Skip to main content
OpenTrials
Completed

NCT Number: NCT02946879

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Leber Congenital Amaurosis (LCA) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Leber Congenital Amaurosis Retinal Diseases

Age range

3 year–100 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Moorfields Eye Hospital NHS Foundation Trust, London, United Kingdom

Loading trial locations.

About this study

The follow up study is designed to collect data on longer-term safety and efficacy of AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Were enrolled and treated in the prior open-label, Phase I/II, dose escalation study involving intraocular administration of AAV2/5-OPTIRPE65

Exclusion criteria

  • Individuals will be excluded if they are unwilling or unable to meet with the requirements of the study.

Treatment and study plan

AAV OPTIRPE65

Biological

comparison of different doses of AAV RPE65

Primary outcomes

  1. Incidence of Adverse Events related to the treatment

    Time frame: 5 years

    Safety is defined as the absence of ATIMP-related safety events

Secondary outcomes

  1. Improvement in the retinal function

    Time frame: 5 years

    Improvements in visual function as assessed by visual assessment

  2. Improvement in the visual function

    Time frame: 5 years

    Improvements in retinal function as assessed by visual assessment

  3. Improvement in quality of life

    Time frame: 5 years

    Improvement in the participant's quality of life which is measurable by QoL questionnaire

Sponsors and collaborators

Lead sponsor

MeiraGTx UK II Ltd

Industry

Registry information

Official study 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)

Important dates

Study start
2016
Primary completion
2023
Study completion
2023
First posted
Oct 27, 2016
Registry last updated
Apr 11, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.