Trial Outcomes & Findings for Study of SkQ1 as Treatment for Dry-eye Syndrome (NCT NCT03764735)

NCT ID: NCT03764735

Last Updated: 2022-01-19

Results Overview

Change of Corneal Fluorescein Staining from baseline to Day 57, scale ranges from 0 to 4, where grade 0 = None, 4 = Severe

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 452 participants
• Primary outcome timeframe: From baseline to Day 57
• Results posted on: 2022-01-19

Participant Flow

Participant milestones

Measure	SkQ1 Vehicle Vehicle for SkQ1 ophthalmic solution	Low Dose - SkQ1 SkQ1 Low dose ophthalmic solution	High Dose - SkQ1 SkQ1 High Dose ophthalmic solution
Overall Study STARTED	151	150	151
Overall Study COMPLETED	147	144	147
Overall Study NOT COMPLETED	4	6	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of SkQ1 as Treatment for Dry-eye Syndrome

Baseline characteristics by cohort

Measure	SkQ1 Vehicle n=151 Participants Vehicle for SkQ1 ophthalmic solution	Low Dose - SkQ1 n=149 Participants SkQ1 Low dose ophthalmic solution	High Dose - SkQ1 n=151 Participants SkQ1 High Dose ophthalmic solution	Total n=451 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	68 Participants n=5 Participants	74 Participants n=7 Participants	87 Participants n=5 Participants	229 Participants n=4 Participants
Age, Categorical >=65 years	83 Participants n=5 Participants	75 Participants n=7 Participants	64 Participants n=5 Participants	222 Participants n=4 Participants
Age, Continuous	64.4 years STANDARD_DEVIATION 12.98 • n=5 Participants	64.4 years STANDARD_DEVIATION 12.35 • n=7 Participants	61.4 years STANDARD_DEVIATION 13.58 • n=5 Participants	63.4 years STANDARD_DEVIATION 13.03 • n=4 Participants
Sex: Female, Male Female	115 Participants n=5 Participants	99 Participants n=7 Participants	102 Participants n=5 Participants	316 Participants n=4 Participants
Sex: Female, Male Male	36 Participants n=5 Participants	50 Participants n=7 Participants	49 Participants n=5 Participants	135 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=5 Participants	7 Participants n=7 Participants	14 Participants n=5 Participants	27 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	145 Participants n=5 Participants	142 Participants n=7 Participants	137 Participants n=5 Participants	424 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	4 Participants n=7 Participants	4 Participants n=5 Participants	10 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Black or African American	23 Participants n=5 Participants	14 Participants n=7 Participants	24 Participants n=5 Participants	61 Participants n=4 Participants
Race (NIH/OMB) White	124 Participants n=5 Participants	130 Participants n=7 Participants	120 Participants n=5 Participants	374 Participants n=4 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	4 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Region of Enrollment United States	151 Participants n=5 Participants	149 Participants n=7 Participants	151 Participants n=5 Participants	451 Participants n=4 Participants
Central CFS	1.72 units on a scale STANDARD_DEVIATION 0.73 • n=5 Participants	1.67 units on a scale STANDARD_DEVIATION 0.69 • n=7 Participants	1.67 units on a scale STANDARD_DEVIATION 0.68 • n=5 Participants	1.69 units on a scale STANDARD_DEVIATION 0.70 • n=4 Participants

PRIMARY outcome

Timeframe: From baseline to Day 57

Change of Corneal Fluorescein Staining from baseline to Day 57, scale ranges from 0 to 4, where grade 0 = None, 4 = Severe

Outcome measures

Measure	SkQ1 Vehicle n=151 Participants Vehicle for SkQ1 ophthalmic solution	Low Dose - SkQ1 n=149 Participants SkQ1 Low dose ophthalmic solution	High Dose - SkQ1 n=151 Participants SkQ1 High Dose ophthalmic solution
Central Corneal Staining Change From Baseline	-0.28 score on a scale Standard Deviation 0.773	-0.3 score on a scale Standard Deviation 0.816	-0.2 score on a scale Standard Deviation 0.763

