PRIMARY outcome

Timeframe: Part 1: 12 days time frame; Part 2: 35 days time frame

Population: Number analyzed comes from Part 1 and Part 2

Changes in vital signs is performed to assess changes from baseline

Outcome measures

Measure	Active n=31 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=30 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Vital Signs - Systolic Blood Pressure Part 1	128.0 mmHg Standard Deviation 29.32	148.5 mmHg Standard Deviation 3.54
Vital Signs - Systolic Blood Pressure Part 2	120.9 mmHg Standard Deviation 15.97	128.2 mmHg Standard Deviation 17.10

PRIMARY outcome

Timeframe: Part 1: 12 days time frame; Part 2: 35 days time frame

Population: Number analyzed comes from Part 1 and Part 2

Changes in vital signs is performed to assess changes from baseline

Outcome measures

Measure	Active n=31 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=30 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Vital Signs - Diastolic Blood Pressure Part 1	72.7 mmHg Standard Deviation 8.69	89.5 mmHg Standard Deviation 9.19
Vital Signs - Diastolic Blood Pressure Part 2	71.2 mmHg Standard Deviation 9.53	72.0 mmHg Standard Deviation 9.39

SECONDARY outcome

Timeframe: Part 2: 35 days time frame

Ocular discomfort Scale severity assessment (0-4 scale where 0 = none and 4 = constant)

Outcome measures

Measure	Active n=31 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=30 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Ocular Discomfort	3.5 score on a scale Standard Deviation 0.78	3.9 score on a scale Standard Deviation 0.31

SECONDARY outcome

Timeframe: Part 2: 35 days time frame

Dry eye syndrome symptom assessment Scale (grittiness) (0-5 scale where 0 = none and 5 = worst)

Outcome measures

Measure	Active n=31 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=30 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Dry Eye Symptoms	1.7 score on a scale Standard Deviation 1.42	2.8 score on a scale Standard Deviation 1.38

SECONDARY outcome

Timeframe: Part 2: 35 days time frame

Dry eye syndrome staining Score assessments (total lissamine green staining score, 0-20 where 0 = no staining)

Outcome measures

Measure	Active n=31 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=30 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Dry Eye Signs	5.88 score on a scale Standard Deviation 2.37	6.60 score on a scale Standard Deviation 2.55

SECONDARY outcome

Timeframe: Part 2: 35 days time frame

Tear film break up time measured (in seconds) after instillation of sodium fluorescein solution

Outcome measures

Measure	Active n=31 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=30 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Tear Film Break up Time	1.629 Seconds Standard Deviation 0.85	1.480 Seconds Standard Deviation 0.39

SECONDARY outcome

Timeframe: Part 2: 35 days time frame

Measurement of Schirmer test strips (mm length of moistened area after 5 minutes)

Outcome measures

Measure	Active n=31 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=30 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Schirmer's Test	6.2 mm in 5 minutes Standard Deviation 4.19	6.7 mm in 5 minutes Standard Deviation 4.94

SECONDARY outcome

Timeframe: Assessed daily between visit 3b (day 27) to visit 4b (day 35)

Daily symptom assessment using diary cards - outcome for worst symptom, ie symptom with highest severity score at baseline for each patient; calculated using the daily average between visit 3b to visit 4b Ora Calibra Ocular Discomfort & 4-Symptom Questionnaire (0-5 scale where 0 = none and 5 = worst)

Outcome measures

Measure	Active n=31 Participants TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=30 Participants Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Daily Symptom Assessment	2.33 score on a scale Standard Deviation 0.832	2.69 score on a scale Standard Deviation 0.648

Adverse Events

Active

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Active n=37 participants at risk TOP1630 Ophthalmic Solution TOP1630 Ophthalmic Solution: Bilateral ocular drug administration	Placebo n=32 participants at risk Placebo (Vehicle) Ophthalmic Solution Placebo to TOP1630 Ophthalmic Solution: Bilateral ocular drug administration
Eye disorders Visual acuity reduced	2.7% 1/37 • Number of events 1 • 4 months	3.1% 1/32 • Number of events 1 • 4 months
Eye disorders Eye Discharge	0.00% 0/37 • 4 months	3.1% 1/32 • Number of events 1 • 4 months
Eye disorders Vision blurred	2.7% 1/37 • Number of events 1 • 4 months	0.00% 0/32 • 4 months
Eye disorders Vitreous floaters	2.7% 1/37 • Number of events 1 • 4 months	0.00% 0/32 • 4 months
Eye disorders Ocular itching	2.7% 1/37 • Number of events 1 • 4 months	0.00% 0/32 • 4 months
General disorders Instillation site pain	2.7% 1/37 • Number of events 1 • 4 months	6.2% 2/32 • Number of events 2 • 4 months
General disorders Instillation site discomfort	0.00% 0/37 • 4 months	3.1% 1/32 • Number of events 1 • 4 months
Injury, poisoning and procedural complications Procedural pain	2.7% 1/37 • Number of events 1 • 4 months	0.00% 0/32 • 4 months
Respiratory, thoracic and mediastinal disorders Common cold	2.7% 1/37 • Number of events 1 • 4 months	0.00% 0/32 • 4 months
Nervous system disorders Headache	2.7% 1/37 • Number of events 1 • 4 months	0.00% 0/32 • 4 months

Additional Information

Director of Clinical Trials

Topivert Pharma Ltd

Phone: +44 203 763 9469

Email: info@topivert.com

Results disclosure agreements

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