<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/NCT03001310</url>
  </required_header>
  <id_info>
    <org_study_id>MGT006</org_study_id>
    <secondary_id>2016-002290-35</secondary_id>
    <nct_id>NCT03001310</nct_id>
  </id_info>
  <brief_title>Gene Therapy for Achromatopsia (CNGB3)</brief_title>
  <acronym>CNGB3</acronym>
  <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</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>MeiraGTx UK II Ltd</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Janssen Research &amp; Development, LLC</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>MeiraGTx UK II Ltd</source>
  <oversight_info>
    <has_dmc>Yes</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>
      A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      CNGB3 retinal gene therapy for patients with achromatopsia&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">January 16, 2017</start_date>
  <completion_date type="Actual">October 25, 2019</completion_date>
  <primary_completion_date type="Actual">October 25, 2019</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>Sequential Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>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.</measure>
    <time_frame>6 weeks</time_frame>
    <description>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:&#xD;
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&#xD;
Severe unresponsive inflammation&#xD;
Infective endophthalmitis&#xD;
Ocular malignancy&#xD;
Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Improvements in Visual Function as Assessed by Visual Acuity</measure>
    <time_frame>6 months</time_frame>
    <description>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.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Improvements in Retinal Function as Assessed by Static Perimetry</measure>
    <time_frame>6 months</time_frame>
    <description>Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Quality of Life Measured by QoL Questionnaires in Children and Adolescents</measure>
    <time_frame>6 months</time_frame>
    <description>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.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Quality of Life Measured by QoL Questionnaires in Adults</measure>
    <time_frame>6 Months</time_frame>
    <description>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.</description>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">23</enrollment>
  <condition>Achromatopsia</condition>
  <arm_group>
    <arm_group_label>Biological-Low dose AAV - CNGB3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Subretinal administration of a single low dose of AAV - CNGB3</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Biological-Medium dose AAV - CNGB3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Subretinal administration of a single intermediate dose of AAV - CNGB3</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Biological-High dose AAV - CNGB3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Subretinal administration of a single high dose of AAV - CNGB3</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>AAV - CNGB3</intervention_name>
    <description>Comparison of different dosages of AAV-CNGB3</description>
    <arm_group_label>Biological-High dose AAV - CNGB3</arm_group_label>
    <arm_group_label>Biological-Low dose AAV - CNGB3</arm_group_label>
    <arm_group_label>Biological-Medium dose AAV - CNGB3</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Are aged 3 years or older&#xD;
&#xD;
          -  Have achromatopsia confirmed by a retinal specialist (CI or PI)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Are females who are pregnant or breastfeeding&#xD;
&#xD;
          -  Have participated in another research study involving an investigational medicinal&#xD;
             therapy for ocular disease within the last 6 months&#xD;
&#xD;
          -  Have any other condition that the CI/PI considers makes them inappropriate for entry&#xD;
             into the trial&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>3 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>James Bainbridge, Prof</last_name>
    <role>Principal Investigator</role>
    <affiliation>Chief Investigator</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of Michigan Kellog Eye Centre</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>MI 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>November 22, 2016</study_first_submitted>
  <study_first_submitted_qc>December 19, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 23, 2016</study_first_posted>
  <results_first_submitted>January 3, 2023</results_first_submitted>
  <results_first_submitted_qc>February 9, 2023</results_first_submitted_qc>
  <results_first_posted type="Actual">March 8, 2023</results_first_posted>
  <disposition_first_submitted>October 14, 2020</disposition_first_submitted>
  <disposition_first_submitted_qc>October 14, 2020</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">October 20, 2020</disposition_first_posted>
  <last_update_submitted>February 9, 2023</last_update_submitted>
  <last_update_submitted_qc>February 9, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">March 8, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <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>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>January 29, 2019</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/10/NCT03001310/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>December 18, 2019</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/10/NCT03001310/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <recruitment_details>Participants were recruited from medical centers in the United Kingdom (UK) and the United States (US). A total of 23 participants were enrolled in the study.</recruitment_details>
      <group_list>
        <group group_id="P1">
          <title>Low Dose AAV - CNGB3</title>
          <description>Subretinal administration of a single low dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
        <group group_id="P2">
          <title>Intermediate Dose AAV-CNGB3</title>
          <description>Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
        <group group_id="P3">
          <title>Other Dose - AAV - CNGB3</title>
          <description>Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
        <group group_id="P4">
