Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) (OPTIRPE65)

MeiraGTx

Status and phase: Completed; Phase 2; Phase 1

Conditions: Leber Congenital Amaurosis

Treatments: Biological: AAV RPE65

Study type: Interventional

Funder types: Industry

Identifiers

NCT02781480

MGT003

Details and patient eligibility

About

A clinical trial of AAV2/5 vector for patients with Defects in RPE65

Full description

A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65. ATIMP will be administered to one eye only in a single sub-retinal procedure

Enrollment: 15 patients

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Aged 3 years or older
• Early-onset severe retinal dystrophy consistent with RPE65 deficiency

Key Exclusion Criteria:

• Females who are pregnant or breastfeeding
• Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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15 participants in 3 patient groups

Low dose AAV-RPE65

Experimental group

Description:

Subretinal administration of a single low dose of range AAV-RPE65

Treatment:

Biological: AAV RPE65

Intermediate dose AAV-RPE65

Experimental group

Description:

Subretinal administration of a single intermediate dose of range AAV-RPE65

Treatment:

Biological: AAV RPE65

High dose AAV-RPE65

Experimental group

Description:

Subretinal administration of a single high dose of range AAV-RPE65

Treatment:

Biological: AAV RPE65