Clinical Trial: Clinical Evaluation of Patients With X-linked Retinoschisis

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: Clinical Evaluation of Patients With X-linked Retinoschisis (XLRS)

Brief Summary: The purpose of this study is to evaluate subjects with X-linked retinoschisis in a clinical setting to collect data on disease progression.

Detailed Summary: The objective of the study is to evaluate subjects with XLRS in a clinical setting and gather data on disease progression. The data from this study will enhance the understanding of the natural history of this rare disease and will facilitate appropriately powered safety studies in a future gene therapy trial in humans.
Sponsor: Applied Genetic Technologies Corp

Current Primary Outcome: Disease progression in subjects with XLRS [ Time Frame: Every 6 months for 18 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Disease progression using microperimetry, a non-standard of care visual function test [ Time Frame: Every 6 months for 18 months ]
  • Disease progression using electroretinograms (ERGs), a non-standard of care visual function test [ Time Frame: Every 6 months for 18 months ]
  • Disease progression using the reading speed test, a non-standard of care visual function test [ Time Frame: Every 6 months for 18 months ]
  • Disease progression using the contrast sensitivity test, a non-standard of care visual function test [ Time Frame: Every 6 months for 18 months ]
  • Disease progression using a quality of life questionnaire [ Time Frame: Every 6 months for 18 months ]
  • Change in maculoschisis while receiving carbonic anhydrase inhibitors [ Time Frame: All study visits, Months 1, 3, 6, 12, and 18 ]
  • Change in visual function while receiving carbonic anhydrase inhibitors [ Time Frame: All study visits, Months 1, 3, 6, 12, and 18 ]


Original Secondary Outcome: Same as current

Information By: Applied Genetic Technologies Corp

Dates:
Date Received: December 8, 2014
Date Started: November 2012
Date Completion:
Last Updated: March 10, 2017
Last Verified: March 2017