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

X-linked Retinoschisis Clinical Trial

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 Retinoschisis

Status Completed

Clinical Trial 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.

Clinical Trial Description

This will be a non-randomized, open label, Phase 1/2 dose escalation study.

Up to 27 participants will be enrolled. Each participant will receive the study agent by intravitreal injection in one eye on a single occasion. Enrollment will begin with the lowest dose and will proceed to higher doses only after review of safety data by a Data and Safety Monitoring Committee (DSMC). Participants in the dose escalation phase will be ≥ 18 years of age. After the maximum tolerated dose is identified individuals ≥ 6 years of age will be enrolled.

Safety will be measured by the number and proportion of participants experiencing adverse events and immune response to RS1. Efficacy will be measured by evaluation of changes in visual function and schisis cavity size. ;

Related Conditions & MeSH terms

• Retinoschisis
• X-linked Retinoschisis

• NCT number: NCT02416622
• Study type: Interventional
• Source: Applied Genetic Technologies Corp
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: May 2015
• Completion date: May 9, 2023