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Active, Not Recruiting

NCT Number: NCT06825858

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Age range

50 year–85 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Kanghong Investigative Site, Phoenix, Arizona, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

-

  • 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).

Exclusion criteria

-

  • 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;

Treatment and study plan

KH658

Drug

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

Primary outcomes

  1. Safety

    Time frame: 52 Weeks

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

  2. Safety

    Time frame: 52 Weeks

    Change in Best Corrected Visual Acuity

  3. Safety

    Time frame: 24Weeks

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

  4. Rescue Injections

    Time frame: 24Weeks

    Mean Number of Rescue Injections

  5. Rescue Injections

    Time frame: 52Weeks

    Mean Number of Rescue Injections

Secondary outcomes

  1. Safety

    Time frame: 24 Weeks

    Change in Best Corrected Visual Acuity

Sponsors and collaborators

Lead sponsor

Chengdu Origen Biotechnology Co., Ltd.

Industry

Registry information

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Feb 13, 2025
Registry last updated
Jan 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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