Clinical Trial: Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)

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.


Detailed Summary: The follow up 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 in the OPTIRPE65 trial. Safety and efficacy is being assessed by clinical examination and investigations according to a pre-defined schedule of follow up visits. The OPTIRPE65 trial is an on-going open-label, Phase I/II dose-escalation study to determine the safety and efficacy of a subretinal administration of the Advanced Therapy Investigational Medicinal Product in participants with RPE65-related retinal dystrophy.
Sponsor: MeiraGTx UK II Ltd

Current Primary Outcome: Incidence of Adverse Events related to the subretinal administration of AAV2/5-OPTIRPE65 (administered in the OPTIRPE65 clinical trial). [ Time Frame: 5 years ]

The primary research objective is to assess the longer term safety of AAV2/5-OPTIRPE65 administered to participants in the OPTIRPE65 trial, measured by the presence or absence of adverse events.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Improvement in the retinal function [ Time Frame: 5 years ]
    Improvement in the retinal function from baseline that is measurable by electroretinography
  • Improvement in the visual function [ Time Frame: 5 years ]
    Any improvement in the visual function from baseline that is sustained for at least two consecutive assessment
  • Improvement in quality of life [ Time Frame: 5 years ]
    Improvement in the participant's quality of life which is measurable by EQ-5D-5L and vision specific QoL questionnaires


Original Secondary Outcome: Same as current

Information By: MeiraGTx UK II Ltd

Dates:
Date Received: July 22, 2016
Date Started: November 2016
Date Completion: December 2023
Last Updated: February 22, 2017
Last Verified: February 2017