Gene Therapy for Achromatopsia (CNGB3)

Plain-language summary

This study examines a gene therapy (AAV-CNGB3) delivered using an AAV vector in people with achromatopsia (an inherited eye disease causing color blindness) linked to the CNGB3 gene. It enrolled people aged 3 and over whose achromatopsia was confirmed by a retinal specialist. The study was carried out in Phase 1/Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Completed

Sponsor

MeiraGTx UK II Ltd

Intervention

AAV - CNGB3

Start date

1/16/2017

Estimated completion

10/25/2019

Countries

United States, United Kingdom

Related genes

Locations

• Ann Arbor, United States
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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