Safety and Efficacy Study of LX102-C01 Treatment of Neovascular Age-Related Macular Degeneration (nAMD)

Study Overview
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Brief Summary
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The goal of this study is to evaluate the safety and efficacy of LX102-C01 treatment of nAMD. This study will enroll subjects aged ≥ 50 years old to receive a single unilateral intravitreal (IVT) injection of LX102-C01 to evaluate its safety and efficacy.

Official Title
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An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Efficacy of LX102-C01 in Patients With Neovascular Age-Related Macular Degeneration (nAMD)

Conditions
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Neovascular Age-related Macular Degeneration

Intervention / Treatment
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* Genetic: LX102-C01 Injection


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 1. Willing to sign the informed consent, and willing to attend follow-up visits. Age ≥ 50 Diagnosis of active CNV secondary to neovascular AMD The study eye must have a best-corrected E-ETDRS visual acuity letter score of less than or equal to 63. Subjects must have received a minimum of 2 injections within 6 months prior to screening. Demonstrated a meaningful response to anti-VEGF therapy Exclusion Criteria: CNV or macular edema in the study eye secondary to diseases other than nAMD Retinal detachment, uveitis, uncontrolled glaucoma in the study eye, or any condition preventing visual acuity improvement Acute coronary syndrome, myocardial infarction or coronary artery revascularization, CVA, TIA in the last 6 months Uncontrolled hypertension defined as average SBP ≥160 mmHg or an average DBP ≥100 mmHg Uncontrolled diabetes defined as HbA1c >7.5%

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: LX102-C01 Injection<br>Potential doses: 3E10 vg, 0.06 mL/eye/dose 1E11 vg, 0.06 mL/eye/dose | Genetic: LX102-C01 Injection<br>* Qualified subjects will receive a single unilateral intravitreal injection of LX102-C01 at Day 0 in the trial.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of adverse events (AEs) | Incidence of adverse events (AEs) within 52 weeks of LX102-C01 intravitreal injection at different doses | 52 weeks | 
| Incidence of serious adverse events (SAEs) | Incidence of serious adverse events (SAEs) within 52 weeks of LX102-C01 intravitreal injection at different doses | 52 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change in BCVA from Baseline | BCVA measured by ETDRS | 24 weeks, 52weeks | 
| Mean change in Central Subfield Thickness (CST) from Baseline | To evaluate the effect of LX102-C01 on CST | 24 weeks, 52 weeks | 
| Mean number of anti-VEGF injections over time | Mean number of anti-VEGF injections over time | 52 weeks | 
| Change of quality of life scores (VFQ-25) from Baseline | Changes of VFQ-25 questionaire from Baseline to 52 weeks | 52 weeks |