Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3

MeiraGTx

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Achromatopsia

Treatments: Biological: Prior exposure to AAV-CNGA3 or AAV-CNGB3

Study type: Observational

Funder types: Industry

Identifiers

NCT03278873

MGT007

2016-003856-59 (EudraCT Number)

Details and patient eligibility

About

This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia associated with defects in CNGB3 who participated in a clinical trial in which they received AAV-CNGB3 retinal gene therapy.

Full description

The follow-up study is designed to collect data on the longer-term safety and efficacy of AAV-CNGA3 retinal gene therapy and AAV-CNGB3 retinal gene therapy.

Enrollment: 34 patients

Sex: All

Ages: 3 to 100 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion in the study will be limited to individuals who:

1. Are able to give informed consent or assent, with or without the guidance of their parent(s)/guardian(s), where appropriate
2. Received AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 by intraocular administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-002290-35 or EudraCT 2018-003431-29)
3. Are willing to adhere to the protocol and long-term follow-up

Individuals will be excluded who:

Are unwilling or unable to meet the requirements of the study

Trial design

34 participants in 4 patient groups

Low dose of AAV-CNGA3 or AAV-CNGB3

Description:

Subretinal administration of a single low dose of AAV-CNGA3 or AAV-CNGB3

Treatment:

Biological: Prior exposure to AAV-CNGA3 or AAV-CNGB3

Intermediate dose of AAV-CNGA3 or AAV-CNGB3

Description:

Subretinal administration of a single intermediate dose of AAV-CNGA3 or AAV-CNGB3

Treatment:

Biological: Prior exposure to AAV-CNGA3 or AAV-CNGB3

Other dose of AAV-CNGA3 or AAV-CNGB3

Description:

Subretinal administration of a single other dose (between the intermediate and high dose) of AAV-CNGA3 or AAV-CNGB3

Treatment:

Biological: Prior exposure to AAV-CNGA3 or AAV-CNGB3

High dose of AAV-CNGA3 or AAV-CNGB3

Description:

Subretinal administration of a single high dose of AAV-CNGA3 or AAV-CNGB3

Treatment:

Biological: Prior exposure to AAV-CNGA3 or AAV-CNGB3