Clinical trial 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
Official title: 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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
Conditions: Age-Related Macular Degeneration
Keywords: Age-Related Macular Degeneration
Lead sponsor: Chengdu Kanghong Pharmaceutical Group Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2025-02-13
Enrollment: 9
Sex: ALL
Minimum age (years): 50
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

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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:

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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;

Primary outcomes:
measure: Safety
timeFrame: 52 Weeks
measure: Safety
timeFrame: 52 Weeks
measure: Safety
timeFrame: 24Weeks
measure: Rescue Injections
timeFrame: 24Weeks
measure: Rescue Injections
timeFrame: 52Weeks
Secondary outcomes:
measure: Safety
timeFrame: 24 Weeks
Other outcomes:
Locations:
Study officials:
References: