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

Tracking Information

• First Submitted Date: April 17, 2014
• First Posted Date: April 23, 2014
• Results First Submitted Date: October 7, 2016
• Results First Posted Date: October 14, 2020
• Last Update Posted Date: November 2, 2020
• Study Start Date: April 2014
• Actual Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 19, 2020)

• 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
• 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.

Original Primary Outcome Measures (submitted: April 22, 2014)

• Fluorescein Staining [ Time Frame: Day 29 ]
• Worst Dry Eye Symptom [ Time Frame: Day 29 ]

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures (submitted: April 22, 2014): Number of participants with Adverse events [ Time Frame: Day 29 ]

Descriptive Information

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

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
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Keratoconjunctivitis Sicca

Intervention

• Drug: Low Dose 0.155µg/mL SkQ1 ophthalmic solution
  eyedrops
• Drug: High Dose 1.55µg/mL SkQ1 ophthalmic solution
  eyedrops
• Drug: Placebo (Vehicle) opthalmic solution
  eyedrops

Study Arms

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

• 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: September 19, 2020): 91
• Original Estimated Enrollment (submitted: April 22, 2014): 90
• Actual Study Completion Date: June 2014
• Actual Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02121301
• Other Study ID Numbers: 13-110-0006
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Mitotech, SA
• Original Responsible Party: Same as current
• Current Study Sponsor: Mitotech, SA
• Original Study Sponsor: Same as current
• Collaborators: ORA, Inc.

Investigators

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

• PRS Account: Mitotech, SA
• Verification Date: October 2020