Clinical trial NCT06888492: To Evaluate the Safety and Preliminary Efficacy of Intravitreal EXG202 Injection in Patients with Wet (neovascular) Age-related Macular Degeneration (wAMD)
Official title: An Open-label, Dose-escalation Study to Evaluate the Safety and Efficacy of Intravitreal EXG202 Injection in Patients with Wet (neovascular) Age-related Macular Degeneration (wAMD)
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: EARLY_PHASE1
Conditions: Wet Age-related Macular Degeneration (wAMD)
Wet Age-related Macular Degeneration
Keywords: wAMD
AAV
Lead sponsor: Hangzhou Jiayin Biotech Ltd
Sponsor class: INDUSTRY
First posted: 2025-03-21
Enrollment: 8
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Dose escalation
Primary purpose: TREATMENT

Brief summary: The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of intravitreal injection of EXG202 in subjects with neovascular age-related Macular Degeneration (nAMD).

Detailed description: EXG 202 is a gene therapy product developed for the treatment of neovascular age-related macular degeneration (nAMD). Neovascular AMD is the main cause of blindness among elderly individuals. The available therapies for treating nAMD require life-long intravitreal (IVT) injections every 4-12 weeks to maintain efficacy. Administration of EXG202 has the potential to treat nAMD by providing durable expression of therapeutic levels of intraocular protein and maintaining the vision of patients. EXG 202 is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with nAMD receiving anti-VEGF therapy in clinical practice.

Eligibility criteria: Inclusion Criteria:

1. Male or female, aged ≥50 years old;
2. The study eye must meet the following criteria:

   A. Diagnosed of wAMD and current active lesions; active, defined as the presence of any of the following lesions in the macular area upon OCT examination: a) intraretinal hemorrhage or fluid; b) subretinal fluid; c) subretinal hemorrhage; B. The BCVA score tested during screening using the ETDRS eye chart (see Appendix 3 for details) must be between 73-19 letters (including boundary values), (equivalent to Snellen visual acuity of 20/40 to 20/400);
3. Clinically meaningful response to standard anti-VEGF therapy within 6 months before screening or during the lead-in period of screening.
4. The subject has clear refractive media and sufficient pupil dilation at the time of screening to obtain high-quality retinal images for confirmation of diagnosis;
5. Subjects (including male subjects) have no pregnancy plans during the screening period and the entire trial period and voluntarily take effective contraceptive measures and have no sperm or egg donation plans;
6. Voluntarily participate in this clinical trial, understand the research procedures and sign the informed consent form before screening; have good compliance and are willing to abide by the research procedures.

Exclusion Criteria:

1. The study eye has any eye disease other than wAMD that may affect central vision and/or macular detection (such as macular hole, macular epiretinal membrane, etc.);
2. The study eye has a history of retinal detachment or retinal detachment during the screening period (such as rhegmatogenous retinal detachment, tractional retinal detachment, etc.);
3. The study eye has MNV caused by reasons other than wAMD (such as diabetic retinopathy, pathological myopia, retinal vein occlusion, angioid streak disease, ocular histoplasmosis, trauma, etc.), and a history of macular pathology unrelated to wAMD;
4. The study eye plans to undergo any intraocular surgery during the study period;

Primary outcomes:
measure: Safety and tolerability after EXG202 injection
timeFrame: up to 52 weeks after treatment
Secondary outcomes:
measure: To evaluate the efficacy after EXG202 injection
timeFrame: up to 52 weeks after treatment
measure: To evaluate the efficacy after EXG202 injection
timeFrame: 52 weeks following EXG202 treatment.
Other outcomes:
Locations:
city: XingTai
country: China
facility: Hebei Eye Hospital
state: Hebei
city: YiWu
country: China
facility: The Fourth Affiliated Hospital of Zhejiang University School of Medicine
state: Zhejiang
Study officials:
References: