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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
Results First Posted : February 11, 2022
Last Update Posted : December 1, 2022
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 adeno-associated virus vector (AAV) CNGA3 retinal gene therapy for patients with achromatopsia

Condition or disease Intervention/treatment Phase
Achromatopsia Biological: adeno-associated virus vector 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 : August 12, 2019
Actual Primary Completion Date : June 10, 2021
Actual Study Completion Date : June 10, 2021

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Low dose adeno-associated virus (AAV) CNGA3
Subretinal administration of a single low dose AAV CNGA3
 Biological: adeno-associated virus vector AAV- CNGA3
Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene
Experimental: Intermediate dose adeno-associated virus (AAV) CNGA3
Subretinal administration of a single intermediate dose AAV CNGA3
 Biological: adeno-associated virus vector AAV- CNGA3
Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene
Experimental: High dose adeno-associated virus (AAV) CNGA3
Subretinal administration of a single high dose AAV CNGA3
 Biological: adeno-associated virus vector AAV- CNGA3
Adeno-associated virus (AAV) gene therapy for defects in CNGA3 gene


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to the Advanced Therapy Investigational Medicinal Products (ATIMP), Not Surgery Alone. [ Time Frame: 6 Weeks ]

    The primary outcome is defined as any of the below occurring during the 6 weeks following administration, at least possibly related to the Advanced Therapy Investigational Medicinal Products (ATIMP), not surgery alone:

    • Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more that fails to resolve to within 15 letters of baseline in a 4-week period once prophylactic treatment commences
    • Severe unresponsive inflammation
    • Infective endophthalmitis
    • Ocular malignancy
    • Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)


Secondary Outcome Measures :
  1. Improvements in Visual Function as Assessed by Visual Acuity [ Time Frame: 6 Months ]
    Change from baseline to Week 24 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time.

  2. Improvements in Retinal Function as Assessed by Static Perimetry [ Time Frame: 6 Months ]
    Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.

  3. Quality of Life Measured by QoL Questionnaires in Children and Adolescents [ Time Frame: 6 Months ]
    Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.

  4. Quality of Life Measured by QoL Questionnaires in Adults [ Time Frame: 6 Months ]
    Change from baseline to Week 24 in EuroQol Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement and a negative reflects worsening.


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 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
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
  Study Documents (Full-Text)

Documents provided by MeiraGTx UK II Ltd:
Study Protocol  [PDF] April 16, 2021
Statistical Analysis Plan  [PDF] September 2, 2021

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
Results First Posted: February 11, 2022
Last Update Posted: December 1, 2022
Last Verified: November 2022
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