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

MeiraGTx

Status: Completed

Conditions: Eye Diseases, Hereditary; Eye Diseases; Retinal Diseases; Leber Congenital Amaurosis (LCA)

Treatments: Biological: AAV OPTIRPE65

Study type: Observational

Funder types: Industry

Identifiers

NCT02946879

MGT004

2016-000898-20 (EudraCT Number)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 15 patients

Sex: All

Ages: 3 to 100 years old

Volunteers: No 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.

Trial design

15 participants in 3 patient groups

Low dose AAV OPTIRPE65

Description:

subretinal administration of a single low dose of AAV RPE65

Treatment:

Biological: AAV OPTIRPE65

Intermediate dose AAV OPTIRPE65

Description:

subretinal administration of a single intermediate dose of AAV RPE65

Treatment:

Biological: AAV OPTIRPE65

High dose AAV OPTIRPE65

Description:

subretinal administration of a single highdose of AAV RPE65

Treatment:

Biological: AAV OPTIRPE65