AAVCAGsCD59 for the Treatment of Wet AMD

NCT03585556 | Completed Phase 1 FDA Drug

• 2 other identifiers: 81201887MDG1002HMR-1002
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Wet Age-related Macular Degeneration

Keywords

Wet Age-related Macular Degeneration; Gene therapy; Wet AMD

Outcome Measures

Primary Outcomes (1)

• Number of intravitreal anti-VEGF injections

  Measure the number of intravitreal anti VEGF injections from Month 1 through Month 12 following an intravitreal injection of AAVCAGsCD59 at Day 7

  12 months

Secondary Outcomes (2)

• Number of patients with change in vision of ≥ 15 letters

  12 months

• Number of patients with inflammation, endophthalmitis, IOP>30, retinal detachment, cataract, and systemic adverse events

  24 months

Study Arms (1)

AAVCAGsCD59 Treated Arm

EXPERIMENTAL

An anti-VEGF injection will be given at Day 0 followed by an intravitreal injection of AAVCAGsCD59 at Day 7. All eyes will then be treated with intravitreal anti-VEGF monthly as needed based on disease activity.

Drug: Intravitreal anti-VEGF; Biological: Intravitreal AAVCAGsCD59; Drug: Oral prednisolone

Interventions

Intravitreal anti-VEGF DRUG

Intravitreal injection of anti-VEGF at Day 0 then monthly as needed

Also known as: Bevacizumab (Avastin), Ranibizumab (Lucentis), Aflibercept (Eylea)

AAVCAGsCD59 Treated Arm

Intravitreal AAVCAGsCD59 BIOLOGICAL

An intravitreal injection of AAVCAGsCD59 will occur at Day 7 once

Also known as: HMR59

AAVCAGsCD59 Treated Arm

Oral prednisolone DRUG

A 7 day tapering dose of oral prednisone will be taken at Day 30 by all patients

Also known as: oral corticosteroid

AAVCAGsCD59 Treated Arm

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Men or women 50 years of age or older.
• Treatment naive Wet AMD with no evidence of subretinal fibrosis under the fovea.
• Presence of intraretinal and/or subretinal fluid on OCT.
• Best corrected visual acuity (BCVA) Snellen equivalent 20/25 to 20/400 in the study eye using ETDRS charts at a starting distance of 4m.
• Adequate pupillary dilation to permit ocular examination and testing.
• Ability and willingness to return for all scheduled visits and assessments.
• Understand and comply with the clinical protocol and provide written informed consent prior to any study-related procedure.
• All fertile men must be willing to use barrier contraception during the study.
• Women of childbearing potential must have a negative pregnancy test and agree to use effective contraception for the duration of the trial. A woman of childbearing potential is defined as any female who has had menses within the last two years or has not undergone a hysterectomy or surgical sterilization.

You may not qualify if:

• Wet AMD secondary to non-AMD etiologies.
• Subretinal hemorrhage that interferes with the ability to adequately measure visual acuity or follow retinal or subretinal fluid collection on OCT.
• Serous pigment epithelial detachment (PED) that is \>50% of the CNV lesion, \>400µm in any diameter, or presence of a RPE tear.
• Presence of polypoidal choroidal vasculopathy (PCV), retinal angiomatous proliferation (RAP), central serous retinopathy, or symptomatic vitreomacular adhesion.
• Previous macular laser photocoagulation for CNV, photodynamic therapy (PDT), ocular radiation, or subretinal surgery for CNV in the study eye.
• History of conditions in the study eye which might alter visual acuity or interfere with study testing including clinically significant macular edema, central retinal vein occlusion, macular branch retinal vein occlusion, and optic neuropathy.
• Active uncontrolled glaucoma with IOP\>30 mmHg despite treatment with glaucoma medications, cup-to-disc ratio of \>0.9, visual field defects secondary to glaucoma that involve the macula, and optic atrophy from glaucoma.
• Likely candidate for intraocular surgery (including cataract surgery) in the study eye during the clinical trial.
• Acute or chronic infection in the study eye.
• History of uveitis unrelated to eye surgery in the study eye or opposite eye requiring treatment with topical corticosteroids or systemic immunosuppression within 24 months of enrollment.
• Any contraindication to intravitreal injection.
• Use intravitreal (study eye) corticosteroids within 3 months prior to screening.
• Any of the following underlying systemic diseases:
• Unstable or severe cardiovascular disease, e.g., congestive heart failure (New York Heart Association Functional class III or IV), myocardial infarction within 6 months, ventricular tachyarrhythmias requiring ongoing treatment, unstable angina, or critical limb ischemia;
• Cerebrovascular disease within 12 months prior to Screening that impairs the patient's ability to participate in the clinical trial;

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead

Study Sites (2)

Ophthalmic Consultants of Boston

Boston, Massachusetts, 02114, United States

Location

Vitreo-Retina Associates

Worcester, Massachusetts, 01605, United States

Location

MeSH Terms

Interventions

Bevacizumab; Ranibizumab; aflibercept; Prednisolone; Adrenal Cortex Hormones

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: All patients with treatment naive, new onset wet AMD will be treated with an intravitreal anti-VEGF at Day 0 followed by intravitreal AAVCAGsCD59 at Day 7. Patients will be followed for 12 months and treated with additional anti-VEGF monthly as needed if recurrent disease is identified.

Study Record Dates

• First Submitted: July 1, 2018
• First Posted: July 13, 2018
• Study Start: September 13, 2018
• Primary Completion: January 29, 2020
• Study Completion: January 18, 2022
• Last Updated: March 30, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)