Clinical Trial: Study of UshStat in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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 1

Brief Summary:

To evaluate the safety and tolerability of ascending doses of subretinal injections of UshStat in patients with Usher syndrome type 1B.

To evaluate for possible biological activity of UshStat.

Detailed Summary:

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.

At the end of the study, the patient will be invited to enter in an open-label safety study and long-term follow-up visits (at least once every six months) including ophthalmological examinations and recording of adverse events will continue for 5 years; then the Investigator will follow the patient for a subsequent 10 years at a minimum interval of once a year to monitor delayed adverse events.

Sponsor: Sanofi

Current Primary Outcome: The incidence of adverse events [ Time Frame: 48 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome: To determine delay in retinal degeneration. [ Time Frame: 48 weeks ]

Original Secondary Outcome: Same as current

Information By: Sanofi

Dates:
Date Received: January 4, 2012
Date Started: March 2012
Date Completion: April 2019
Last Updated: March 15, 2017
Last Verified: March 2017