An observational study in Leber Congenital Amaurosis (LCA), Eye Diseases and Eye Diseases, Hereditary, sponsored by MeiraGTx UK II Ltd. Completed at 2 sites in 2 countries. Open to participants aged 3 Years to 100 Years. Per ClinicalTrials.gov, last updated 2024-04-11.
Sponsored by MeiraGTx UK II Ltd · Observational
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.
32 studies on the registry are indexed under Leber Congenital Amaurosis; 4 are open to participants now.
Browse Leber Congenital Amaurosis studies →MeiraGTx UK II Ltd is the lead sponsor of 10 studies on the registry; none are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 4 (80%) have results posted.
Counted across the registry records on this site, refreshed daily.
The patient population are those with Leber Congenital Amaurosis (LCA) with gene mutation RPE65 who have participated in the OPTIRPE65 trial.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
subretinal administration of a single low dose of AAV RPE65
Biological: AAV OPTIRPE65
subretinal administration of a single intermediate dose of AAV RPE65
Biological: AAV OPTIRPE65
subretinal administration of a single highdose of AAV RPE65
Biological: AAV OPTIRPE65
comparison of different doses of AAV RPE65
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
Plan to share: Undecided
No publications or documents are linked to this record.
This study is completed, as verified in Apr 2024. You cannot join it, but the record below documents what was studied.
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MeiraGTx UK II Ltd