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

NCT02416622 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 27

Last updated 2023-06-12

Study results available
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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.

Conditions

  • X-linked Retinoschisis

Interventions

BIOLOGICAL

rAAV2tYF-CB-hRS1

adeno-associated virus vector expressing retinoschisin

Sponsors & Collaborators

  • Beacon Therapeutics

    lead INDUSTRY

Principal Investigators

  • Theresa Heah, MD · Applied Genetics Technologies Corporation

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-05-31
Primary Completion
2019-04-30
Completion
2023-05-09

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02416622 on ClinicalTrials.gov