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.
Age ≥8 years old;
Patients diagnosed with hereditary retinal degeneration caused by ABCA4 biallelic mutations through genetic testing, and without other ophthalmic genetic diseases;
The target eye must meet the following requirements: the best corrected visual acuity is 0.5 to 2.0 LogMAR (including 0.5 and 2.0 LogMAR, equivalent to the decimal visual acuity index up to 0.3);
The subject and his or her spouse agree to take effective contraceptive measures during the trial period and for at least one year after administration.
The researchers determined that the target eye currently has or has previously had other macular lesions such as retinal schisis or epiretinal membrane. Or have other eye diseases that may hinder the surgery or interfere with the interpretation of the study endpoint;
Having received drug treatment that may affect the observation of the trial within the three months prior to screening;
The target eye has undergone the following intraocular surgeries: retinal repositioning and vitrectomy;
There are known eye/visual diseases, disorders or lesions that cause or are related to vision loss, or whose related treatments or therapies are known to cause or are related to vision loss;
Single group
ZVS106e
DrugZVS106e 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.
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.
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.
Change from Baseline in OCT
Compare changes in retinal morphology and alterations in cell layers of the retina before and after drug administration.
Change from Baseline in Fundus autofluorescence (FAF)
Compare the atrophy of the RPE/ photoreceptor complex in the subjects before and after administration
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Zhongshan Ophthalmic Center, Sun Yat-sen University
Lead sponsor
Chigenovo Co., Ltd
Collaborator
