Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa

Primary Outcome Measures:

• best corrected visual acuity [ Time Frame: 12 months ]
  assessed by E-ETDRS
  
  

Secondary Outcome Measures:

• mobility [ Time Frame: 12 months ]
  maze testing
  
  
• effect of treatment on functional vision [ Time Frame: 12 months ]
  assessed by visual quality of life evaluation
  
  
• visual fields [ Time Frame: 12 months ]
  Goldmann
  
  
• contrast sensitivity [ Time Frame: 12 months ]
  assessment of the ability to distinguish between finer and finer increments of light versus dark (contrast)
  
  
• safety of intravitreal injection of hRPC [ Time Frame: 12 months ]
  assessed by treatment emergent adverse events, immunogenicity and safety visual assessments
  
  

There is no effective treatment for RP; once photoreceptors are lost, they do not regenerate. The rate of deterioration of vision varies from person to person, with most people with RP legally blind by age 40. Preclinical studies demonstrated that transplantation of retinal progenitor cells into the eye can result in both photoreceptor replacement and significant slowing of host photoreceptor loss. Thus, the primary goal of this therapy is to preserve, and potentially improve, vision by intervening in the disease at a time when dystrophic host photoreceptors can be protected and reactivated. Based on the demonstration of acceptable safety and tolerability in a phase 1/2a study, this phase 2b study is designed as a controlled comparison of the changes in visual function and functional vision in subjects who receive a single jCell injection in comparison to a comparable sham-treated control group of subjects with RP.