GVB-2001 Gene Therapy Via Intracameral Injection for the Treatment of Primary Open Angle Glaucoma
brief summary
This is an investigator initiated, single center, open label, non-randomized, exploratory clinical study. The purpose is to assess the safety and preliminary efficacy of gene therapy GVB-2001 (ScAAV2-dnRhoA) delivered via intracameral injection to the anterior segment of the eye for the treatment of subjects with primary open-angle glaucoma. At least 12 primary open angle glaucoma (POAG) patients with high intraocular pressure (IOP) will be divided into two experimental groups. Each experimental group will receive either a low dose or a high dose interacameral injection of GVB-2001. The optimal dose will be selected based on the trial results for future development of GVB-2001. Safety Review Committee, SRC will be set up for safety assessment of the study. The study subjects included will be 18 to 65 years of age (inclusion) and are diagnosed with primary open-angle glaucoma (POAG) for 1 year or more. Only participants who provide informed consent prior to all screening procedures will be eligible for enrollment into the trial.
detailed description
This research plans to recruit at least 12 high intraocular pressure of primary open-angle glaucoma (POAG) patients in one clinical center and divide into two groups. Six primary glaucoma (POAG) patients with elevated intraocular pressure and no vision in the target eye will be enrolled in trial group 1. They will be divided into two dose groups: low-dose group (n = 3) and high-dose group (n = 3). The safety review committee (SRC) will determine whether to enter the high- dose group after the last subject in the low-dose group has completed the Dose limited toxicity (DLT) assessment. In group 2, 6 primary open-angle (POAG) patients with normal visual acuity and high intraocular pressure will be enrolled. The patients will be divided into low-dose group (3 subjects) and high-dose group (3 subjects). The safety review committee (SRC) will determine whether to enter the low-dose group of trial group 2, and whether to enter the high-dose group of trial group 2, after the completion of the evaluation of DLT in the low-dose group of trial group 1. During the study of trial group 2, the safety review committee (SRC) will determine whether to add another optimized dose group to trial group 2, and determine the optimized therapeutic dose and the number of subjects in the optimized dose group.
official title
Proof of Concept Clinical Trial of Gene Therapy GVB-2001 Delivered Via Intracameral Injection for the Treatment of Primary Open Angle Glaucoma