X-Linked Retinoshisis (RS1) Gene Therapy Trial
A Phase I/II Dose Escalation Safety Study of Intravitreally Injected AAV expressing RS1, Administered to Patients with X-linked Retinoschisis
The purpose of this study is to learn about a new gene therapy that may help patients with X-Linked Retinoschisis (XLRS). This is the first study that aims to treat XLRS 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 into the vitreous (a thick, gel-like transparent substance that fills the center of the eye) of one eye. The eye with worse vision will receive the gene therapy.
- Adults 18 years and older for Groups 1-3. Ages 6 years and older for Groups 4-5.
- Must have a documented mutation in the RS1 gene, which is the gene linked to XLRS.
- Must have visual acuity no better than:
- 20/80 in the eye that will receive gene therapy for Group 1
- 20/63 in the eye that will receive gene therapy for Group 2-3
- 20/50 in the eye that will receive gene therapy for Group 4-5
- Good general health
Mark Pennesi, M.D., Ph.D.
Casey Eye Institute
3375 SW Terwilliger Blvd.
Portland, OR 97239
View more information about this study on clinicaltrials.gov