The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II \& III, and Choroideremia. This research is being done because there are no effective therapies for people with these retinal degenerations. They are genetic disorders that affect our ability to see at night, and later cause tunnel vision and loss of central vision. Retinal degenerations affect the retina, a light sensitive layer of cells in the back of the eye. Slowly over time, these cells die and cause permanent loss of vision. The implant is a small capsule that contains human retinal pigment epithelium cells. These cells have been given the ability to make CNTF and release it through the capsule membrane into the surrounding fluid. This study will look at the effect of the implant on vision loss by retinitis pigmentosa, Usher type II \& III, and Choroideremia. In this study, two different CNTF dose levels will be used: a high dose and a low dose in one eye, as well as a sham (or placebo) surgery in the other eye.
This study will involve about 16 visits over 1½ years for specific tests of the participant's vision and health. These visits may include visual exams, blood draw for laboratory testing, brief medical history and exam, and occasionally a questionnaire (survey), in addition to the visit for the surgical procedures. The primary effectiveness outcome for this study will be a visual acuity score one year after the implant surgery. There will be about 13 centers participating in this study, and up to 60 people enrolled across the US. Each participant joining the study who has completed initial screening will then be scheduled to have a brief surgical procedure performed on each eye, one of which will include a very small cell-filled implant. Follow-up visits for repeat assessments will be required regularly to determine if the implant being tested is safe and effective for use to treat RP.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
67
Low Dose NT-501 Implanted in study eye and fellow eye received sham surgery
High Dose NT-501 Implanted in study eye and fellow eye received sham surgery
Retina-Vitreous Associates Medical Group
Beverly Hills, California, United States
University of Califoria, Davis
Sacramento, California, United States
University of California, San Francisco
San Francisco, California, United States
Retina Group of Florida
Hollywood, Florida, United States
University of Florida
Jacksonville, Florida, United States
Kellogg Eye Center
Ann Arbor, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
NY University Medical Center
New York, New York, United States
Casey Eye Institue
Portland, Oregon, United States
The Hamilton Eye Institute
Memphis, Tennessee, United States
...and 1 more locations
The Primary Outcome is the Change in Best-corrected Visual Acuity (BCVA) Using the Electronic Visual Acuity (EVA) Technology at Month 12.
The primary efficacy endpoint was the proportion of patients demonstrating an improvement in best-corrected visual acuity (BCVA), defined as an increase of 10 letters or more at the 1-Year post-implant visit (pre-explant, if explant is completed). No response is defined as an improvement of \<10 letters.
Time frame: 12 months post-implant
Number of Participants With and Without Best Corrected Visual Acuity Response Over the 18-month Follow-up Period (Months 1, 3, 6, 12 and 18)
The key visits for efficacy assessments were Months 1, 3, 6, 12 and 18. At these time points, BCVA was measured 3 times and the mean outcome reported. The assessments were conducted by masked technicians. The change from baseline for BCVA was summarized by time. Response is defined as an improvement in visual acuity by an increase of 10 letters or more whereas no response is \<10 letters.
Time frame: From initial implant to 18 months post-implant
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