Last updated on October 2017

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


Brief description of study

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 Study Description

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.

Clinical Study Identifier: NCT01505062

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Investigational Site Number 840001
Portland, OR United States
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Investigational Site Number 250001
Paris, France
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