At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 15 enrolled
Drug / intervention
AAV RPE65biological
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA)
In Brief
A Phase 2 clinical trial evaluating AAV RPE65 for Leber Congenital Amaurosis. Completed, enrolled 15 participants across 2 sites in 2 countries.
Detailed Summary
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsLeber Congenital Amaurosis
CountriesUnited Kingdom, United States
Collaborators--
Timeline
Phase 2CompletedFinished
20162017201820192020202120222023202420252026
Enrollment StartApr 2016
First PostedMay 2016
Primary CompletionDec 2018
TodayJul 2026
First PostedMay 24, 2016
Enrollment StartApr 1, 2016
Primary CompletionDec 1, 2018
TodayJul 2, 2026
Enrollment to primary: 2.7 yearsPosted 10.1 years ago
Interventions
AAV RPE65biological
Comparison of different dosages of AAV RPE65