Usher Syndrome Type 1B (MYO7A) Gene Therapy Trial

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 purpose of this study is to learn about a new gene therapy being studied in patients with Retinitis Pigmentosa (RP) as a result of Usher Syndrome.  This is the first study that aims to treat RP due to Usher Syndrome by gene therapy.  The study investigators want to find out if UshStat is safe for use in humans.  The gene therapy is given by surgical injection underneath the retina of one eye.  The eye with worse vision will receive the gene therapy.   

Eligibility criteria:       

  • Adults 18 years and older 
  • Must have two documented mutations (changes) in the MYO7A gene that are disease-causing.  The MYO7A gene has been linked to Usher Syndrome Type 1B.  Genetic testing must also be completed within the subject's family (one or both parents or siblings) to confirm the results of the affected subject.  
  • Legal blindness as defined by peripheral (side) vision being no greater than 20 degrees.  This can be determined by visual field testing.   
  • Good general health   

Interested participants will come to the Casey Eye Institute in Portland, OR, for a screening visit that will last 2-3 days.  If the participant is eligible, the gene therapy will be given by a surgical procedure to one eye within one month of the screening visit.      

Following surgery, subjects will have eight more visits at the Casey Eye Institute.  Participation will last for at least one year with an option for long-term follow-up.  Each study visit will last about 4-8 hours and will include vision function tests, dilated eye exams, and blood draws. 

Principal Investigator:
Richard G. Weleber, MD
Casey Eye Institute
3375 SW Terwilliger Blvd.
Portland, OR  97239
eIRB# 7917

For more information