NCT03585556

AAVCAGsCD59 for the Treatment of Wet AMD

A Phase 1 Proof of Concept Study Evaluating Intravitreal AAVCAGsCD59 for the Treatment of Wet Age-Related Macular Degeneration (AMD)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: Janssen Research & Development, LLC · Industry
• Sex: All
• Age: 50 Years
• Healthy volunteers: Not accepted

Summary

Patients with treatment naive wet AMD will receive an intravitreal anti-VEGF injection at Day 0 followed by an intravitreal injection of AAVCAGsCD59 at Day 7. Patients will be followed monthly through Month 12 and receive an intravitreal anti-VEGF injection as needed based on an increase in central subfoveal thickness (CST) of \>50 micrometers on OCT from Day 0, new subretinal hemorrhage on clinical exam, and/or loss of 10 or more ETDRS letters from the previous month exam.

Detailed description

This is a Phase 1, multi-center, open label study to assess the efficacy and safety of two doses of the adeno-associated viral vector serotype 2 (AAVCAGsCD59) expressing sCD59 administered via intravitreal injection seven days after a single intravitreal injection of anti-VEGF. All patients considered for enrollment in this study must have treatment naive wet AMD, adequate pupillary dilation to permit a thorough ocular exam, and best corrected distance visual acuity in the study eye of 20/25 to 20/400 using the Snellen eye chart. Written informed consent will be obtained from each study patient prior to his/her participation in any study related procedures. Screening will determine patient eligibility for the study according to written inclusion and exclusion criteria, which include both general medical and AMD-specific criteria. Patients will be enrolled into the study upon verification that they fulfill all eligibility criteria and after completion of all screening assessments. This study consists of a screening and injection of anti-VEGF (Day 0), injection of AAVCAGsCD59 (Day 7), and a monthly follow-up period (Month 1 through Month 12) where enrolled patients are treated as needed with intravitreal anti-VEGF based on an increase in central subfoveal thickness of \>50 micrometers on OCT from Day 0, new subretinal hemorrhage, a decrease in \> or equal to 10 ETDRS letters from the previous exam, or as needed based on the treating clinician. The purpose of the study is to evaluate the number of anti-VEGF injections that are required after a single intravitreal injection of AAVCAGsCD59 at a dose of 3.56x10e11vg for subjects 1-22 and 1.071x10e12vg for subjects 23-25 is administered on Day 7. Anti-VEGF will be injected at Day 0 to treat the CNV per standard of care and enable the AAVCAGsCD59 adequate time (up to two weeks) to enter the ganglion cells in the retina and start producing the transgene product, sCD59. Up to twenty-five (25) patients will be enrolled at to 2 clinical sites in this study. A long-term follow-up safety exam will occur at Month 24.

Conditions

• Wet Age-related Macular Degeneration

Interventions

Type	Name	Description
DRUG	Intravitreal anti-VEGF	Intravitreal injection of anti-VEGF at Day 0 then monthly as needed
BIOLOGICAL	Intravitreal AAVCAGsCD59	An intravitreal injection of AAVCAGsCD59 will occur at Day 7 once
DRUG	Oral prednisolone	A 7 day tapering dose of oral prednisone will be taken at Day 30 by all patients

Timeline

• Start date: 2018-09-13
• Primary completion: 2020-01-29
• Completion: 2022-01-18
• First posted: 2018-07-13
• Last updated: 2025-03-30

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study