This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65.
The follow up study is designed to collect data on longer-term safety and efficacy of AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial.
Study Type
OBSERVATIONAL
Enrollment
15
comparison of different doses of AAV RPE65
Kellogg Eye Center, University of Michigan Health
Ann Arbor, Michigan, United States
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom
Incidence of Adverse Events related to the treatment
Safety is defined as the absence of ATIMP-related safety events
Time frame: 5 years
Improvement in the retinal function
Improvements in visual function as assessed by visual assessment
Time frame: 5 years
Improvement in the visual function
Improvements in retinal function as assessed by visual assessment
Time frame: 5 years
Improvement in quality of life
Improvement in the participant's quality of life which is measurable by QoL questionnaire
Time frame: 5 years
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