Frequently Asked Questions

General Questions

What are the symptoms of neovascular, or wet, age-related macular degeneration?

Signs of wet AMD may include:

    • Reduced central vision in one or both eyes
    • Visual distortions, such as straight lines appearing to be bent
    • Printed words looking increasingly blurry
    • The need for brighter light to assist while reading or doing close-up work

    • Difficulty adjusting to entering a space with low light levels, like a dimly lit restaurant or theater

Who can apply for this study?

    • Are between the ages of 50 and 89 years old
    • Have an ETDRS best-corrected visual acuity (BCVA) score between 40 and 78 in the study eye
    • Have been diagnosed with subfoveal choroidal neovascularization secondary to age-related macular degeneration (AMD) in the eye previously treated with anti-VEGF
    • Are pseudophakic at least 12 weeks post-cataract surgery in the study eye

       

    • Have been responsive to anti-VEGF therapy, such as Eylea or Lucentis

What are the benefits of participating?

The information gleaned from this study may help research new treatment options in the future for people with wet age-related macular degeneration. By participating in this study, you will have access to specialists who understand wet Macular Degeneration and can help answer your questions and address concerns.

Are there any costs to participate in this study?

No, there is no cost to participate. Qualified participants do not pay for the study medication, clinic visits, or study-related medical procedures and laboratory tests. There is a small study stipend and if necessary, transportation to and from your appointments can be provided at no cost to you.

Are there any risks to participating?

As all drugs and medical procedures carry a risk of side effects, the possibility that participants may experience some discomfort or other reactions does exist. The study staff will explain these potential risks before participants decide whether to participate, and address any concerns.

Can I change my mind about participating in the study?

Participation is entirely voluntary. Even if you are already involved in the study, you can change your mind at any time for any reason.

What is being researched in the trial?

The purpose of this clinical study is to research the efficacy, safety and tolerability of an investigational gene therapy treatment for patients with wAMD. This therapy uses proprietary technology to deliver specific transgenes via adeno associated virus (AAV) vectors, directly to retinal cells, with the goal of reducing or potentially eliminating the need for repeated anti-VEGF injections.

What To Expect

Does qualifying ensure my participation in the study?

Before a potential participant decides whether to take part in the study, the study staff will review the informed consent form with them and answer any questions. Once the consent form has been signed by the participant, the screening period of the study begins. During this period, the staff will review medical history and conduct any relevant assessments. Participants who meet all of the study criteria will be invited to participate.

What will happen during the trial?

The purpose of this study is to evaluate the efficacy, safety and tolerability of an investigational gene therapy in participants with “wet” macular degeneration. You will be asked to attend an initial screening visit to determine if you’re eligible to enroll in the study. We will be evaluating 2 dose levels of the investigational gene therapy relative to an active comparator, in this case a current standard of care therapy called Eylea. If you are enrolled in this study, you will receive either the gene therapy at the outset of the study and will experience a 108-week clinical trial duration with an option to continue for an additional 3 years, or you will begin with anti-VEGF intervention and you will have the option to receive the investigational gene therapy after you have completed 60 weeks of the trial and will be followed for an additional 48 weeks with an option to continue for an additional 3 years after the 108 week timepoint. There is no cost to participate, and participants do not pay for the study medication, clinic visits, or study-related laboratory tests. Do you think you might be interested in participating?

What are the chances I receive the placebo?

There is not a placebo group in this trial. 33% of patients will be randomly assigned to a control group that will receive aflibercept, currently a standard of care for wAMD, according to the product label. At week 60, the patients randomized to the control group will have the option of receiving a single dose of gene therapy and continuing in the study for an additional 48 weeks. All study participants are invited to continue participating in this trial for an additional 3 years beyond the initial 108 week study period.  

What if I have more questions?

If you are interested in learning more, please contact us at any time. You may email us at [email protected]

What happens after I complete the trial?

After finishing the initial 108-week phase of this study, participants who receive the investigational gene therapy at the beginning of or at week 60 of the trial will be encouraged to enroll into a long-term follow-up (LTFU) study after completion of the current study. Those electing to continue treatment will sign a separate informed consent for the LTFU study at that time and will receive continued care for an additional 3 years.