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

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)

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT02946879

Enrollment

Registered

2016-10-27

Start date

2016-11-30

Completion date

2023-07-31

Last updated

2024-04-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Eye Diseases, Eye Diseases, Hereditary, Leber Congenital Amaurosis (LCA), Retinal Diseases

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.

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.

Interventions

BIOLOGICALAAV OPTIRPE65

comparison of different doses of AAV RPE65

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

3 Years to 100 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
Incidence of Adverse Events related to the treatment	5 years	Safety is defined as the absence of ATIMP-related safety events

Secondary

Measure	Time frame	Description
Improvement in the retinal function	5 years	Improvements in visual function as assessed by visual assessment
Improvement in the visual function	5 years	Improvements in retinal function as assessed by visual assessment
Improvement in quality of life	5 years	Improvement in the participant's quality of life which is measurable by QoL questionnaire

Countries

United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026