Safety and Efficacy Study of CBT-004 in Patients With Vascularized Pinguecula

Study Overview
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Brief Summary
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STUDY DESIGN: Structure: Multicenter, randomized, double-masked, vehicle-controlled, parallel-group study. Duration of Treatment: 4 weeks of study treatment with 4 weeks follow-up observations. Control: Vehicle for CBT-004 (hereafter referred to as Vehicle). Masking: Subjects, Investigators, Study Monitors and the Independent Image Reading Center are all masked to the study medications. Dosage/Dose regimen: One drop of the assigned study medication will be administered in the study eye BID for 4 weeks. The study eye is defined as the qualified eye (i.e., the eye meeting the inclusion criterion for vascularized pinguecula). If both eyes are qualified, then the eye with the more severe vascularity grade at Visit 1 will be the study eye. If both eyes meet the criterion and have the same severity, the left eye will be the study eye. Patients with bilateral disease will administer study medication only in the study eye. The fellow eyes in all study subjects will be untreated. Randomization: Patients will be randomized in equal ratio to receive vehicle, 0.01%, and 0.024% CBT-004. The randomization will be stratified by pinguecula vascularity grade (3 or 4) derived from Visit 1 photo. Visit schedule: The study will consist of 5 scheduled visits: Screening visit (Day-7) (Visit 1) Day 1: baseline and randomization visit (Visit 2) Weeks 2: Visits 3 Week 4: Visit 4 Week 8: final follow-up and exit visit (Visit 5)

Official Title
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A Phase 2 Multicenter, Randomized, Vehicle-Controlled Study to Evaluate the Safety and Efficacy of CBT-004 Ophthalmic Emulsion in Patients With Vascularized Pinguecula

Conditions
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Pinguecula

Intervention / Treatment
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* Drug: CBT-004


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Pinguecula with a vascularity grade ≥ 3 on a 5-point (0-4) scale. Ocular bothersome questionnaire score ≥ 2 on a 5-point (0-4) scale. ≥ 18 years of age. Able to provide written informed consent and comply with study assessments for the full duration of the study. Exclusion Criteria: Uncontrolled systemic disease, in the opinion of the investigator. Active ocular disease other than pinguecula that may confound the study data, including but not limited to severe dry eye disease, pterygium, uncontrolled blepharitis, anterior membrane dystrophy, Salzmann's, glaucoma, or active ocular infection. History of ocular herpes disease, iritis/uveitis, in either eye. Any ocular surgical procedure within the last 3 months or anticipated ocular surgery during the study, in either eye. Anticipated wearing of contact lenses during any portion of the study. Patients, who wear soft contact lenses should discontinue wearing them at least 7 days prior to Day 1 visit. Patients wearing rigid gas permeable or hard contact lenses should discontinue wearing them at least 3 weeks prior to Day 1 visit. Female patients who are pregnant, nursing, or planning a pregnancy during the study. Current enrollment in an investigational drug or device study or participation in such a study within 30 days prior to entry into this study. History of myocardial infarction or stroke. Any condition or situation which, in the investigator's opinion, may put the patient at significant risk, may confound the study results, or may interfere significantly with the patient's participation in the study. Known allergy or sensitivity to the study medication(s) or its components. Current or anticipated use of topical ophthalmic medications in the study eye. Patients must have discontinued use of ophthalmic medications in the study eye for at least 2 weeks (4 weeks for Restasis® or Xiidra®) prior to Day 1 visit. Artificial tears are allowed in the study eye until 7 days prior to Visit 1 and should not be used during the treatment phase of the study.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: CBT-004<br> | Drug: CBT-004<br>* Dose response study<br>|
| Experimental: 0.01% CBT-004<br> | Drug: CBT-004<br>* Dose response study<br>|
| Experimental: 0.024% CBT-004<br> | Drug: CBT-004<br>* Dose response study<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent adverse events | Visual acuity, biomicroscopy, intraocular pressure, ophthalmoscopy | 1 month | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pinguecula Vascularity | Change of the pinguecula vascularity grade (0-4) from baseline (Day 1) at Week 4; Score 0 is the least severe while score 4 is the most severe | 1 month |