Study of UshStat in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B
The purpose of this first in man study is to examine the safety of an experimental gene transfer agent, UshStat designed to treat retinitis pigmentosa associated with Usher Syndrome Type 1B.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected UshStat, Administered to Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B|
- The incidence of adverse events [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]The number and percentage of patients with treatment emergent adverse events.
- To determine delay in retinal degeneration. [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]Changes in function relative to the contralateral eye utilizing retinal analytical techniques.
|Study Start Date:||March 2012|
|Estimated Study Completion Date:||May 2017|
|Estimated Primary Completion Date:||May 2017 (Final data collection date for primary outcome measure)|
Following screening procedures the gene transfer agent will be injected once only under one retina by an opthalmic surgeon under anesthesia. Patients will then have regular follow up visits where general health examinations, blood tests and ophthalmic examinations including best corrected visual acuity, slit lamp examination, intraocular pressure, fundoscopy, autofluorescence, Optical Coherence Tomography, perimetry and Electroretinogram will be undertaken.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01505062
|Contact: For site information, send an email with site number to||Contact-Us@sanofi.com|
|United States, Oregon|
|Investigational Site Number 840001||Recruiting|
|Portland, Oregon, United States, 97239-3098|
|Investigational Site Number 250001||Recruiting|
|Paris, France, 75012|
|Principal Investigator:||Richard Weleber, MD||Casey Eye Institute, Portland, Oregon|
|Principal Investigator:||Jose-Alain Sahel, MD, PhD||Hopital Nationale des Quinze-Vingt, Paris France|