A Clinical Study to Assess the Safety and Efficacy of an Ophthalmic Solution (SkQ1) in the Treatment of Dry Eye Syndrome (DES)

• Sponsor: Mitotech, SA (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02121301
• Collaborator: ORA, Inc. (Industry)
• Enrollment: 91
• Location: 1
• Arms: 3
• Duration (Months): 2
• Patients Per Site Per Month: 45.4

Study Details

Study Description

Brief Summary

The purpose of this study is to evaluate whether SkQ1 ophthalmic solutions are safe and effective compared to placebo for the treatment of the signs and symptoms of dry eye syndrome.

Condition or Disease	Intervention/Treatment	Phase
Keratoconjunctivitis Sicca	Drug: Low Dose 0.155µg/mL SkQ1 ophthalmic solution; Drug: High Dose 1.55µg/mL SkQ1 ophthalmic solution; Drug: Placebo (Vehicle) opthalmic solution	Phase 2

Detailed Description

Single-center, double-masked, randomized, placebo-controlled study comprising 4 visits over the course of approximately 5 weeks. The study included a 1-week placebo (SkQ1 vehicle) run-in period between Visit 1 and Visit 2, and approximately 28 days of twice daily (BID) dosing (Visit 2 - Visit 4). Qualified subjects were randomized 1:1:1 to receive either 1.55 µg/mL SkQ1 ophthalmic solution, 0.155 µg/mL SkQ1 ophthalmic solution, or placebo (vehicle of SkQ1 ophthalmic solution).

The following primary endpoints were tested:

• Corneal Fluorescein staining in the inferior region Pre-CAE at Visit 4 (Day 29), as measured by the Ora Calibra Scale in the worst eye at baseline;

• Worst dry eye symptom (ocular discomfort, dryness, or grittiness - as determined from subject diary data recorded during the 1-week run-in period between Visit 1 and Visit 2) evaluated over the seven days preceding Visit 4 (not including day of visit) during the treatment period.

Study Design

• Study Type: Interventional
• Actual Enrollment: 91 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Phase 2, Single-Center, Randomized, Double-Masked, Placebo-Controlled Clinical Study to Assess the Safety and Efficacy of SkQ1 Ophth Sol'n in the Environment and During Challenge in the Controlled Adverse Environment (CAE) Model for the Treatment of DES
• Study Start Date: Apr 1, 2014
• Actual Primary Completion Date: Jun 1, 2014
• Actual Study Completion Date: Jun 1, 2014

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Low Dose SkQ1 Drug: Low Dose 0.155µg/mL SkQ1 ophthalmic solution administered twice daily for 28 days	Drug: Low Dose 0.155µg/mL SkQ1 ophthalmic solution; eyedrops
Experimental: High Dose SkQ1 Drug: High Dose 1.55µg/mL SkQ1 ophthalmic solution administered twice daily for 28 days	Drug: High Dose 1.55µg/mL SkQ1 ophthalmic solution; eyedrops
Placebo Comparator: Placebo (vehicle) Drug: Placebo (vehicle) ophthalmic solution administered twice daily for 28 days	Drug: Placebo (Vehicle) opthalmic solution; eyedrops

Outcome Measures

Primary Outcome Measures

1. Inferior Corneal Fluorescein Staining [Day 29]

   The dry eye sign primary efficacy variable of corneal fluorescein staining of the inferior region of the eye measured on a 0-4 point scale where 0 = no staining and 4 = most staining between treatment arms at day 29

2. Worst Symptom Based on Diary Data [Day 29]

   Worst dry eye symptom (as determined from subject diary data recorded during the 1-week run-in period) evaluated over the seven days preceding Day 29 during the treatment period. The scale was on a 0-5 range where 0= no symptoms and 5= worst symptoms measured for a number of dry eye symptoms.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Be male or female of any race, at least 18 years of age

• Have provided written informed consent

• Have a reported history of dry eye syndrome

• Have a history of use or desire to use eye drops for dry eye

Exclusion Criteria:

• Have any clinically significant eye findings that require therapeutic treatment or may interfere with study parameters

• Have previously had LASIK surgery within the last 12 months

• Have used Restasis® within 30 days of Visit 1

• Be a woman who is pregnant, nursing or planning a pregnancy

• Be unwilling to submit a urine pregnancy test if of childbearing potential

• Have a known allergy and/or sensitivity to the test article or its components, including the preservative benzalkonium chloride

• Be currently enrolled in an investigational drug or device study or have used an investigational drug or device within 30 days of Visit 1

• Have a condition or be in a situation which the investigator feels may put the subject at significant risk, may confound the study results, or may interfere significantly with the subject's participation in the study

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Lawrence General Hospital	Andover	Massachusetts	United States	01841

Sponsors and Collaborators

• Mitotech, SA
• ORA, Inc.

