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

• ClinicalTrials.gov Identifier: NCT02121301
• Recruitment Status: Completed
• First Posted: April 23, 2014
• Results First Posted: October 14, 2020
• Last Update Posted: November 2, 2020
• Sponsor: Mitotech, SA
• Collaborator: ORA, Inc.
• Information provided by (Responsible Party): Mitotech, SA

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 (Clinical Trial)
• 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: April 2014
• Actual Primary Completion Date: June 2014
• Actual Study Completion Date: June 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 [ Time Frame: 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 [ Time Frame: 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

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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

United States, Massachusetts

Lawrence General Hospital

Andover, Massachusetts, United States, 01841

Sponsors and Collaborators

Mitotech, SA

ORA, Inc.

Investigators

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

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Petrov A, Perekhvatova N, Skulachev M, Stein L, Ousler G. SkQ1 Ophthalmic Solution for Dry Eye Treatment: Results of a Phase 2 Safety and Efficacy Clinical Study in the Environment and During Challenge in the Controlled Adverse Environment Model. Adv Ther. 2016 Jan;33(1):96-115. doi: 10.1007/s12325-015-0274-5. Epub 2016 Jan 5.

• Responsible Party: Mitotech, SA
• ClinicalTrials.gov Identifier: NCT02121301
• Other Study ID Numbers: 13-110-0006
• First Posted: April 23, 2014
• Results First Posted: October 14, 2020
• Last Update Posted: November 2, 2020
• Last Verified: October 2020

Individual Participant Data (IPD) Sharing Statement:

• 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; Lacrimal Apparatus Diseases; Eye Diseases; Conjunctivitis; Conjunctival Diseases; Keratitis; Corneal Diseases; Pharmaceutical Solutions; Ophthalmic Solutions