Gene Therapy for Achromatopsia (CNGA3)

Plain-language summary

This study examines a gene therapy (an adeno-associated virus vector called AAV-CNGA3) in patients with achromatopsia, an inherited eye condition caused by CNGA3 gene changes that leads to color blindness. Patients whose achromatopsia had been confirmed by a retinal specialist were able to take part. 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

adeno-associated virus vector AAV- CNGA3

Start date

8/12/2019

Estimated completion

6/10/2021

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 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