Clinical Trial: Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional




Official Title: A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Retinoschisin (rAAV2tYF-CB-hRS1) in Patients With X-linked Retinos

Brief Summary: This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector expressing retinoschisin (rAAV2tYF-CB-hRS1) in patients with X-linked retinoschisis. Up to 27 participants will be enrolled and 3 dose levels will be evaluated in a dose escalation format.