Safety and Bioactivity of AXT107 in Subjects With Neovascular Age-Related Macular Degeneration

Study Overview
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Brief Summary
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This is an open-label, dose-escalating, 48-week study assessing the safety, tolerability, bioactivity and duration of action of a single intravitreal injection of 0.1 mg, 0.25 mg, or 0.5 mg AXT107 in approximately 18 subjects (up to 6 subjects per dose) with nAMD.

Official Title
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Phase 1/2a Study of the Safety and Bioactivity of AXT107 in Subjects With Neovascular Age-Related Macular Degeneration (nAMD)

Conditions
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Neovascular Age-Related Macular Degeneration (nAMD)

Intervention / Treatment
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* Drug: AXT107 0.1 mg
* Drug: AXT107 0.25 mg
* Drug: AXT107 0.5 mg


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients 50 years of age or older diagnosed in the study eye with subfoveal choroidal neovascularization (CNV) or juxtafoveal CNV secondary to AMD Best corrected visual acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score of 65 to 10 in the study eye Willing and able to comply with clinic visits and study-related procedures Provide signed inform consent Exclusion Criteria: Macular edema or CMV secondary to any causes other than AMD in the study eye Previously-treated patients who are not responders to anti-VEGF Any condition that may preclude improvement in visual acuity in the study eye Previous vitreoretinal surgery, filtration surgery, and cataract surgery within 3 months in the study eye Note: Other inclusion/exclusion criteria apply

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
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Low Dose<br>AXT107 0.1 mg/eye | Drug: AXT107 0.1 mg<br>* Single intravitreal injection of AXT107 0.1 mg/eye (low dose)<br>|
| Experimental: Mid Dose<br>AXT107 0.25 mg/eye | Drug: AXT107 0.25 mg<br>* Single intravitreal injection of AXT107 0.25 mg/eye (mid dose)<br>|
| Experimental: High Dose<br>AXT107 0.5 mg/eye | Drug: AXT107 0.5 mg<br>* Single intravitreal injection of AXT107 0.5 mg/eye (high dose)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety as Assessed by Incidence of Adverse Events (AEs) | Incidence of ocular (study eye) and systemic AEs | Screening to Week 48 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy as Assessed by Central Retinal Thickness (CST) | Mean change in CST assessed by spectral domain optical coherence tomography | Day 0 to Week 48 | 
| Efficacy as Assessed by Early Treatment Diabetic Retinopathy Study (EDTRS) Chart | Mean change in Best Corrected Visual Acuity (BCVA) | Day 0 to Week 48 | 
| Efficacy as Assessed by Early Treatment Diabetic Retinopathy Study (EDTRS) Chart | Percentage of subjects improving ≥5, ≥10, and ≥15 letters in Best Corrected Visual Acuity (BCVA) | Day 0 to Week 48 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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dose escalation, AsclepiX, Neovascular Age-Related Macular Degeneration, nAMD, eye diseases, retinal diseases, AXT107, duration of action, macular degeneration