Clinical Trial: A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients With Usher Syndrome Type 1B

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

Official Title: An Open-Label Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients With Usher Syndrome Type 1B

Brief Summary:

Primary Objective:

To evaluate the long-term safety and tolerability of UshStat® in patients with Usher syndrome type 1B

Secondary Objective:

To assess long-term safety and biological activity of UshStat®


Detailed Summary:
Sponsor: Sanofi

Current Primary Outcome: The incidence of adverse events. [ Time Frame: During the first 5 years of follow up, every six months, then on an annual basis for a subsequent 10 years ]

The number and percentage of patients with treatment emergent adverse events.


Original Primary Outcome: The incidence of adverse events. [ Time Frame: 15 years ]

The number and percentage of patients with treatment emergent adverse events.


Current Secondary Outcome:

  • Change from baseline in ocular safety assessments [ Time Frame: During the first 5 years of follow up, every six months, then on an annual basis for a subsequent 10 years ]
    Best Corrected Visual Acuity (BCVA), Slit-lamp, Funduscopy, Intraocular Pressure, Lab parameters, concomitant meds
  • Delay in retinal degeneration [ Time Frame: During the first 5 years of follow up, every six months, then on an annual basis for a subsequent 10 years ]
    Measured as change from baseline in function relative to untreated contralateral eye on: BCVA, kinetic, static perimetry, microperimetry, autofluorescence, ERG, OCT


Original Secondary Outcome: Determine delay in retinal degeneration [ Time Frame: 15 years ]

Biological Activity: To determine a delay in retinal degeneration following subretinal injection of UshStat®, through changes in function relative to the untreated contralateral eye utilising retinal analytical techniques


Information By: Sanofi

Dates:
Date Received: February 14, 2014
Date Started: September 2013
Date Completion: April 2034
Last Updated: March 15, 2017
Last Verified: March 2017