Ireland
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Active, not recruiting Phase 1

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

NCT05657301 · tracked via the Priya Life Science USA tracker
Phase
Phase 1
Started
2023-10-16
Last updated
2025-12-31

Condition(s) studied

Age-Related Macular Degeneration

Investigational drug(s) / intervention(s)

KH631 →

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

Study 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.

Eligibility

Sex
ALL
Min age
50 Years
Max age
85 Years
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 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). 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 ; 8. Any use of long-acting intraocular steroids, including implants, within six months prior;

Primary outcome measure(s)

  • Safety (Type, Severity and Incidence of ocular and systemic AEs and SAEs) — 52 Weeks
    type, severity and incidence of ocular and systemic AEs and SAEs
  • Best Corrected Visual Acuity — 52 Weeks
    Change in Best Corrected Visual Acuity

Trial sites (7)

FacilityCityRegionStatus
Kanghong Investigative Site Phoenix Arizona
Kanghong Investigative Site Lemont Illinois
Kanghong Investigative Site Boston Massachusetts
Kanghong Investigative Site Reno Nevada
Kanghong Investigative Site Cherry Hill New Jersey
Kanghong Investigative Site Germantown Tennessee
Kanghong Investigative Site Dallas Texas
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05657301 on ClinicalTrials.gov ↗ ← All trials in the USA