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Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and 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: NCT03278873
Recruitment Status : Recruiting
First Posted : September 12, 2017
Last Update Posted : September 12, 2019
Sponsor:
Collaborators:
Syne Qua Non Limited
EMAS Pharma
Information provided by (Responsible Party):
MeiraGTx UK II Ltd

Study Description
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Brief Summary:
This study is a longer-term follow-up study for patients who participated in one of the clinical trials: AAV - CNGB3 retinal gene therapy for patients with achromatopsia, or AAV - CNGA3 retinal gene therapy for patients with achromatopsia.

Condition or disease Intervention/treatment Phase
Achromatopsia Biological: either AAV - CNGB3 or AAV - CNGA3 Phase 1 Phase 2

Detailed Description:
The follow up study is designed to collect data on longer-term safety and efficacy.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 72 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description: Follow-up of patients from two separate trials but there is no difference in follow-up assessments for the patients therefore they can be considered a "single group"
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3 and AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3 or CNGA3
Actual Study Start Date : June 27, 2017
Estimated Primary Completion Date : July 10, 2024
Estimated Study Completion Date : August 7, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3
Subretinal administration of a single low dose of either AAV - CNGB3 or AAV - CNGA3
 Biological: either AAV - CNGB3 or AAV - CNGA3
Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3
Experimental: Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3
Subretinal administration of a single medium dose of either AAV - CNGB3 or AAV - CNGA3
 Biological: either AAV - CNGB3 or AAV - CNGA3
Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3
Experimental: Biological-high dose of either AAV - CNGB3 or AAV - CNGA3
Subretinal administration of a single high dose of either AAV - CNGB3 or AAV - CNGA3
 Biological: either AAV - CNGB3 or AAV - CNGA3
Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of Adverse Events related to the treatment [ Time Frame: 5 Years ]
    longer term safety as the absence of ATIMP-related safety events


Secondary Outcome Measures :
  1. Improvement in the visual function [ Time Frame: 5 years ]
    Improvements in visual function as assessed by visual assessment

  2. Improvement in retinal function [ Time Frame: 5 years ]
    Improvement in the retinal function as assessed by visual assessment

  3. Improvement in quality of life [ Time Frame: 5 years ]
    Quality of life will be measured by QoL questionnaires


Eligibility Criteria
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Information from the National Library of Medicine

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 to 100 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion in the study will be limited to individuals who:

  1. Are able to give informed consent or assent, with or without the guidance of their parent(s)/guardian(s) where appropriate
  2. Received AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 by intraocular administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-002290-35 or EudraCT 2018-003431-29)
  3. Are willing to adhere to the protocol and long-term follow-up

Individuals will be excluded who:

Are unwilling or unable to meet with the requirements of the study

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): NCT03278873


Contacts
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Contact: MeiraGTx UK II Ltd +44 (0)20 3866 4320 ocularinfo@meiragtx.com

Locations
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United Kingdom
Moorfields Eye Hospital NHS Foundation Trust Recruiting
London, United Kingdom
Contact: Michel Michaelides       michel.michaelides@ucl.ac.uk   
Principal Investigator: James Bainbridge, Prof         
Sub-Investigator: Michel Michaelides, Prof         
Sponsors and Collaborators
MeiraGTx UK II Ltd
Syne Qua Non Limited
EMAS Pharma
More Information
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Responsible Party: MeiraGTx UK II Ltd
ClinicalTrials.gov Identifier: NCT03278873    
Other Study ID Numbers: MGT007
2016-003856-59 ( EudraCT Number )
First Posted: September 12, 2017    Key Record Dates
Last Update Posted: September 12, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by MeiraGTx UK II Ltd:
Achromatopsia
CNGB3
CNGA3
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
Signs and Symptoms