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 overall safety and efficacy of LX102 gene therapy for nAMD.
Detailed description
In this Phase 2, multi-center, randomized controlled study, subjects will be randomized to receive one of the two dose levels of LX102 (n=20 for each dose level), or aflibercept (n=10). Safety, tolerability, and efficacy will be evaluated for a period of approximately 1 year from baseline.
Interventions
GENETICLX102 subretinal injection
LX102: AAV-based gene therapy comprised of codon-optimized sequence encoding VEGF-trap
BIOLOGICALAflibercept intravitreal injection
Commercially available Active Comparator
Sponsors
Innostellar Biotherapeutics Co.,Ltd
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
50 Years to 89 Years
Healthy volunteers
No
Inclusion criteria
1. Willing to sign the informed consent, and willing to attend follow-up visits. 2. Age ≥ 50, and ≤ 89. 3. Diagnosis of active CNV secondary to neovascular AMD. 4. BCVA ETDRS letters between 19 and 73. 5. 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. Absence of RPE tear at Screening. 4. Acute coronary syndrome, myocardial infarction or coronary artery revascularization, CVA, TIA in the last 6 months. 5. Uncontrolled hypertension defined as average SBP ≥160 mmHg or an average DBP ≥100 mmHg. 6. Uncontrolled diabetes defined as HbA1c \>8.0%.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean change from baseline in Best Corrected Visual Acuity (BCVA) | 36 weeks | BCVA measured by ETDRS |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean change from baseline in Best Corrected Visual Acuity (BCVA) | 52 weeks | BCVA measured by ETDRS |
| Mean change in Central Subfield Thickness (CST) from Baseline | 36 weeks, 52 weeks | CST measured by spectral domain optical coherence tomography (SD-OCT) |
| Durability of LX102 treatment | 52 weeks | Mean time from LX102 administration to anti-VEGF rescue injection for the first time, percentage of participants requiring anti-VEGF rescue injection, and mean number of anti-VEGF rescue injections through 52 weeks following LX102 administration. |
| Incidence of adverse events (AEs) and serious adverse events (SAEs) | 36 weeks, 52 weeks | Incidence of ocular and systemic adverse events (AEs) and serious adverse events (SAEs) following LX102 subretinal injection |
Countries
China
Outcome results
None listed