Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and
Brief Summary:
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 study is designed to collect data on longer-term safety and efficacy at 9-, 12-, 18-, 24-, 36-, 48- and 60-month time-points following AAV2/5-OPTIRPE65 administration.