It takes a village
Designing a surgical gene therapy trial requires great planning regarding the invasiveness of the procedure, the standardization of as many dimensions as possible tailored to the available equipment and techniques used by the surgeons.
Sessions were conducted in each participating study site’s operating room with attention to each individual vitrectomy machine to determine the ideal parameters for utilizing the MedOne syringe. After each procedure, the investigators get together to refine techniques and share their pearls.
During the phase I trial, all patients will be treated. Phase II offers additional challenges, such as controls and masking.
“The investigators spend a great deal of time standardizing and maximizing the procedures,” Dr. Heier said. “We developed a careful procedure manual and conducted extensive wet lab training to ensure that all surgeons approached the procedure in a similar fashion.”
The trial design requires administration of ranibizumab (Lucentis, Genentech) at baseline. One week later they are evaluated to determine a response to the treatment.
Eighteen patients are being randomized to 1 of 3 doses of 6 patients each, i.e., 3x109 genome copies (GC)/eye, 1x1010 GC/eye, and 6x1010 GC/eye. Safety reviews occurred 4 weeks after the last patients in each cohort were treated.