Phase 2 Clinical Trial to Evaluate OC-01 Nasal Spray in Subjects With Neurotrophic Keratopathy

A Phase 2, Multicenter, Randomized, Controlled, Double-Masked, Clinical Trial to Evaluate the Efficacy and Safety of OC-01 (Varenicline) Nasal Spray in Subjects With Neurotrophic Keratopathy (the Olympia Study)

The objective of this study is to evaluate the safety and effectiveness of OC-01 (varenicline) nasal spray as compared to placebo nasal spray for mean change from baseline in corneal fluorescein staining in subjects with Stage 1 (corneal epithelial hyperplasia/punctate keratopathy) neurotrophic keratopathy (NK) in one or both eyes.

Study Overview

• Status: Completed
• Conditions: Neurotrophic Keratopathy
• Intervention / Treatment: Drug: OC-01 (varenicline) nasal spray 1.2 mg/ml; Drug: Placebo (vehicle) nasal spray

Detailed Description

Protocol OPP-102 is a Phase 2, multicenter, randomized, controlled, double- masked study designed to evaluate the safety and efficacy of OC-01 (varenicline) nasal spray in subjects with NK. Approximately 100 subjects at least 18 years of age with a physicians' diagnosis of Stage 1 NK as defined by the Mackie Criteria and meeting all other study eligibility criteria will be randomized 1:1 and will receive OC-01 (varenicline)/ nasal spray or placebo nasal spray for 8 weeks three times daily (TID) as follows:

• OC-01 (varenicline) nasal spray, 1.2 mg/mL
• Placebo (vehicle control) nasal spray All doses will be delivered as a 50 microliter (µL) nasal spray.

• Study Type: Interventional
• Enrollment (Actual): 113
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Dothan, Alabama, United States, 36301
      • Dothan Alabama
  • California
    • Irvine, California, United States, 92697
      • Irvine, California
    • Los Angeles, California, United States, 90095
      • Los Angeles California
    • Mission Hills, California, United States, 91345
      • Mission Hills California
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Aurora Colorado
    • Littleton, Colorado, United States, 80120
      • Littleton, Colorado
    • Longmont, Colorado, United States, 80503
      • Longmont, Colorado
  • Connecticut
    • Danbury, Connecticut, United States, 06810
      • Danbury, Connecticut
  • Florida
    • Brandon, Florida, United States, 33511
      • Brandon Florida
    • Brandon, Florida, United States, 33511
      • Brandon, Florida
    • Jacksonville, Florida, United States, 32256
      • Jacksonville Florida
    • Miami, Florida, United States, 33136
      • Miami Florida
  • Georgia
    • Atlanta, Georgia, United States, 30339
      • Atlanta, Georgia
  • Illinois
    • Hoffman Estates, Illinois, United States, 60169
      • Hoffman Estates, Illinois
    • Urbana, Illinois, United States, 61801
      • Urbana Illinois
  • Kentucky
    • Edgewood, Kentucky, United States, 41017
      • Union Kentucky
    • Louisville, Kentucky, United States, 40206
      • Louisville KY
  • Maryland
    • Bowie, Maryland, United States, 20716
      • Bowie, Maryland
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Boston Massachusetts
  • Missouri
    • Kansas City, Missouri, United States, 64111
      • Kansas City Missouri
  • Nevada
    • Henderson, Nevada, United States, 89052
      • Henderson Nevada
  • New Jersey
    • Bloomfield, New Jersey, United States, 07003
      • Bloomfield, New Jersey
    • Dover, New Jersey, United States, 07801
      • Dover New Jersey
  • New York
    • Babylon, New York, United States, 11702
      • Babylon New York
    • New York, New York, United States, 10029
      • New York NY
  • Pennsylvania
    • York, Pennsylvania, United States, 17408
      • York, Pennsylvania
  • Texas
    • Houston, Texas, United States, 77030
      • Houston Texas
    • Katy, Texas, United States, 77494
      • Katy, Texas
    • San Antonio, Texas, United States, 78229
      • San Antonio Texas
  • Virginia
    • Lynchburg, Virginia, United States, 24502
      • Lynchburg Virginia
  • Washington
    • Seattle, Washington, United States, 98119
      • Seattle Washington

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. Be at least 18 years of age at Visit 1.
2. Patients with Stage 1 (corneal epithelial hyperplasia/punctate keratopathy) NK in one or both eyes, as defined by the Mackie Criteria.
3. Evidence of decreased corneal sensitivity (≤ 4 cm using the Cochet-Bonnet aesthesiometer) in at least 1 corneal quadrant.
4. Schirmer's test without anesthesia ≥3 mm/ 5 minutes in the affected eye.
5. If a female is of childbearing potential, they must: use an acceptable means of birth control (acceptable methods of contraception include hormonal - oral, implantable, injectable, or transdermal contraceptives, mechanical - spermicide in conjunction with a barrier such as a diaphragm or condom, IUD, or surgical sterilization of partner), and have a negative urine pregnancy test on Day 1.

Exclusion Criteria:

1. Have Stage 2 or Stage 3 NK affecting one or both eyes.
2. Have ocular graft versus host disease or Stevens-Johnson syndrome.
3. Have any active ocular infection (COVID-19 conjunctivitis, bacterial, viral, fungal, or protozoal) or active ocular inflammation not related to NK in the affected eye.
4. Be currently receiving autologous serum tears, amniotic membrane, cenegermin, fresh frozen plasma or cord blood derivative tears.
5. Have severe blepharitis and/or severe meibomian gland disease in the study eye.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: OC-01 (varenicline) nasal spray, 1.2 mg/mL	Drug: OC-01 (varenicline) nasal spray 1.2 mg/ml; OC-01
Placebo Comparator: Placebo (vehicle control) nasal spray	Drug: Placebo (vehicle) nasal spray; Placebo (vehicle)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Change From Baseline in Corneal Fluorescein Staining	Corneal Fluorescein Staining was measured utilizing the Modified Oxford Grading scale. Scores range from a minimum of 0 (no staining) to a maximum of 5. A higher score indicates a worse outcome. The study eye is defined as the eye with the worst staining grade (if both eyes are eligible).	8 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Change From Baseline in Visual Acuity at Week 8	Best Corrected Visual Acuity was assessed based on the number of letters read correctly on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. ETDRS letters score range from 0 to 100 letters at each assessment. A higher score in ETDRS letters indicates a better outcome. A positive change from Baseline in ETDRS letter score indicates an improvement in visual acuity.	8 weeks

Collaborators and Investigators

• Sponsor: Oyster Point Pharma, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 17, 2021
• Primary Completion (Actual): January 27, 2023
• Study Completion (Actual): April 28, 2023

Study Registration Dates

• First Submitted: June 28, 2021
• First Submitted That Met QC Criteria: July 7, 2021
• First Posted (Actual): July 12, 2021

Study Record Updates

• Last Update Posted (Actual): May 7, 2024
• Last Update Submitted That Met QC Criteria: April 11, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Cholinergic Agents; Nicotinic Agonists; Cholinergic Agonists; Varenicline
• Other Study ID Numbers: OPP-102

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No