          <title>High Dose AAV - CNGB3</title>
          <description>Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="12"/>
                <participants group_id="P3" count="3"/>
                <participants group_id="P4" count="5"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="12"/>
                <participants group_id="P3" count="3"/>
                <participants group_id="P4" count="5"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
                <participants group_id="P3" count="0"/>
                <participants group_id="P4" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Low Dose AAV - CNGB3</title>
          <description>Subretinal administration of a single low dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
        <group group_id="B2">
          <title>Intermediate Dose AAV-CNGB3</title>
          <description>Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
        <group group_id="B3">
          <title>Other Dose - AAV - CNGB3</title>
          <description>Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
        <group group_id="B4">
          <title>High Dose AAV - CNGB3</title>
          <description>Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
        <group group_id="B5">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="3"/>
            <count group_id="B2" value="12"/>
            <count group_id="B3" value="3"/>
            <count group_id="B4" value="5"/>
            <count group_id="B5" value="23"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="8"/>
                    <measurement group_id="B3" value="3"/>
                    <measurement group_id="B4" value="2"/>
                    <measurement group_id="B5" value="13"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="3"/>
                    <measurement group_id="B5" value="10"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="10"/>
                    <measurement group_id="B3" value="3"/>
                    <measurement group_id="B4" value="3"/>
                    <measurement group_id="B5" value="18"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="2"/>
                    <measurement group_id="B5" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="12"/>
                    <measurement group_id="B3" value="3"/>
                    <measurement group_id="B4" value="5"/>
                    <measurement group_id="B5" value="23"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="12"/>
                    <measurement group_id="B3" value="3"/>
                    <measurement group_id="B4" value="5"/>
                    <measurement group_id="B5" value="23"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>United Kingdom</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="11"/>
                    <measurement group_id="B3" value="1"/>
                    <measurement group_id="B4" value="5"/>
                    <measurement group_id="B5" value="20"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>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.</title>
        <description>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:&#xD;
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&#xD;
Severe unresponsive inflammation&#xD;
Infective endophthalmitis&#xD;
Ocular malignancy&#xD;
Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)</description>
        <time_frame>6 weeks</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Low Dose AAV - CNGB3</title>
            <description>Subretinal administration of a single low dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O2">
            <title>Intermediate Dose AAV-CNGB3</title>
            <description>Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O3">
            <title>Other Dose - AAV - CNGB3</title>
            <description>Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O4">
            <title>High Dose AAV - CNGB3</title>
            <description>Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
        </group_list>
        <measure>
          <title>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.</title>
          <description>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:&#xD;
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&#xD;
Severe unresponsive inflammation&#xD;
Infective endophthalmitis&#xD;
Ocular malignancy&#xD;
Grade III or above non-ocular Suspected Unexpected Serious Adverse Reaction (SUSAR)</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="12"/>
                <count group_id="O3" value="3"/>
                <count group_id="O4" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="0"/>
                    <measurement group_id="O4" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Improvements in Visual Function as Assessed by Visual Acuity</title>
        <description>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.</description>
        <time_frame>6 months</time_frame>
        <population>22 of the 23 participants performed the visual acuity assessment at baseline and Week 24.</population>
        <group_list>
          <group group_id="O1">
            <title>Low Dose AAV - CNGB3</title>
            <description>Subretinal administration of a single low dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O2">
            <title>Intermediate Dose AAV-CNGB3</title>
            <description>Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O3">
            <title>Other Dose - AAV - CNGB3</title>
            <description>Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O4">
            <title>High Dose AAV - CNGB3</title>
            <description>Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
        </group_list>
        <measure>
          <title>Improvements in Visual Function as Assessed by Visual Acuity</title>
          <description>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.</description>
          <population>22 of the 23 participants performed the visual acuity assessment at baseline and Week 24.</population>
          <units>number of ETDRS letters</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="11"/>
                <count group_id="O3" value="3"/>
                <count group_id="O4" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.44" lower_limit="-2.0" upper_limit="1.3"/>
                    <measurement group_id="O2" value="-0.58" lower_limit="-4.3" upper_limit="3.3"/>