Investigators

• Principal Investigator: Gail Torkildsen, MD, Lawrence General Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Mitotech, SA
• ClinicalTrials.gov Identifier: NCT02121301
• Other Study ID Numbers: 13-110-0006
• First Posted: Apr 23, 2014
• Last Update Posted: Nov 2, 2020
• Last Verified: Oct 1, 2020

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Mitotech, SA: Dry Eye; Dry Eye Syndrome; SkQ1; Keratoconjunctivitis; Dry Eye Disease
• Additional relevant MeSH terms: Dry Eye Syndromes; Keratoconjunctivitis; Keratoconjunctivitis Sicca; Pharmaceutical Solutions; Ophthalmic Solutions

Study Results

Participant Flow

Arm/Group Title	Low Dose SkQ1	High Dose SkQ1	Placebo (Vehicle)
Arm/Group Description	Low Dose 0.155 μg/mL SkQ1 ophthalmic solution	High Dose 1.55 μg/mL SkQ1 ophthalmic solution	Placebo (vehicle) ophthalmic solution
Period Title: Overall Study
STARTED	30	30	31
COMPLETED	29	26	30
NOT COMPLETED	1	4	1

Baseline Characteristics

Arm/Group Title	Low Dose - SkQ1	High Dose - SkQ1	Placebo (Vehicle)	Total
Arm/Group Description	Low Dose 0.155 μg/mL SkQ1 ophthalmic solution	High Dose 1.55 μg/mL SkQ1 ophthalmic solution	Placebo (vehicle) ophthalmic solution	Total of all reporting groups
Overall Participants	30	30	31	91
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%	0 0%
Between 18 and 65 years	20 66.7%	19 63.3%	10 32.3%	49 53.8%
>=65 years	10 33.3%	11 36.7%	21 67.7%	42 46.2%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	61.9 (10.25)	59.5 (11.69)	64.6 (9.76)	62 (10.68)
Sex: Female, Male (Count of Participants)
Female	21 70%	21 70%	25 80.6%	67 73.6%
Male	9 30%	9 30%	6 19.4%	24 26.4%
Region of Enrollment (participants) [Number]
United States	30 100%	30 100%	31 100%	91 100%

Outcome Measures

1. Primary Outcome

Title	Inferior Corneal Fluorescein Staining
Description	The dry eye sign primary efficacy variable of corneal fluorescein staining of the inferior region of the eye measured on a 0-4 point scale where 0 = no staining and 4 = most staining between treatment arms at day 29
Time Frame	Day 29

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Low Dose SkQ1	High Dose SkQ1	Placebo (Vehicle)
Arm/Group Description	Low Dose 0.155 μg/mL SkQ1 ophthalmic solution	High Dose 1.55 μg/mL SkQ1 ophthalmic solution	Placebo (vehicle) ophthalmic solution
Measure Participants	30	30	31
Mean (Standard Deviation) [units on a scale]	2.40 (0.675)	2.07 (0.704)	2.11 (0.771)

2. Primary Outcome

Title	Worst Symptom Based on Diary Data
Description	Worst dry eye symptom (as determined from subject diary data recorded during the 1-week run-in period) evaluated over the seven days preceding Day 29 during the treatment period. The scale was on a 0-5 range where 0= no symptoms and 5= worst symptoms measured for a number of dry eye symptoms.
Time Frame	Day 29

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Low Dose SkQ1	High Dose SkQ1	Placebo (Vehicle)
Arm/Group Description	Low Dose 0.155 μg/mL SkQ1 ophthalmic solution	High Dose 1.55 μg/mL SkQ1 ophthalmic solution	Placebo (vehicle) ophthalmic solution
Measure Participants	30	30	31
Mean (Standard Deviation) [units on a scale]	2.54 (0.993)	2.52 (0.899)	2.39 (1.063)

Adverse Events

Time Frame	From dilating drop at Day -7 through end of study (Day 29)
Arm/Group Title	Low Dose SkQ1		High Dose SkQ1		Placebo (Vehicle)
Arm/Group Description	Low Dose 0.155 µg/mL SkQ1 opthalmic solution		High Dose 1.55 µg/mL SkQ1 opthalmic solution		Placebo (vehicle) ophthalmic solution
All Cause Mortality
	Low Dose SkQ1		High Dose SkQ1		Placebo (Vehicle)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)		/ (NaN)
Serious Adverse Events
	Low Dose SkQ1		High Dose SkQ1		Placebo (Vehicle)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/30 (0%)		0/30 (0%)		0/31 (0%)
Other (Not Including Serious) Adverse Events
	Low Dose SkQ1		High Dose SkQ1		Placebo (Vehicle)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/30 (3.3%)		1/30 (3.3%)		3/31 (9.7%)
Eye disorders
discomfort	1/30 (3.3%)	1	1/30 (3.3%)	1	3/31 (9.7%)	3

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

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

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.