<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03278873</url>
  </required_header>
  <id_info>
    <org_study_id>MGT007</org_study_id>
    <secondary_id>2016-003856-59</secondary_id>
    <nct_id>NCT03278873</nct_id>
  </id_info>
  <brief_title>Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3</brief_title>
  <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</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>MeiraGTx UK II Ltd</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Syne Qua Non Limited</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
    <collaborator>
      <agency>EMAS Pharma</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>MeiraGTx UK II Ltd</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study is a longer-term follow-up study for patients who participated in one of the&#xD;
      clinical trials: AAV - CNGB3 retinal gene therapy for patients with achromatopsia, or AAV -&#xD;
      CNGA3 retinal gene therapy for patients with achromatopsia.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The follow up study is designed to collect data on longer-term safety and efficacy.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">June 29, 2017</start_date>
  <completion_date type="Anticipated">January 15, 2026</completion_date>
  <primary_completion_date type="Anticipated">January 15, 2026</primary_completion_date>
  <phase>Phase 1/Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <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 &quot;single group&quot;</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Incidence of Adverse Events related to the treatment</measure>
    <time_frame>5 Years</time_frame>
    <description>longer term safety as the absence of ATIMP-related safety events</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Improvement in the visual function</measure>
    <time_frame>5 years</time_frame>
    <description>Improvements in visual function as assessed by visual assessment</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Improvement in retinal function</measure>
    <time_frame>5 years</time_frame>
    <description>Improvement in the retinal function as assessed by visual assessment</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Improvement in quality of life</measure>
    <time_frame>5 years</time_frame>
    <description>Quality of life will be measured by QoL questionnaires</description>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">34</enrollment>
  <condition>Achromatopsia</condition>
  <arm_group>
    <arm_group_label>Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Subretinal administration of a single low dose of either AAV - CNGB3 or AAV - CNGA3</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Subretinal administration of a single medium dose of either AAV - CNGB3 or AAV - CNGA3</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Biological-high dose of either AAV - CNGB3 or AAV - CNGA3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Subretinal administration of a single high dose of either AAV - CNGB3 or AAV - CNGA3</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>either AAV - CNGB3 or AAV - CNGA3</intervention_name>
    <description>Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3</description>
    <arm_group_label>Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3</arm_group_label>
    <arm_group_label>Biological-high dose of either AAV - CNGB3 or AAV - CNGA3</arm_group_label>
    <arm_group_label>Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion in the study will be limited to individuals who:&#xD;
&#xD;
          1. Are able to give informed consent or assent, with or without the guidance of their&#xD;
             parent(s)/guardian(s) where appropriate&#xD;
&#xD;
          2. Received AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 by intraocular administration in&#xD;
             the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-002290-35 or&#xD;
             EudraCT 2018-003431-29)&#xD;
&#xD;
          3. Are willing to adhere to the protocol and long-term follow-up&#xD;
&#xD;
        Individuals will be excluded who:&#xD;
&#xD;
        Are unwilling or unable to meet with the requirements of the study&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>3 Years</minimum_age>
    <maximum_age>100 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Kellog Eye Center</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Moorfields Eye Hospital NHS Foundation Trust</name>
      <address>
        <city>London</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>February 2023</verification_date>
  <study_first_submitted>August 16, 2017</study_first_submitted>
  <study_first_submitted_qc>September 7, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 12, 2017</study_first_posted>
  <last_update_submitted>February 13, 2023</last_update_submitted>
  <last_update_submitted_qc>February 13, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">February 14, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Achromatopsia</keyword>
  <keyword>CNGB3</keyword>
  <keyword>CNGA3</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Color Vision Defects</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03278873
org study id: MGT007
secondary id: 2016-003856-59
nct id: NCT03278873
lead sponsor: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
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.
The follow up study is designed to collect data on longer-term safety and efficacy.
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"
primary purpose: Treatment
masking: None (Open Label)
measure: Incidence of Adverse Events related to the treatment
time frame: 5 Years
description: longer term safety as the absence of ATIMP-related safety events
measure: Improvement in the visual function
time frame: 5 years
description: Improvements in visual function as assessed by visual assessment
measure: Improvement in retinal function
time frame: 5 years
description: Improvement in the retinal function as assessed by visual assessment
measure: Improvement in quality of life
time frame: 5 years
description: Quality of life will be measured by QoL questionnaires
arm group label: Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3
arm group type: Experimental
description: Subretinal administration of a single low dose of either AAV - CNGB3 or AAV - CNGA3
arm group label: Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3
arm group type: Experimental
description: Subretinal administration of a single medium dose of either AAV - CNGB3 or AAV - CNGA3
arm group label: Biological-high dose of either AAV - CNGB3 or AAV - CNGA3
arm group type: Experimental
description: Subretinal administration of a single high dose of either AAV - CNGB3 or AAV - CNGA3
intervention type: Biological
intervention name: either AAV - CNGB3 or AAV - CNGA3
description: Comparison of different dosages of either AAV - CNGB3 or AAV - CNGA3
arm group label: Biological-Low dose of either AAV - CNGB3 or AAV - CNGA3
arm group label: Biological-high dose of either AAV - CNGB3 or AAV - CNGA3
arm group label: Biological-medium dose of either AAV - CNGB3 or AAV - CNGA3
criteria: 
gender: All
minimum age: 3 Years
maximum age: 100 Years
healthy volunteers: No
facility: 
facility: 
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Color Vision Defects
sharing ipd: No