Gene Therapy for Achromatopsia (CNGA3)

MeiraGTx

Status and phase: Completed; Phase 2; Phase 1

Conditions: Achromatopsia

Treatments: Biological: adeno-associated virus vector AAV- CNGA3

Study type: Interventional

Funder types: Industry

Identifiers

NCT03758404

MGT012

Details and patient eligibility

About

A clinical trial of adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia

Full description

CNGA3 retinal gene therapy for patients with achromatopsia

Enrollment: 11 patients

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Are aged years or over
• Have achromatopsia confirmed by a retinal specialist investigator

Exclusion criteria

• Are females who are pregnant or breastfeeding
• Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
• Have any other condition that the investigator considers makes them inappropriate for entry into the trial

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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11 participants in 3 patient groups

Low dose adeno-associated virus (AAV) CNGA3

Experimental group

Description:

Subretinal administration of a single low dose AAV CNGA3

Treatment:

Biological: adeno-associated virus vector AAV- CNGA3

Intermediate dose adeno-associated virus (AAV) CNGA3

Experimental group

Description:

Subretinal administration of a single intermediate dose AAV CNGA3

Treatment:

Biological: adeno-associated virus vector AAV- CNGA3

High dose adeno-associated virus (AAV) CNGA3

Experimental group

Description:

Subretinal administration of a single high dose AAV CNGA3

Treatment:

Biological: adeno-associated virus vector AAV- CNGA3