clinical trial · NCT02946879
Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
Leber Congenital Amaurosis (LCA)Eye DiseasesEye Diseases, HereditaryRetinal DiseasesAAV OPTIRPE65
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.
started
Nov 1, 2016
primary completion
Jul 1, 2023
completion
Jul 1, 2023
last updated
Apr 11, 2024
detailed description
The follow up study is designed to collect data on longer-term safety and efficacy of AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial.
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 Children With Retinal Dystrophy Owing to Defects in RPE65 (LCA2)
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol