Safety and Tolerability Study of UBX1325 in Patients With Diabetic Macular Edema or Neovascular Age-Related Macular Degeneration

Study Overview
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Brief Summary
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A study to evaluate safety, tolerability, and pharmacokinetics of a single intravitreal injection of UBX1325 in patients diagnosed with diabetic macular edema (DME) or neovascular Age-Related Macular Degeneration.

Detailed Description
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This is a Phase 1, open-label, first-in-human (FIH), single-ascending dose (SAD) study consisting of approximately 4 cohorts. The total number of patients will be at least 3 per cohort plus 3 additional patients in the maximum tolerated dose (MTD) cohort in order to address the primary objective. Up to an additional 6 nAMD patients will be enrolled in the highest dose cohort. A single dose of UBX1325 will be administered intravitreally and all patients will be followed for approximately 6 months.

Official Title
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A Phase 1, Open-Label, Single Ascending Dose Study to Assess the Safety and Tolerability of a Single Intravitreal Injection of UBX1325 in Patients With Diabetic Macular Edema or Neovascular Age-Related Macular Degeneration

Conditions
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Diabetic Macular Edema, Neovascular Age-related Macular Degeneration

Intervention / Treatment
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* Drug: UBX1325


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: nAMD patients who, in the opinion of the Investigator, are inadequately responding to or failed current treatment options. Active choroidal neovascularization (CNV) associated with age-related macular degeneration as evidenced on FA and SD-OCT at Day 1, including presence of intraretinal or subretinal fluid. BCVA in the study eye (most affected) of 70 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or worse at screening and on Day 1. Patients who have the capacity to give informed consent and who are willing and able to comply with all study-related procedures and assessments. Exclusion Criteria: Any ocular/intraocular/periocular infection or inflammation in either eye in the past 4 weeks prior to screening. Subretinal hemorrhage with bleeding area ≥4 disc area in the study eye Concomitant therapy with anti-VEGF therapies (e.g., Avastin®, Lucentis®, or Eylea®) or previous use of these agents in the study eye within approximately 28 days of study enrollment Any retinovascular disease or retinal degeneration other than nAMD in the study eye. History of systemic and intraocular steroid use for 6 months prior to Day 1. The use of intravitreal nonbiodegradable steroid implants (ex. Iluvien®, Yutiq®, Retisert®) is prohibited. Significant media opacities, including cataract, which might interfere with VA, assessment of toxicity, or fundus imaging. Any uncontrolled medical condition that, in the opinion of the investigator, would preclude participation in this study.

Ages Eligible for Study
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Minimum Age: 50 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Treatment with UBX1325<br>UBX1325, single intravitreal injection, ascending dose | Drug: UBX1325<br>* Investigational drug intravitreal injection<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Ocular and systemic safety and tolerability of a single intravitreal injection of UBX1325 evaluated by the incidence of dose limiting toxicities (DLTs) and treatment emergent adverse events (TEAEs) |  | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Plasma concentration of UBX1325 following a single intravitreal injection |  | up to 24 hours post dose | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Retinal disease, Macular edema, Diabetes mellitus, Diabetic macular edema, Neovascular Age-Related Macular Degeneration