NCT05657301

Safety and Tolerability of KH631 Gene Therapy in Participants With 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

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 25 (estimated)
• Sponsor: Chengdu Origen Biotechnology Co., Ltd. · Industry
• Sex: All
• Age: 50 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

VAN-2201 is Phase I clinical trial to assess the safety and tolerability of KH631 in subjects with neovascular AMD. KH631 is gene therapy designed to deliver a protein which targets and blocks VEGF via an adeno-associated viral vector. The standard of care for patients with neovascular AMD are anti-VEGF treaments, which have demonstrated improvement in vision and reduction in fluid. A one time placement of a product which inhibits VEGF has the potential to reduce the patient burden of regular intraocular injections.

Detailed description

The VAN-2201 clinical trial is a multicenter, open-label, dose-escalating clinical study. The primary objective of the study will be to establish a safe and tolerable dose range in subjects with neovascular AMD. Five dose cohorts are planned to be included in the study, with approximately five subjects per cohort. Subjects who meet the eligibility criteria (i.e. all the inclusion and no exclusion criteria) will be enrolled to receive KH631. KH631 will be delivered at the dose level according to the cohort via intraocular injection. Subjects will be seen monthly until the primary outcome measurement at 24 weeks and through week 52. The subjects will be continued to followed with regular visits until they complete the 104 week visit.

Conditions

• Age-Related Macular Degeneration

Interventions

Type	Name	Description
DRUG	KH631	KH631: AAV vector containing a coding sequence for an anti-VEGF protein

Timeline

• Start date: 2023-10-16
• Primary completion: 2026-09-01
• Completion: 2027-09-01
• First posted: 2022-12-20
• Last updated: 2025-12-31

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study