Proof of Concept Clinical Trial of Gene Therapy GVB-2001 Delivered Via Intracameral Injection for the Treatment of Primary Open Angle Glaucoma
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.
• The subjects volunteered to participate in the clinical study and signed written informed consent;
• Aged 18 to 70 years old (inclusive), men and women;
• Primary open angle glaucoma (POAG) with a history of diagnosis ≥1 year;
• Participants in good general health and have no clinically significant systemic disease, as determined by medical history, physical examination, and screening laboratory evaluation.
• To comply with the requirements, willing to accept all the diagnosis and treatment plan, laboratory tests and other specified testing, etc.;
• Consent is obtained for an extended safety visit after 1 year.
⁃ Special eligibility criteria for trial group 1:
• no vision in the target eye;
• The intraocular pressure (IOP) of the target eye was ≤40mmHg and \> 21 mmHg after combined treatment with 2 or more IOP-lowering drugs.
⁃ Special inclusion criteria for trial group 2:
• The intraocular pressure (IOP) of the target intervention eye was no more than 30mmHg, and the IOP was more than 21 mmHg after receiving combination therapy of 2 or more IOP-lowering drugs;
• The Shaffer gonioscopy scores of the target intervention eyes were all greater than 3.
• The best corrected distance visual acuity of the target intervention eyes was at least 0.8 (logMAR4.9) and above.