Safety and Tolerability of KH631 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration

NCT05657301 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: VAN-2201
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 7

Brief 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.

Conditions

Age-Related Macular Degeneration

Outcome Measures

Primary Outcomes (2)

• Safety (Type, Severity and Incidence of ocular and systemic AEs and SAEs)

  type, severity and incidence of ocular and systemic AEs and SAEs

  52 Weeks

• Best Corrected Visual Acuity

  Change in Best Corrected Visual Acuity

  52 Weeks

Secondary Outcomes (3)

• Safety (type, severity and incidence of ocular and systemic AEs and SAEs)

  104 Weeks

• Rescue Injections

  104 Weeks

• Best Corrected Visual Acuity

  104 Weeks

Study Arms (5)

KH631 Dose 1

EXPERIMENTAL

KH631 One-Time Intraocular Injection Dose Level 1

Drug: KH631

KH631 Dose 2

EXPERIMENTAL

KH631 One-Time Intraocular Injection Dose Level 2

Drug: KH631

KH631 Dose 3

EXPERIMENTAL

KH631 One-Time Intraocular Injection Dose Level 3

Drug: KH631

KH631 Dose 4

EXPERIMENTAL

KH631 One-Time Intraocular Injection Dose Level 4

Drug: KH631

KH631 Dose 5

EXPERIMENTAL

KH631 One-Time Intraocular Injection Dose Level 5

Drug: KH631

Interventions

KH631 DRUG

KH631: AAV vector containing a coding sequence for an anti-VEGF protein

KH631 Dose 1; KH631 Dose 2; KH631 Dose 3; KH631 Dose 4; KH631 Dose 5

Eligibility Criteria

• Age: 50 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. 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).

You may not qualify if:

• Have had any prior ocular or systemic treatment (investigational or approved) or surgery for the treatment of neovascular AMD except IVT anti-VEGF
• Retinal pigment epithelial tears or rips at screening
• Any history or presence of vitreous hemorrhage;
• Have any condition preventing visual acuity improvement;
• 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;
• History of intraocular or periocular surgery in the prior 3 months;
• Prior trabeculectomy or other filtration surgery ;
• Any use of long-acting intraocular steroids, including implants, within six months prior;

Sponsors & Collaborators

• Chengdu Origen Biotechnology Co., Ltd.: lead

Study Sites (7)

Kanghong Investigative Site

Phoenix, Arizona, 85016, United States

Location

Kanghong Investigative Site

Lemont, Illinois, 60439, United States

Location

Kanghong Investigative Site

Boston, Massachusetts, 02114, United States

Location

Kanghong Investigative Site

Reno, Nevada, 89502, United States

Location

Kanghong Investigative Site

Cherry Hill, New Jersey, 08034, United States

Location

Kanghong Investigative Site

Germantown, Tennessee, 38138, United States

Location

Kanghong Investigative Site

Dallas, Texas, 75231, United States

Location

MeSH Terms

Conditions

Macular Degeneration

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Officials

• Avner Ingerman, MD, MSc

  Vanotech Ltd.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 10, 2022
• First Posted: December 20, 2022
• Study Start: October 16, 2023
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): September 1, 2027
• Last Updated: December 31, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)