A Phase I, Open-label, Multicenter, Dose-Escalating Study to Evaluate the Safety and Tolerability of KH658 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration

NCT06825858 | Active Not Recruiting Phase 1 FDA Drug

• 1 other identifier: VAN-2401
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 3

Brief Summary

VAN-2401 is Phase I clinical trial to assess the safety and tolerability of KH658 in subjects with neovascular AMD. KH658 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 ocular injections.

Conditions

Age-Related Macular Degeneration

Keywords

Age-Related Macular Degeneration

Outcome Measures

Primary Outcomes (5)

• Safety

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

  52 Weeks

• Safety

  Change in Best Corrected Visual Acuity

  52 Weeks

• Safety

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

  24Weeks

• Rescue Injections

  Mean Number of Rescue Injections

  24Weeks

• Rescue Injections

  Mean Number of Rescue Injections

  52Weeks

Secondary Outcomes (1)

• Safety

  24 Weeks

Study Arms (3)

KH658 Dose 1

EXPERIMENTAL

KH658 One-Time Suprachroidal Space delivery Dose Level 1

Drug: KH658

KH658 Dose 2

EXPERIMENTAL

KH658 One-Time Suprachroidal Space delivery Dose Level 2

Drug: KH658

KH658 Dose 3

EXPERIMENTAL

KH658 One-Time Suprachroidal Space delivery Dose Level 3

Drug: KH658

Interventions

KH658 DRUG

KH658: AAV vector containing a coding sequence for an anti-VEGF protein

KH658 Dose 1; KH658 Dose 2; KH658 Dose 3

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 25 in the study eye at Screening for the first subject in each cohort (sentinel subject), followed by ETDRS BCVA letter score of 73 to 25 for the rest of the subjects each cohort; d. 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 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 (SLT or MIGS are permitted); 8. corneal transplant; 9. Any use of long-acting intraocular steroids, including implants, within six months prior; 10. Uncontrolled glaucoma; 11. Intraocular implant 12. Participated as a subject in any interventional clinical trial within 1 month prior; 13. Have received any gene or cell therapy product at any time in the past (either eye or systemically); 14. Have active intraocular inflammation in either eye at Screening or a history of uveitis in either eye 15.Have active ocular or periocular infection in either eye; 16. Have presence or history of scleromalacia in either eye;

Sponsors & Collaborators

• Chengdu Origen Biotechnology Co., Ltd.: lead

Study Sites (3)

Kanghong Investigative Site

Phoenix, Arizona, 85016, United States

Location

Kanghong Investigative Site

Lemont, Illinois, 60439, United States

Location

Kanghong Investigative Site

Memphis, Tennessee, 38138, United States

Location

MeSH Terms

Conditions

Macular Degeneration

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

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: February 8, 2025
• First Posted: February 13, 2025
• Study Start: April 7, 2025
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: January 8, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)