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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
Last Update Posted : October 20, 2020
Sponsor:
Collaborators:
EMAS Pharma
Syne Qua Non Limited
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: 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-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

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


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 assessment

  2. Improvement in retinal function [ Time Frame: 6 months ]
    Improvements in retinal function as assessed by visual assessment

  3. Improvement in Quality of life [ Time Frame: 6 months ]
    Quality of life will be measured by the QoL questionnaire


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 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
EMAS Pharma
Syne Qua Non Limited
Investigators
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Principal Investigator: James Bainbridge, Prof Chief Investigator
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
Last Update Posted: October 20, 2020
Last Verified: October 2020
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