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Gene Therapy for Achromatopsia (CNGB3) (CNGB3)

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
Results First Posted : March 8, 2023
Last Update Posted : March 8, 2023
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 - CNGB3 retinal gene therapy for patients with achromatopsia

Condition or disease Intervention/treatment Phase
Achromatopsia Biological: AAV - CNGB3 Phase 1 Phase 2

Detailed Description:
CNGB3 retinal gene therapy for patients with achromatopsia
Study Design
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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

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Biological-Low dose AAV - CNGB3
Subretinal administration of a single low dose of AAV - CNGB3
 Biological: AAV - CNGB3
Comparison of different dosages of AAV-CNGB3
Experimental: Biological-Medium dose AAV - CNGB3
Subretinal administration of a single intermediate dose of 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 AAV - CNGB3
 Biological: AAV - CNGB3
Comparison of different dosages of AAV-CNGB3


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 in the treated eye. 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-5D-Y 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 change from baseline reflects worsening.

  4. Quality of Life Measured by QoL Questionnaires in Adults [ Time Frame: 6 Months ]
    Change from baseline to Week 24 in EuroQol-5D-5L 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 change from baseline reflects worsening.


Eligibility Criteria
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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.


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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 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
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): NCT03001310


Locations
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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
Sponsors and Collaborators
MeiraGTx UK II Ltd
Janssen Research & Development, LLC
Investigators
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Principal Investigator: James Bainbridge, Prof Chief Investigator
  Study Documents (Full-Text)

Documents provided by MeiraGTx UK II Ltd:
Study Protocol  [PDF] January 29, 2019
Statistical Analysis Plan  [PDF] December 18, 2019

More Information
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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
Results First Posted: March 8, 2023
Last Update Posted: March 8, 2023
Last Verified: February 2023
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