Stargardt's Macular Degeneration (ABCA4) Gene Therapy Trial

A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen, Administered to Patients with Stargardt's Macular Degeneration.

The purpose of this study is to learn about a new gene therapy that may help patients with Stargardt's Macular Degeneration (SMD). This is the first study that aims to treat Stargardt's disease by gene therapy. The study investigators want to find out whether it is safe for use in humans. The gene therapy is given by a surgical injection underneath the retina of one eye.  The eye with worse vision will receive the gene therapy.   

Eligibility criteria:

  • Adults 18 years and older
  • Must have 2 documented mutations in the ABCA4 gene, which is the gene linked to Stargardt's. Genetic testing must also be completed within the subject's family (one or both parents or siblings) to confirm the affected subject's results.  
  • Vision must be no better than 20/200 in the eye that will receive the gene therapy 
  • Good general health 

This study will occur at the Casey Eye Institute in Portland, OR.  Participation in this study will last for at least one year with an option for long-term follow-up. There are 11 required study visits in the first year of the study. Each study visit will involve vision function tests, dilated eye exams, and blood draws. 

Principal Investigator: 

Richard G. Weleber, M.D.
Casey Eye Institute
3375 SW Terwilliger Blvd.
Portland, OR 97239
eIRB#: 7240

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