PRIMARY outcome

Timeframe: From baseline to Day 57

Change of patient-reported grittiness from baseline to Day 57, scale ranges from 0 to 5 for each symptom, where 0 = None and 5 = Worst

Outcome measures

Measure	SkQ1 Vehicle n=151 Participants Vehicle for SkQ1 ophthalmic solution	Low Dose - SkQ1 n=149 Participants SkQ1 Low dose ophthalmic solution	High Dose - SkQ1 n=151 Participants SkQ1 High Dose ophthalmic solution
Grittiness Change From Baseline	-0.42 score on a scale Standard Deviation 1.108	-0.36 score on a scale Standard Deviation 1.176	-0.43 score on a scale Standard Deviation 1.387

Adverse Events

SkQ1 Vehicle

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Low Dose - SkQ1

Serious events: 3 serious events

Other events: 4 other events

Deaths: 0 deaths

High Dose - SkQ1

Serious events: 2 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Measure	SkQ1 Vehicle n=151 participants at risk Vehicle for SkQ1 ophthalmic solution	Low Dose - SkQ1 n=149 participants at risk SkQ1 Low dose ophthalmic solution	High Dose - SkQ1 n=151 participants at risk SkQ1 High Dose ophthalmic solution
Reproductive system and breast disorders Ovarian cancer	0.00% 0/151 • From Day -7 through end of study (Day 57)	0.67% 1/149 • From Day -7 through end of study (Day 57)	0.00% 0/151 • From Day -7 through end of study (Day 57)
Vascular disorders Peripheral arterial occlusive disorder	0.00% 0/151 • From Day -7 through end of study (Day 57)	0.67% 1/149 • From Day -7 through end of study (Day 57)	0.00% 0/151 • From Day -7 through end of study (Day 57)
Infections and infestations Osteomyelitis	0.00% 0/151 • From Day -7 through end of study (Day 57)	0.00% 0/149 • From Day -7 through end of study (Day 57)	0.66% 1/151 • From Day -7 through end of study (Day 57)
Gastrointestinal disorders Colitis	0.00% 0/151 • From Day -7 through end of study (Day 57)	0.00% 0/149 • From Day -7 through end of study (Day 57)	0.66% 1/151 • From Day -7 through end of study (Day 57)
Surgical and medical procedures Procedural pain	0.66% 1/151 • From Day -7 through end of study (Day 57)	0.00% 0/149 • From Day -7 through end of study (Day 57)	0.00% 0/151 • From Day -7 through end of study (Day 57)
Nervous system disorders Migraine	0.00% 0/151 • From Day -7 through end of study (Day 57)	0.67% 1/149 • From Day -7 through end of study (Day 57)	0.00% 0/151 • From Day -7 through end of study (Day 57)

Other adverse events

Measure	SkQ1 Vehicle n=151 participants at risk Vehicle for SkQ1 ophthalmic solution	Low Dose - SkQ1 n=149 participants at risk SkQ1 Low dose ophthalmic solution	High Dose - SkQ1 n=151 participants at risk SkQ1 High Dose ophthalmic solution
General disorders Instillation site pain	1.3% 2/151 • From Day -7 through end of study (Day 57)	2.7% 4/149 • From Day -7 through end of study (Day 57)	2.0% 3/151 • From Day -7 through end of study (Day 57)
Eye disorders Visual acuity reduced	0.66% 1/151 • From Day -7 through end of study (Day 57)	0.00% 0/149 • From Day -7 through end of study (Day 57)	2.0% 3/151 • From Day -7 through end of study (Day 57)

Additional Information

Chief Operating Officer

Mitotech S.A.

Phone: +352621692785

Email: info@mitotechpharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee Authorship and manuscript composition will reflect cooperation among all parties involved in the study. Authorship will be established before writing the manuscript. The study sponsor will have the final decision regarding the manuscript and publication.
• Publication restrictions are in place

Restriction type: OTHER