Patient Enrollment Has Begun For A Trial Testing A Treatment For Stargardt Disease

According to an article from, the pharmaceutical company Ophthotech announced that the first of its patients are being enrolled in its Phase 2b clinical trial. This trial is meant to test the safety and effectiveness of Zimura (avacincaptad pegol) as a treatment for Stargardt disease.

Stargardt disease is an juvenile inherited form of macular degeneration. This degeneration causes steady loss of vision to the point of legal blindness. There are multiple genes that are associated with the development of the disorder. The symptoms develop before the sufferer’s twentieth birthday, and can begin as early as age six. Before vision loss, other symptoms include blurriness, impaired color vision, blind spots, wavy vision, and problems with night vision or dim lighting. As of today, there are no certified treatments for the disease; most medical advice involves suggestions to reduce the severity of symptoms and the speed of their progression. The illness has no other bodily effects beyond vision, but most people with the disease will eventually become legally blind. To learn more about this condition, click here.

Ophthotech collaborated with the nonprofit organization Foundation Fighting Blindness in order to help design the clinical trial. The trial plans to use 120 patients and data is projected to be available sometime in 2020. Zimura is also being tested in ongoing trials to treat other forms of macular degeneration. The drug is classed as a complement C5 protein inhibitor. It prevents the protein from separating into C5a and C5b, which is an essential mechanism to the cell death and degeneration that is characteristic of Stargardt disease. The drug is also planned for use in combination with anti-VEGF medications that are normally used to treat age-related forms of macular degeneration.

Over 30 sites have been selected for the trial which makes it one of the largest trials targeting the treatment of Stargardt disease so far. Zimura is not the only treatment being tested for Stargardt disease. Other clinical trials are ongoing and are testing a variety of potential approaches. These include stem cell therapy, pharmacotherapy, and gene therapy. The data looks promising so far, with researchers hoping that new treatments could halt or even reverse some the effects of the disease.

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