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Wet Age-related Macular DegenerationPhase 1NCT03585556

AAVCAGsCD59 for the Treatment of Wet AMD

A Phase 1 study testing Intravitreal anti-VEGF, Intravitreal AAVCAGsCD59 and Oral prednisolone in people with Wet Age-related Macular Degeneration, run by Janssen Research & Development, LLC at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 50 years and older
Conditions the study is for
Wet Age-related Macular Degeneration
What is being tested?
Intravitreal anti-VEGF, Intravitreal AAVCAGsCD59 and Oral prednisolone
Could I receive a placebo?
No, everyone receives an active treatment
How many people
25 participants
Where
2 sitesUnited States
Study dates
13 Sep 2018to 18 Jan 2022
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
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  • 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.

Who cannot join

  • 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.
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  • 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;
  • Dementia or neurodegenerative disease (e.g., Alzheimer's disease, Parkinson's disease) of a level that prevents adequate evaluation of the subject during the study;
  • Has an active malignancy or is currently undergoing treatment for an active malignancy at Screening, or has a history of malignancy that precludes completion of this 12-month study;
  • Immunocompromised conditions and/or need for immunosuppressive therapy
  • Any significant poorly controlled illness that would preclude study compliance and follow-up
  • Current or prior use of any medication known to be toxic to the retina or optic nerve including, but not limited, to chloroquine/hydrochloroquine, deferoxamine, phenothiazines and ethambutol
  • Previous treatment with any ocular or systemic gene transfer product
  • Received any investigational product within 120 days prior to screening
  • Any psychological, familial, sociological, geographical, or other condition that would preclude study compliance and follow-up

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Ophthalmic Consultants of Boston

    Boston, Massachusetts, 02114

  • Vitreo-Retina Associates

    Worcester, Massachusetts, 01605

Other trials in United States

About this study

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

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.

Read the 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.

Being tested
Intravitreal anti-VEGF, Intravitreal AAVCAGsCD59, Oral prednisolone
Keywords
Wet Age-related Macular Degeneration, Gene therapy, Wet AMD

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Intravitreal anti-VEGFDrug

    Also called Bevacizumab (Avastin), Ranibizumab (Lucentis), Aflibercept (Eylea)

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

  • Intravitreal AAVCAGsCD59Biological

    Also called HMR59

    An intravitreal injection of AAVCAGsCD59 will occur at Day 7 once

  • Oral prednisoloneDrug

    Also called oral corticosteroid

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

Study groups in NCT03585556
Study groupRoleWhat they receive
AAVCAGsCD59 Treated ArmExperimentalAn 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.
Receives: Drug: Intravitreal anti-VEGF; Biological: Intravitreal AAVCAGsCD59; Drug: Oral prednisolone

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: 12 months

Secondary outcomes (2)

  1. Number of patients with change in vision of ≥ 15 letters

    Evaluate the change in vision in eyes receiving intravitreal AAVCAGsCD59 in conjunction with anti VEGF for new onset wet AMD

    Measured: 12 months

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

    Evaluate SAEs associated with the use of intravitreal AAVCAGsCD59

    Measured: 24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted13 Jul 2018
  2. Study start13 Sep 2018
  3. Primary completion29 Jan 2020
  4. Study completion18 Jan 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
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.
Masking
None (open label)
Enrolment
25 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03585556
Sponsor's protocol ID
81201887MDG1002
Other IDs
HMR-1002, 81201887MDG1002
Status last verified
Mar 2025
First submitted
1 Jul 2018
Last updated
30 Mar 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Intravitreal anti-VEGF, Intravitreal AAVCAGsCD59 and Oral prednisolone in people with Wet Age-related Macular Degeneration, run by Janssen Research & Development, LLC at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 30 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of intravitreal anti-VEGF injections (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03585556.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2025.

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