Trial to Evaluate Safety and Efficacy of KPI-012 Ophthalmic Solution in Participants With PCED

Study Overview
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Brief Summary
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The primary objective of the study is to investigate the safety and efficacy of KPI-012 compared to vehicle in participants who have a documented clinical diagnosis of PCED.

Detailed Description
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Approximately 90 participants diagnosed with persistent corneal epithelial defect (PCED) will be enrolled at multiple US centers in a study to assess the safety and efficacy of KPI-012, a topical mesenchymal stem cell secretome therapy. After an initial cohort of at least 2 participants to evaluate the safety of the high strength product, participants in the second cohort will be randomized to treatment with either the product or vehicle (placebo) for 8 weeks. The percentage of healing will be compared between groups treated with product and vehicle. Total length of study participation will be approximately 34 weeks.

Official Title
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A Study to Evaluate Safety and Efficacy of KPI-012 Ophthalmic Solution in Participants With Persistent Corneal Epithelial Defect (PCED)

Conditions
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Persistent Corneal Epithelial Defect

Intervention / Treatment
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* Drug: KPI-012
* Drug: KPI-012 Vehicle


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Have PCED for at least 7 days prior due to an underlying condition such as diabetic keratopathy, herpetic eye disease, severe dry eye disease, limbal stem cell deficiency, infectious keratitis, neurotrophic keratitis, post ocular surgery, medical trauma, chemical burn, etc. PCED measurements meet study criteria. Exclusion Criteria: Any active ocular infection or any active infectious disease that could impact the PCED. Severe corneal burns in the Study Eye. Severe limbal stem cell deficiency in either eye. The circumference affected by limbal blood vessel ischemia greater than 75% of the circumference in the Study Eye. Severe blepharitis or severe meibomian gland disease. Severe eyelid abnormalities in the Study Eye, contributory to the persistence of the PCED. Evidence of corneal ulceration. Anticipated need for punctal occlusion. Use of Oxervate in the Study Eye within past 30 days. History of any surgical procedure for treatment of the study PCED. History of any other ocular surgery in the Study Eye within 90 days prior to screening. Not willing to suspend use of contact lens in the Study Eye. Any use of Botox injections to induce pharmacologic blepharoptosis in the 90 days. Expected use of systemic doxycycline. Any use of chemotherapeutic agents within 7 days prior to Study, or anticipated use during the study. History of current drug or alcohol abuse or addiction. Use of another investigational agent within 30 days. Participants who are pregnant, breastfeeding, or planning a pregnancy during the study.

Ages Eligible for Study
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Minimum Age: 18 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: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort 1, Arm 1<br>KPI-012 High Dose KPI-012 Ophthalmic Solution 3 U/mL 1 drop 4 times/day for 56 days | Drug: KPI-012<br>* KPI-012 is a secretome product composed of biologically active components secreted from human bone marrow-derived mesenchymal stem cells<br>|
| Experimental: Cohort 2, Arm 1<br>KPI-012 Low Dose KPI-012 Ophthalmic Solution 1 U/mL 1 drop 4 times/day for 56 days | Drug: KPI-012<br>* KPI-012 is a secretome product composed of biologically active components secreted from human bone marrow-derived mesenchymal stem cells<br>|
| Experimental: Cohort 2, Arm 2<br>KPI-012 High Dose KPI-012 Ophthalmic Solution 3 U/mL 1 drop 4 times/day for 56 days | Drug: KPI-012<br>* KPI-012 is a secretome product composed of biologically active components secreted from human bone marrow-derived mesenchymal stem cells<br>|
| Placebo Comparator: Cohort 2, Arm 3<br>KPI-012 Vehicle KPI-012 Ophthalmic Solution 0 U/mL 1 drop 4 times/day for 56 days | Drug: KPI-012 Vehicle<br>* KPI-012 formulation with no active drug<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Response status | Complete healing of the PCED and no corneal fluorescein staining in the area of the study lesion. | Day 56 |