Safety and Bioactivity of AXT107 in Subjects With Diabetic Macular Edema

Study Overview
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Brief Summary
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This study 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 Diabetic Macular Edema (DME).

Official Title
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Phase 1/2a Study of the Safety and Bioactivity of AXT-107 in Subjects With Diabetic Macular Edema (DME)

Conditions
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Diabetic Macular Edema (DME)

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 18 years of age or older with diabetic macular edema (DME) diagnosis secondary to diabetes mellitus Type 1 or 2 Best corrected visual acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score of 65 to 23 in the study eye Willing and able to comply with clinic visits and study-related procedures Provide signed inform consent Exclusion Criteria: Any signs of high risk proliferative diabetic retinopathy in the study Previously-treated patients who are not responders to anti-VEGF Panretinal laser photocoagulation within 6 months and macular laser photocoagulation with 3 months of screening in the study eye Note: Other inclusion/exclusion criteria apply.

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: 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<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<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<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 (ETDRS) Chart | Mean change in BCVA | Day 0 to Week 48 | 
| Efficacy as Assessed by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart | Percentage of subjects improving ≥5, ≥10, and ≥15 letters in 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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DME, AsclepiX, macular edema, eye diseases, retinal diseases, AXT107, diabetes, duration of action, dose escalation, diabetic retinopathy, retinal degeneration