Gene therapy for age-related vision loss

A Phase I/II, Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of KH631 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)

This study is testing a new gene therapy for people aged 50 to 85 with age-related vision loss to see if a single injection can help improve their eyesight and reduce the need for ongoing treatments.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 42 (estimated)
• Ages: 50 Years to 85 Years
• Sex: All
• Sponsor: Chengdu Kanghong Pharmaceutical Group Co., Ltd. (Industry-sponsored)
• Locations: 1 site (Beijing)
• Trial ID: NCT05672121 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and tolerability of KH631, a gene therapy using an adeno-associated virus vector, administered via subretinal 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 ongoing anti-VEGF therapies. Participants must be aged 50 to 85 and currently receiving anti-VEGF treatment, with specific visual acuity and response criteria. The trial is conducted in a phased approach to assess both initial safety and longer-term effects.

Who should consider this trial

Good fit: Ideal candidates are men and women aged 50 to 85 diagnosed with nAMD who have previously responded to anti-VEGF treatment.

Not a fit: Patients with other causes of choroidal neovascularization or those not currently receiving anti-VEGF treatment may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly reduce the need for frequent injections and help maintain vision in patients with nAMD.

How similar studies have performed: While gene therapy approaches for retinal diseases are emerging, this specific application of KH631 for nAMD is novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

* 1.Are willing and able to sign the study written informed consent form (ICF); 2. Men and women ≥ 50 and ≤85 years of age, diagnosed with nAMD at the Screening visit; 3. Subjects must be under active anti-VEGF treatment for nAMD and received a minimum of 3 injections within 6 months prior to screening; 4. Response to anti-VEGF therapy(Response is defined as reduction in CRT≥50μm or at least 30% reduction in fluid by OCT compared to disease at the worst); 5. BCVA between ≤20/63 and ≥20/400(≤63 and ≥19 Early Treatment Diabetic Retinopathy Study \[ETDRS\] letters) for the first patient in each cohort followed by BCVA between ≤20/40 and ≥20/400(≤73 and ≥19 ETDRS letters) for the rest of the cohort; 6. Must be pseudophakic(at least 3 months after intraocular lens implantation) in the study eye; 7.Female subjects must have been postmenopausal for at least 1 year.

Exclusion Criteria:

* 1.Any other cause of CNV, including pathologic myopia, etc, or other diseases except nAMD have an influence on the test of macular or affect the central visual acuity; 2.Presence of an implant, refractive media opacity affects fundus examination or narrow pupil of the study eye; 3.Active or history of retinal detachment in the study eye; 4.Uncontrolled glaucoma or ocular hypertension; 5.Have taken the drug known to have retinal toxicity; 6.History of intraocular surgery; 7.Uncontrolled hypertension despite medication at the screening visit.

Where this trial is running

Beijing

• Beijing Tongren Hospital, Capital Medical University — Beijing, China (Recruiting)

Study contacts

• Principal investigator: Wenbin Wei, PhD — Beijing Tongren Hospital Affiliated to Capital Medical University
• Study coordinator: Qiang Zheng

Conditions Age-Related Macular Degeneration