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

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

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.

Study Overview

• Status: Completed
• Conditions: Keratoconjunctivitis Sicca
• Intervention / Treatment: Drug: Low Dose 0.155µg/mL SkQ1 ophthalmic solution; Drug: High Dose 1.55µg/mL SkQ1 ophthalmic solution; Drug: Placebo (Vehicle) opthalmic solution

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 Type: Interventional
• Enrollment (Actual): 91
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Andover, Massachusetts, United States, 01841
      • Lawrence General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

3

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Inferior Corneal Fluorescein Staining	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	Day 29
Worst Symptom Based on Diary Data	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.	Day 29

Collaborators and Investigators

• Sponsor: Mitotech, SA
• Collaborators: ORA, Inc.
• Investigators: Principal Investigator: Gail Torkildsen, MD, Lawrence General Hospital

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start: April 1, 2014
• Primary Completion (Actual): June 1, 2014
• Study Completion (Actual): June 1, 2014

Study Registration Dates

• First Submitted: April 17, 2014
• First Submitted That Met QC Criteria: April 22, 2014
• First Posted (Estimate): April 23, 2014

Study Record Updates

• Last Update Posted (Actual): November 2, 2020
• Last Update Submitted That Met QC Criteria: October 12, 2020
• Last Verified: October 1, 2020

More Information

Terms related to this study

• Keywords: Dry Eye; Dry Eye Syndrome; Dry Eye Disease; Keratoconjunctivitis; SkQ1
• Additional Relevant MeSH Terms: Eye Diseases; Lacrimal Apparatus Diseases; Conjunctivitis; Conjunctival Diseases; Keratitis; Corneal Diseases; Dry Eye Syndromes; Keratoconjunctivitis Sicca; Keratoconjunctivitis; Ophthalmic Solutions; Pharmaceutical Solutions
• Other Study ID Numbers: 13-110-0006

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No