Update on Clinical Trials for Macular Degeneration

Scheie Eye Institute, University of Pennsylvania
Thursday, December 15, 2016
A doctor reviewing the results of medical tests with a patient.

Learn about some of the new clinical trials for the wet and dry forms of macular degeneration, including details on participation.

Research on age-related macular degeneration (AMD) is rapidly producing new candidate drugs. These are usually discovered or tested first in mice. Next, there is a phase I trial that tests for safety and dose in a small number of patients. If the drug is safe, it may proceed to a phase II trial, usually with approximately one hundred patients, to continue to test safety and begin to assess effectiveness. Finally, a large phase III trial may be conducted to assess effectiveness. If the drug is safe and effective, the FDA may approve it.

AMD patients may wish to consider participating in clinical trials. By doing this you could receive a treatment that may be more effective than currently available options, as well as help future generations of patients.

By doing this you could receive a treatment that may be more effective than currently available options, as well as help future generations of patients.

How to Search for Clinical Trials

Each clinical trial has a list of inclusion and exclusion criteria. A retina specialist can help identify appropriate clinical trials for each patient. The trials may be conducted at one or multiple locations.


Clinical Trials for Wet AMD

The information below represents a sample of the clinical trials for wet AMD.


For wet AMD, a drug called Fovista is being tested in combination with the current standard of care, which is intraocular injection of the VEGF inhibitors Eylea, Lucentis, or Avastin. VEGF stands for vascular endothelial growth factor, and when this protein is inhibited, it can block the growth of new leaky blood vessels, which can obscure vision and damage the retina.

Following injection of one of the VEGF inhibitors, Fovista is then injected. In a phase II trial, patients who received both Fovista and Lucentis had more improvement in vision than those receiving Lucentis alone. In theory, Fovista could complement the activity of VEGF inhibitors, because it targets a different blood vessel growth factor called PDGF.

However, results of a phase II trial from Regeneron showed no benefit when an anti-PDGF drug was added to an anti-VEGF drug. Similarly, the Fovista phase III trial, which enrolled more than 1000 patients and is more definitive than the Fovista phase II trial, showed no benefit for visual acuity after 12 months of treatment compared to Lucentis alone. While these results are disappointing, it remains to be seen whether Fovista, in combination with Lucentis, might be able to reduce the number of required doctor visits and injections, or result in more stable vision in the long term. It is also possible that ongoing trials with Eylea plus Fovista or Avastin plus Fovista will show more favorable results than Lucentis plus Fovista.


Abicipar is a drug that is injected into the eye to target VEGF. Its potential advantage is longer duration of action in the eye. In a phase II trial, the data suggest it may last as long as 12 weeks. A phase III trial is now underway.

Clinical Trials for Dry AMD

The information below represents a sample of the clinical trials for dry AMD.


For patients with advanced dry macular degeneration, also called geographic atrophy, there are several interesting phase III trials. Lampalizumab targets a protein called complement factor D, which is part of the immune system. Both genetic and biochemical evidence indicate that the complement system is involved in AMD. In phase II trials, Lampalizumab decreased the rate of geographic atrophy expansion in a subset of patients with certain complement gene mutations. Lampalizumab is injected into the eye.


Another complement inhibitor is Zimura. This drug inhibits a different complement protein called complement factor 5. Like Lampalizumab, it is injected into the eye.


An oral drug is Oracea, which is the antibiotic doxycycline, which has anti-inflammatory activities that may be beneficial for patients with geographic atrophy.

Lipoic Acid

Another orally administered pill just entering clinical trials is the antioxidant lipoic acid. This drug was able to protect mice against retinal degeneration in my lab, and is retina-protective in several other preclinical models. This study is funded in part by BrightFocus Foundation.


The decision to enter a clinical trial is a personal one, and should be discussed with your retina specialist. Sometimes it will result in better outcomes for trial participants, and it always helps current and future generations of AMD patients when clinical trial results guide them to the most effective treatments.


The information provided here is a public service of the BrightFocus Foundation and should not in any way substitute for personalized advice of a qualified healthcare professional; it is not intended to constitute medical advice. Please consult your physician for personalized medical advice. BrightFocus Foundation does not endorse any medical product, therapy, or resources mentioned or listed in this article. All medications and supplements should only be taken under medical supervision. Also, although we make every effort to keep the medical information on our website updated, we cannot guarantee that the posted information reflects the most up-to-date research.

These articles do not imply an endorsement of BrightFocus by the author or their institution, nor do they imply an endorsement of the institution or author by BrightFocus.

Some of the content may be adapted from other sources, which will be clearly identified within the article.

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