Gene Therapy for Achromatopsia (CNGA3) (CNGA3)
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03758404 |
Recruitment Status :
Recruiting
First Posted : November 29, 2018
Last Update Posted : January 22, 2021
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Achromatopsia | Biological: AAV- CNGA3 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 18 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Children and Adults With Achromatopsia Owing to Defects in CNGA3 |
Actual Study Start Date : | July 18, 2019 |
Estimated Primary Completion Date : | May 2021 |
Estimated Study Completion Date : | January 2022 |

Arm | Intervention/treatment |
---|---|
Experimental: Low dose AAV - CNGA3
Subretinal administration of a single low dose of range AAV - CNGA3
|
Biological: AAV- CNGA3
AAV gene therapy for defects in CNGA3 gene |
Experimental: Intermediate dose AAV - CNGA3
Subretinal administration of a single intermediate dose of range AAV - CNGA3
|
Biological: AAV- CNGA3
AAV gene therapy for defects in CNGA3 gene |
Experimental: High dose AAV - CNGA3
Subretinal administration of a single high dose of range AAV - CNGA3
|
Biological: AAV- CNGA3
AAV gene therapy for defects in CNGA3 gene |
- Incidence of Adverse Events related to the treatment [ Time Frame: 6 Months ]Safety is defined as the absence of ATIMP-related safety events
- Improvement in visual function [ Time Frame: 6 Months ]Improvements in visual function as assessed by visual acuity
- Improvement in retinal function [ Time Frame: 6 Months ]Improvements in retinal function as assessed by microperimetry and perimetry
- Improvement in Quality of life [ Time Frame: 6 Months ]Quality of life will be measured by QoL questionnaires

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 3 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Are aged years or over
- 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03758404
Contact: MeiraGTx UK II Ltd | +44 (0)02 3866 4320 | ocularinfo@meiragtx.com |
United States, Michigan | |
Kellogg Eye Center | Recruiting |
Ann Arbor, Michigan, United States, 48105 | |
Contact: Adrienne Chen 734-232-9167 KelloggResearch@med.umich.edu | |
Principal Investigator: Cagri Besirli | |
United Kingdom | |
Moorfields Eye Hospital NHS Foundation Trust | Recruiting |
London, United Kingdom, EC1V 2PD | |
Contact: James Bainbridge james.bainbridge1@nhs.net | |
Contact: Michel Michaelides michel.michaelides@nhs.net | |
Principal Investigator: James Bainbridge, Prof | |
Sub-Investigator: Michel Michaelides, Prof |
Principal Investigator: | James Bainbridge | Chief Investigator |
Responsible Party: | MeiraGTx UK II Ltd |
ClinicalTrials.gov Identifier: | NCT03758404 |
Other Study ID Numbers: |
MGT012 |
First Posted: | November 29, 2018 Key Record Dates |
Last Update Posted: | January 22, 2021 |
Last Verified: | January 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Color Vision Defects Vision Disorders Sensation Disorders Neurologic Manifestations |
Nervous System Diseases Cone Dystrophy Eye Diseases, Hereditary Eye Diseases |