Neovascular Age-Related Macular Degeneration
Conditions
Keywords
Age-related macular degeneration, AMD, Neovascular AMD, Neovascular age-related macular degeneration
Brief summary
The goal of this study is to evaluate the safety and efficacy of LX102 gene therapy for nAMD.
Detailed description
This study will enroll subjects aged ≥ 50 years old to receive a single unilateral subretinal injection of LX102 to evaluate its safety and efficacy.
Interventions
GENETICLX102 subretinal injection
LX102: AAV-based gene therapy comprised of codon-optimized sequence encoding VEGF-trap
Sponsors
Innostellar Biotherapeutics Co.,Ltd
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Willing to sign the informed consent, and willing to attend follow-up visits. 2. Age ≥ 50 3. Diagnosis of active CNV secondary to neovascular AMD 4. BCVA ETDRS letters between 5 and 63 5. Subjects must have received a minimum of 2 injections within 6 months prior to screening 6. Demonstrated a meaningful response to anti-VEGF therapy
Exclusion criteria
1. CNV or macular edema in the study eye secondary to diseases other than nAMD 2. Retinal detachment, uveitis, uncontrolled glaucoma in the study eye, or any condition preventing visual acuity improvement 3. Acute coronary syndrome, myocardial infarction or coronary artery revascularization, CVA, TIA in the last 6 months 4. Uncontrolled hypertension defined as average SBP ≥160 mmHg or an average DBP ≥100 mmHg 5. Uncontrolled diabetes defined as HbA1c \>7.5%
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of dose-limiting toxicity (DLT) | 4 weeks | Incidence of dose-limiting toxicity (DLT) following LX102 subretinal injection at different doses |
| Incidence of adverse events (AEs) and serious adverse events (SAEs) | 52 weeks | Incidence of ocular and systemic adverse events (AEs) and serious adverse events (SAEs) following LX102 subretinal injection |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean time from LX102 administration to anti-VEGF rescue injection for the first time | 52 weeks | — |
| Mean change in BCVA from Baseline | 12 weeks, 36 weeks 52 weeks | BCVA measured by ETDRS |
| Mean number of anti-VEGF rescue injections | 52 weeks | — |
| Percentage of participants requiring anti-VEGF rescue injection | 52 weeks | — |
| Mean change in Central Subfield Thickness (CST) from Baseline | 12 weeks, 36 weeks 52 weeks | CST measured by spectral domain optical coherence tomography (SD-OCT) |
Countries
China
Outcome results
None listed