Gene Therapy for Achromatopsia (CNGB3)

MeiraGTx

Status and phase: Completed; Phase 2; Phase 1

Conditions: Achromatopsia

Treatments: Biological: AAV - CNGB3

Study type: Interventional

Funder types: Industry

Identifiers

NCT03001310

2016-002290-35 (EudraCT Number)

MGT006

Details and patient eligibility

About

A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia

Full description

CNGB3 retinal gene therapy for patients with achromatopsia

Enrollment: 23 patients

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Are aged 3 years or older
• Have achromatopsia confirmed by a retinal specialist (CI or PI)

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 CI/PI considers makes them inappropriate for entry into the trial

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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23 participants in 3 patient groups

Biological-Low dose AAV - CNGB3

Experimental group

Description:

Subretinal administration of a single low dose of AAV - CNGB3

Treatment:

Biological: AAV - CNGB3

Biological-Medium dose AAV - CNGB3

Experimental group

Description:

Subretinal administration of a single intermediate dose of AAV - CNGB3

Treatment:

Biological: AAV - CNGB3

Biological-High dose AAV - CNGB3

Experimental group

Description:

Subretinal administration of a single high dose of AAV - CNGB3

Treatment:

Biological: AAV - CNGB3