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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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03758404
Recruitment Status : Completed
First Posted : November 29, 2018
Last Update Posted : June 25, 2021
Sponsor:
Collaborator:
Janssen Research & Development, LLC
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Study Description
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Brief Summary:
A clinical trial of AAV - CNGA3 retinal gene therapy for patients with achromatopsia

Condition or disease Intervention/treatment Phase
Achromatopsia Biological: AAV- CNGA3 Phase 1 Phase 2

Detailed Description:
CNGA3 retinal gene therapy for patients with achromatopsia
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 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
Actual Primary Completion Date : June 10, 2021
Actual Study Completion Date : June 10, 2021

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Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of Adverse Events related to the treatment [ Time Frame: 6 Months ]
    Safety is defined as the absence of ATIMP-related safety events


Secondary Outcome Measures :
  1. Improvement in visual function [ Time Frame: 6 Months ]
    Improvements in visual function as assessed by visual acuity

  2. Improvement in retinal function [ Time Frame: 6 Months ]
    Improvements in retinal function as assessed by microperimetry and perimetry

  3. Improvement in Quality of life [ Time Frame: 6 Months ]
    Quality of life will be measured by QoL questionnaires


Eligibility Criteria
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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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United States, Michigan
Kellogg Eye Center
Ann Arbor, Michigan, United States, 48105
United Kingdom
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom, EC1V 2PD
Sponsors and Collaborators
MeiraGTx UK II Ltd
Janssen Research & Development, LLC
Investigators
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Principal Investigator: James Bainbridge Chief Investigator
More Information
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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: June 25, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Color Vision Defects
Vision Disorders
Sensation Disorders
Neurologic Manifestations
 Nervous System Diseases
Cone Dystrophy
Eye Diseases, Hereditary
Eye Diseases