To evaluate the preliminary safety of monocular and single subretinal injection of ZVS106e injection in the treatment of patients with hereditary retinal degeneration (ABCA4-IRDS) caused by ABCA4 biallelic mutations.
The main research objective: To evaluate the preliminary safety of single subretinal injection of ZVS106e injection in one eye for patients with hereditary retinal degeneration (ABCA4-IRDS) caused by biallelic mutations in ABCA4. Secondary research objective: To preliminarily explore the clinical efficacy of ZVS106e injection in the treatment of patients with hereditary retinal degeneration (ABCA4-IRDS) caused by ABCA4 biallelic mutations. The purpose of the exploratory study is to evaluate the improvement of retinal structure and visual function in non-injection eyes compared to the baseline.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
ZVS106e is A colorless, clear and transparent liquid, containing two active ingredients, ZVS106E-A and ZVS106E-B. The viral vectors used for both active ingredients are rAAV8
Evaluate the safety and tolerability of subretinal injection of ZVS106e solution
Types, severity, and incidence of adverse events (AE) and serious adverse events (SAE) in the eyes and throughout the body within 52 weeks post-treatment, including dose-limiting toxicities (DLT) during the dose escalation phase.
Time frame: 52 weeks post-treatment
Change from baseline in best-corrected visual acuity (BCVA)
Change in best-corrected visual acuity (BCVA) of the treated eye at 52 weeks compared to baseline.
Time frame: 52 weeks post-treatment
Change from Baseline in Visual function metrics
Treatment outcomes for visual function metrics include changes from baseline in LLVA, dynamic visual field, microperimetry, FST, contrast sensitivity, color vision, and mfERG; as well as changes from baseline in the NEI-VFQ-25 score reported by the participants.
Time frame: 52 weeks post-treatment
Change from Baseline in OCT
Compare changes in retinal morphology and alterations in cell layers of the retina before and after drug administration.
Time frame: 52 weeks post-treatment
Change from Baseline in multi-luminance mobility test (MLMT)
MLMT was assessed using both eyes at 1 or more of 7 levels of illumination, ranging from 400 lux (a brightly lit office) to 1 lux (a moonless summer night).
Time frame: 52 weeks post-treatment
Change from Baseline in Fundus autofluorescence (FAF)
Compare the atrophy of the RPE/ photoreceptor complex in the subjects before and after administration
Time frame: 52 weeks post-treatment
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