NCT02416622

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

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
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 27 (actual)
• Sponsor: Beacon Therapeutics · Industry
• Sex: Male
• Age: 6 Years
• Healthy volunteers: Not accepted

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 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.

Conditions

• X-linked Retinoschisis

Interventions

Type	Name	Description
BIOLOGICAL	rAAV2tYF-CB-hRS1	adeno-associated virus vector expressing retinoschisin

Timeline

• Start date: 2015-05-01
• Primary completion: 2019-04-01
• Completion: 2023-05-09
• First posted: 2015-04-15
• Last updated: 2023-06-12
• Results posted: 2020-07-01

Locations

9 sites across 1 country: United States