Gene therapy for neovascular age-related macular degeneration

A Phase I, Open-label, Multicenter, Dose-Escalating Study to Evaluate the Safety and Tolerability of KH631 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration

This study is testing a new gene therapy called KH631 to see if it can help people with neovascular age-related macular degeneration need fewer injections for their condition.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 25 (estimated)
• Ages: 50 Years to 85 Years
• Sex: All
• Sponsor: Chengdu Kanghong Pharmaceutical Group Co., Ltd. (Industry-sponsored)
• Locations: 7 sites (Phoenix, Arizona and 6 other locations)
• Trial ID: NCT05657301 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and tolerability of KH631, a gene therapy designed to inhibit VEGF, in patients with neovascular age-related macular degeneration (AMD). The study is a multicenter, open-label, dose-escalating trial that will enroll participants who have previously responded to anti-VEGF treatments. Participants will receive a single intraocular injection of KH631, with follow-up assessments conducted monthly for 24 weeks and continued monitoring for up to 104 weeks. The goal is to establish a safe and effective dose that could potentially reduce the need for regular injections.

Who should consider this trial

Good fit: Ideal candidates are males and females aged 50 to 85 with a documented response to prior anti-VEGF treatment for neovascular AMD.

Not a fit: Patients who have not previously responded to anti-VEGF treatments or have other ocular conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly reduce the treatment burden for patients with neovascular AMD by minimizing the need for frequent injections.

How similar studies have performed: Other studies have shown promise with gene therapies targeting VEGF, indicating potential for success in this novel approach.

Eligibility criteria

Inclusion Criteria:

1. Males and Females ages 50 to 85 (inclusive) with a study eye which meets the following criteria:

a. Previously received IVT treatment of anti-VEGF for neovascular AMD, with documented response to anti-VEGF therapy during the first 2 weeks of screening b. active macular CNV lesion secondary to AMD evidenced by SD-OCT c. Have a ETDRS BCVA letter score of 63 to 19 (approximately 20/63 to 20/400 Snellen equivalent) in the study eye at Screening for the first subject in each cohort (sentinel subject), followed by ETDRS BCVA letter score of 73 to 19 (approximately 20/40 to 20/400 Snellen equivalent) for the rest of the subjects each cohort; d. Pseudophakia in the study eye, with ocular media to permit high quality fundus imaging at screening and allow planned vitrectomy and subretinal injection; e. Are willing and able to sign the study written informed consent form (ICF).

Exclusion Criteria:

1. Have had any prior ocular or systemic treatment (investigational or approved) or surgery for the treatment of neovascular AMD except IVT anti-VEGF
2. Retinal pigment epithelial tears or rips at screening
3. Any history or presence of vitreous hemorrhage;
4. Have any condition preventing visual acuity improvement;
5. Have any other cause of CNV; prior pars plana vitrectomy or scleral buckling or retinal detachment surgery; macular hole, Epiretinal membrane or vitreo-macular traction; full thickness macular hole;
6. History of intraocular or periocular surgery in the prior 3 months;
7. Prior trabeculectomy or other filtration surgery ;
8. Any use of long-acting intraocular steroids, including implants, within six months prior;

Where this trial is running

Phoenix, Arizona and 6 other locations

• Kanghong Investigative Site — Phoenix, Arizona, United States (Recruiting)
• Kanghong Investigative Site — Lemont, Illinois, United States (Recruiting)
• Kanghong Investigative Site — Boston, Massachusetts, United States (Recruiting)
• Kanghong Investigative Site — Reno, Nevada, United States (Not_yet_recruiting)
• Kanghong Investigative Site — Cherry Hill, New Jersey, United States (Recruiting)
• Kanghong Investigative Site — Germantown, Tennessee, United States (Recruiting)
• Kanghong Investigative Site — Dallas, Texas, United States (Not_yet_recruiting)

Study contacts

• Study coordinator: Sponsor Clinical Contact

Conditions Age-Related Macular Degeneration