Patient Enrollment Completed for FFB’s Stargardt Disease Natural History Study

February 03, 2015

The Foundation Fighting Blindness today announced it has met its patient enrollment goal of 250 for ProgSTAR, a natural history study for people with the vision-robbing retinal condition known as Stargardt disease. Reaching the milestone positions the study’s international team of a dozen scientists to collect and analyze vision and retinal imaging data to better understand the disease’s course and identify potential clinical-trial endpoints for approving future treatments. Investigators plan to complete the study in early 2017.

“As more emerging treatments for Stargardt disease are positioned to move into human studies, we greatly need reliable measures that can tell us whether they save vision. The clinical endpoints we hope to derive from ProgSTAR can enable us seek regulatory approval to get therapies out to the patients,” says Patricia Zilliox, Ph.D., chief drug development officer at the Foundation’s Clinical Research Institute. “Importantly, the knowledge we glean from ProgSTAR can be used to help any company or organization working to advance a therapy in a clinical trial. The study team is delighted to collaborate in the treatment development process.”

Launched in 2013, the $4.8 million ProgSTAR study includes retrospective and prospective analyses of changes in vision and retinal structure.

“By combining the patients’ historical imaging and vision data with the data we are actively collecting now, we can obtain a clearer picture of disease progression, and how it affects the retina and the patients’ eyesight,” says Hendrik Scholl, M.D., ProgSTAR’s protocol principal investigator and study director at the Wilmer Eye Institute, Johns Hopkins University School of Medicine.

Researchers will be evaluating a number of measures including changes in vision and the size of the blind spot that typically affects the macula, the center of the retina, in people with Stargardt disease. Approximately 30,000 people in the United States have the inherited condition, which is also known as juvenile macular degeneration.

“We’ve made a major commitment of money and resources to ProgSTAR, because it has the opportunity to change the landscape of Stargardt disease therapy development,” says William T. Schmidt, chief executive officer at the Foundation Fighting Blindness. “This project isn’t about the interests of one research group or one company. It’s about boosting everyone’s efforts to eradicate the condition once and for all.”