Gene therapy for treating neovascular age-related macular degeneration

A Phase I/II Study to Evaluate the Tolerability, Safety and Efficacy of KH658 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD).

This study is testing a new gene therapy for people with neovascular age-related macular degeneration to see if a single injection can provide long-lasting treatment and reduce the need for frequent eye injections.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 44 (estimated)
• Ages: 50 Years to 85 Years
• Sex: All
• Sponsor: Chengdu Kanghong Pharmaceutical Group Co., Ltd. (Industry-sponsored)
• Locations: 1 site (Tianjin)
• Trial ID: NCT06458595 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and tolerability of KH658, a gene therapy using an adeno-associated virus vector, administered via suprachoroidal space injection for patients with neovascular age-related macular degeneration (nAMD). The study aims to provide a long-term, stable therapeutic protein after a single injection, potentially reducing the treatment burden associated with frequent anti-VEGF injections. Participants will be monitored for their response to the therapy and any adverse effects over the course of the trial.

Who should consider this trial

Good fit: Ideal candidates are males and females aged 50 to 85 with a history of nAMD who have previously responded to anti-VEGF treatments.

Not a fit: Patients with other causes of choroidal neovascularization or conditions that prevent visual acuity improvement in the study eye may not benefit from this therapy.

Why it matters

Potential benefit: If successful, this therapy could significantly reduce the frequency of treatments required for nAMD, helping to maintain vision for patients.

How similar studies have performed: While gene therapy for eye conditions is a novel approach, similar studies have shown promise in treating other retinal diseases, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

1. Are willing and able to sign the informed consent form (ICF);
2. Female and male aged 50 to 85 years (inclusive) with nAMD;
3. Have previously received anti-VEGF treatment for nAMD, with documented response to anti-VEGF therapy and a minimum of 2 anti-VEGF IVT injections in the study eye in the 6 months prior to screening;
4. Have a ETDRS BCVA letter score of 83 to 19 (approximately 20/25 to 20/400 Snellen equivalent) in the study eye at Screening;
5. Have a ETDRS BCVA letter score of ≥19 in the fellow eye at Screening;
6. Females must be at least 1 year postmenopausal.

Exclusion Criteria:

1. Have any other cause of CNV (such as pathological myopia, etc.) in the study eye; or non-nAMD disease in the study eye judged by the investigator that could impact macular examination or affect central vision (such as diabetic retinopathy, central retinal vein occlusion, etc.);
2. Any condition preventing visual acuity improvement in the study eye, e.g. fovea scar, fibrosis or atrophy;
3. Have a subretinal hemorrhage that is either 50% or more of the total lesion area, or blood is greater than 1.0 mm2 under the fovea in the study eye at screening;
4. Have any vitreous hemorrhage or history of vitreous hemorrhage in the study eye;
5. Presence of an implant (excluding intraocular lens), opacification of the refractive medium or miosis that affects fundus examination;
6. Have a history of or presence of retinal detachment or choroidal detachment in the study eye.

Where this trial is running

Tianjin

• Tianjin Medical University General Hospital — Tianjin, China (Recruiting)

Study contacts

• Study coordinator: Jiarong Du

Conditions Age-related Macular Degeneration