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

a study on X-linked Retinoschisis

Summary

for males ages 6 years and up (full criteria)
at San Francisco, California and other locations
study started
estimated completion:
Anthony Moore

Description

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.

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

Details

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.

Keywords

X-linked Retinoschisis XLRS maculoschisis retinal degeneration RS1 adeno-associated virus gene therapy AAV Retinoschisis rAAV2tYF-CB-hRS1

Eligibility

You can join if…

Open to males ages 6 years and up

include:

  1. Retinal disease consistent with a diagnosis of XLRS and documented mutations in the RS1 gene
  2. Male individual at least 18 years of age (dose escalation phase) or at least 6 years of age (maximum tolerated dose phase),
  3. Able to perform tests of visual and retinal function,
  4. Visual acuity specified for each group
  5. Not treated with CAIs currently or within 3 months prior to study enrollment,
  6. Have acceptable laboratory parameters.

You CAN'T join if...

include:

  1. Prior receipt of any AAV gene therapy product,
  2. Pre-existing eye conditions that would preclude the planned intravitreal injection or interfere with interpretation of study endpoints or complications of vector administration.

Locations

  • University of California San Francisco, Dept. of Ophthalmology accepting new patients
    San Francisco California 94143 United States
  • Casey Eye Institute, Oregon Health and Sciences University accepting new patients
    Portland Oregon 97239 United States

Lead Scientist

  • Anthony Moore
    Professor, Ophthalmology. Authored (or co-authored) 293 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Applied Genetic Technologies Corp
Links
AGTC website
ID
NCT02416622
Phase
Phase 1/2
Study Type
Interventional
Last Updated