Patient Enrollment Underway for Stargardt Disease Natural History Study

August 29, 2013
ProgSTAR, the Foundation-funded natural history study for people with Stargardt disease, has officially begun with the enrollment of four patients at the Scheie Eye Institute, University of Pennsylvania. Taking place at nine clinical sites around the world, the primary goal of the two-year, $4.2-million study is to gain a better understanding of disease and vision-loss progression so that the best outcome measures can be used in forthcoming clinical trials of emerging therapies. As many as 250 patients may be enrolled in ProgSTAR.

“We are excited to get this invaluable natural history study on Stargardt disease up and running,” says Hendrik Scholl, M.D., of the Wilmer Eye Institute, Johns Hopkins University School of Medicine, and ProgSTAR’s principal investigator and study director. “The information we gather about the progression of neurodegeneration in Stargardt disease and associated vision loss will greatly increase our chances of success in forthcoming clinical trials of emerging treatments and cures.”

In many retinal conditions, including Stargardt disease, significant degeneration can occur over several years before vision loss is easily measured through an eye chart. Using a variety of imaging techniques, ProgSTAR investigators are evaluating structural changes in the retina that correlate with vision loss, but can be reliably quantified in a shorter period of time. These techniques include: optical coherence tomography, which uses near-infrared light to image the layers of the retina; autofluoresence imaging, which measures the degenerated regions underneath the retina; and microperimetry for correlating a patient’s field of vision with retinal structure

The study will combine prospective and retrospective analyses. The prospective portion, for which physicians will track the progression of disease in participants, will last at least a year, with a possible one-year extension. Physicians will also retrospectively analyze disease progression by reviewing participants’ past medical records and imaging exams. By combining retrospective and prospective patient data, the investigators will have a longer history and more informative picture of disease progression.

The study’s clinician-scientists are recruiting patients from their practices. No additional recruitment will be conducted, and ProgSTAR will not involve administration of any treatments.

“The timing of ProgSTAR is ideal with several potential therapies poised to move into human studies soon,” says Patricia Zilliox, Ph.D., chief drug development officer with the Foundation’s Clinical Research Institute. “Both researchers and drug development companies can benefit from what we learn. Ultimately, those benefits will be passed on to the patients with more FDA-approved treatments.”

The ProgSTAR team plans to publish results of the study in a peer-reviewed journal to make the new body of disease knowledge widely available to the research community and therapy developers.

Principal investigators for the study are:
  • Paul Bernstein, M.D., Ph.D., University of Utah (Salt Lake City)
  • David Birch, Ph.D., Retina Foundation of the Southwest (Dallas)
  • Gerald Fishman, M.D., The Chicago Lighthouse (Chicago)
  • Artur Cideciyan, Ph.D., University of Pennsylvania (Philadelphia)
  • Michel Michaelides, M.D., Moorfields Eye Hospital (London, United Kingdom)
  • José Sahel, M.D., Institut de la Vision (Paris, France)
  • Hendrik Scholl, M.D., Johns Hopkins University School of Medicine (Baltimore)
  • Janet Sunness, M.D., Greater Baltimore Medical Center (Baltimore)
  • Eberhart Zrenner, M.D., University of Tübingen (Tübingen, Germany)

Participating in study design are Frederick Ferris, M.D., Ph.D., and Brian Brooks, M.D., Ph.D., of the National Eye Institute, Johanna Seddon, M.D., of Tufts Medical Center, and Richard Weleber, M.D., of Oregon Health & Science University.

Srinivas Sadda, M.D., of the Doheny Eye Institute, University of Southern California, will be analyzing imaging and visual field data collected from the study centers.