Gene Therapy for Achromatopsia (CNGB3) (CNGB3)
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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. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03001310 |
Recruitment Status :
Completed
First Posted : December 23, 2016
Last Update Posted : January 22, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Achromatopsia | Biological: AAV - CNGB3 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 23 participants |
Allocation: | Non-Randomized |
Intervention Model: | Sequential 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-hCARp.hCNGB3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3 |
Actual Study Start Date : | January 16, 2017 |
Actual Primary Completion Date : | October 25, 2019 |
Actual Study Completion Date : | October 25, 2019 |

Arm | Intervention/treatment |
---|---|
Experimental: Biological-Low dose AAV - CNGB3
Subretinal administration of a single low dose of range AAV - CNGB3
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Biological: AAV - CNGB3
Comparison of different dosages of AAV-CNGB3 |
Experimental: Biological-medium dose AAV - CNGB3
Subretinal administration of a single medium dose of range AAV - CNGB3
|
Biological: AAV - CNGB3
Comparison of different dosages of AAV-CNGB3 |
Experimental: Biological-high dose AAV - CNGB3
Subretinal administration of a single high dose of range AAV - CNGB3
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Biological: AAV - CNGB3
Comparison of different dosages of AAV-CNGB3 |
- 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 assessment
- Improvement in retinal function [ Time Frame: 6 months ]Improvements in retinal function as assessed by visual assessment
- Improvement in Quality of life [ Time Frame: 6 months ]Quality of life will be measured by the QoL questionnaire

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 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

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): NCT03001310
United States, Michigan | |
University of Michigan Kellog Eye Centre | |
Ann Arbor, Michigan, United States, MI 48105 | |
United Kingdom | |
Moorfields Eye Hospital NHS Foundation Trust | |
London, United Kingdom |
Principal Investigator: | James Bainbridge, Prof | Chief Investigator |
Responsible Party: | MeiraGTx UK II Ltd |
ClinicalTrials.gov Identifier: | NCT03001310 |
Other Study ID Numbers: |
MGT006 2016-002290-35 ( EudraCT Number ) |
First Posted: | December 23, 2016 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 |