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Active, not recruitingNCT06825858Updated Jan 8, 2026

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

A Phase 1 interventional study of KH658 in Age-Related Macular Degeneration, sponsored by Chengdu Origen Biotechnology Co., Ltd.. Active, not recruiting at 3 sites in United States. Open to participants aged 50 Years to 85 Years. Per ClinicalTrials.gov, last updated 2026-01-08.

Sponsored by Chengdu Origen Biotechnology Co., Ltd. · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
9
Allocation
Not applicable
Ages
50 Years to 85 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Age-Related Macular Degeneration

Keywords

  • Age-Related Macular Degeneration
03

In context

Macular Degeneration

1,474 studies on the registry are indexed under Macular Degeneration; 206 are open to participants now.

This study's planned enrollment of 9 is below the median of 51 across 985 interventional studies indexed under Macular Degeneration.

Browse Macular Degeneration studies →

Lead sponsor

Chengdu Origen Biotechnology Co., Ltd. is the lead sponsor of 4 studies on the registry; 2 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
50 Years to 85 Years
Sexes eligible
All
Accepts healthy volunteers
No

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 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

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 (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;

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
9 participants (estimated)

Study arms

  • Experimental
    KH658 Dose 1

    KH658 One-Time Suprachroidal Space delivery Dose Level 1

    Drug: KH658

  • Experimental
    KH658 Dose 2

    KH658 One-Time Suprachroidal Space delivery Dose Level 2

    Drug: KH658

  • Experimental
    KH658 Dose 3

    KH658 One-Time Suprachroidal Space delivery Dose Level 3

    Drug: KH658

Interventions

  • DrugKH658

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

06

What researchers measure

Primary outcomes

  1. Safety

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

    Time frame: 52 Weeks

  2. Safety

    Change in Best Corrected Visual Acuity

    Time frame: 52 Weeks

  3. Safety

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

    Time frame: 24Weeks

  4. Rescue Injections

    Mean Number of Rescue Injections

    Time frame: 24Weeks

  5. Rescue Injections

    Mean Number of Rescue Injections

    Time frame: 52Weeks

Secondary outcomes

  1. Safety

    Change in Best Corrected Visual Acuity

    Time frame: 24 Weeks

07

Study locations

3 sites
  • Kanghong Investigative Site
    Phoenix, Arizona 85016, United States
  • Kanghong Investigative Site
    Lemont, Illinois 60439, United States
  • Kanghong Investigative Site
    Memphis, Tennessee 38138, United States
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 8, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06825858
Lead sponsor
Chengdu Origen Biotechnology Co., Ltd.
Responsible party
Sponsor
First posted
Feb 13, 2025
Start date
Apr 7, 2025
Primary completion
Jan 2027 (estimated)
Completion
Mar 2027 (estimated)
Last update
Jan 8, 2026

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Jan 2026. You cannot join it, but the record below documents what was studied.

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