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.

Detailed Summary:

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.


Sponsor: Applied Genetic Technologies Corp

Current Primary Outcome: Number of participants experiencing adverse events [ Time Frame: From Day 0 to Month 12 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Changes in visual acuity [ Time Frame: From Day 0 to Month 12 ]
  • Changes in schisis cavity size on optical coherence tomography (OCT) [ Time Frame: From Day 0 to Month 12 ]
  • Changes in b-wave amplitude in electroretinogram (ERG) responses [ Time Frame: From Day 0 to Month 12 ]


Original Secondary Outcome: Same as current

Information By: Applied Genetic Technologies Corp

Dates:
Date Received: April 2, 2015
Date Started: May 2015
Date Completion: December 2020
Last Updated: March 7, 2017
Last Verified: March 2017