                    <measurement group_id="O3" value="1.78" lower_limit="0.3" upper_limit="4.7"/>
                    <measurement group_id="O4" value="1.45" lower_limit="-4.0" upper_limit="5.3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Improvements in Retinal Function as Assessed by Static Perimetry</title>
        <description>Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.</description>
        <time_frame>6 months</time_frame>
        <population>20 of the 23 participants had mean retinal sensitivity data available at both baseline and Week 24 in the treated eye.</population>
        <group_list>
          <group group_id="O1">
            <title>Low Dose AAV - CNGB3</title>
            <description>Subretinal administration of a single low dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O2">
            <title>Intermediate Dose AAV-CNGB3</title>
            <description>Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O3">
            <title>Other Dose - AAV - CNGB3</title>
            <description>Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O4">
            <title>High Dose AAV - CNGB3</title>
            <description>Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
        </group_list>
        <measure>
          <title>Improvements in Retinal Function as Assessed by Static Perimetry</title>
          <description>Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.</description>
          <population>20 of the 23 participants had mean retinal sensitivity data available at both baseline and Week 24 in the treated eye.</population>
          <units>decibel</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="9"/>
                <count group_id="O3" value="3"/>
                <count group_id="O4" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.58" lower_limit="-3.1" upper_limit="0.0"/>
                    <measurement group_id="O2" value="-0.39" lower_limit="-2.4" upper_limit="1.8"/>
                    <measurement group_id="O3" value="8.13" lower_limit="1.7" upper_limit="11.4"/>
                    <measurement group_id="O4" value="-0.77" lower_limit="-7.1" upper_limit="3.3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Quality of Life Measured by QoL Questionnaires in Children and Adolescents</title>
        <description>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.</description>
        <time_frame>6 months</time_frame>
        <population>EQ-VAS data were available for 11 children/adolescents enrolled in the study.</population>
        <group_list>
          <group group_id="O1">
            <title>Low Dose AAV - CNGB3</title>
            <description>Subretinal administration of a single low dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O2">
            <title>Intermediate Dose AAV-CNGB3</title>
            <description>Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O3">
            <title>Other Dose - AAV - CNGB3</title>
            <description>Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O4">
            <title>High Dose AAV - CNGB3</title>
            <description>Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
        </group_list>
        <measure>
          <title>Quality of Life Measured by QoL Questionnaires in Children and Adolescents</title>
          <description>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.</description>
          <population>EQ-VAS data were available for 11 children/adolescents enrolled in the study.</population>
          <units>units on a scale</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="7"/>
                <count group_id="O3" value="3"/>
                <count group_id="O4" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O2" value="6.9" lower_limit="-2" upper_limit="20"/>
                    <measurement group_id="O3" value="2.3" lower_limit="-3" upper_limit="10"/>
                    <measurement group_id="O4" value="-9.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Quality of Life Measured by QoL Questionnaires in Adults</title>
        <description>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.</description>
        <time_frame>6 Months</time_frame>
        <population>EQ-VAS data were available for 11 adults enrolled in the study.</population>
        <group_list>
          <group group_id="O1">
            <title>Low Dose AAV - CNGB3</title>
            <description>Subretinal administration of a single low dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O2">
            <title>Intermediate Dose AAV-CNGB3</title>
            <description>Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O3">
            <title>Other Dose - AAV - CNGB3</title>
            <description>Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
          <group group_id="O4">
            <title>High Dose AAV - CNGB3</title>
            <description>Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
          </group>
        </group_list>
        <measure>
          <title>Quality of Life Measured by QoL Questionnaires in Adults</title>
          <description>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.</description>
          <population>EQ-VAS data were available for 11 adults enrolled in the study.</population>
          <units>units on a scale</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="0"/>
                <count group_id="O4" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0" lower_limit="-10" upper_limit="10"/>
                    <measurement group_id="O2" value="-3.8" lower_limit="-10" upper_limit="5"/>
                    <measurement group_id="O4" value="4.3" lower_limit="-5" upper_limit="20"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>6 months</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Low Dose AAV - CNGB3</title>
          <description>Subretinal administration of a single low dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
        <group group_id="E2">
          <title>Intermediate Dose AAV-CNGB3</title>
          <description>Subretinal administration of a single intermediate dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
        <group group_id="E3">
          <title>Other Dose - AAV - CNGB3</title>
          <description>Subretinal administration of a single other dose (between intermediate and high dose levels) of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
        <group group_id="E4">
          <title>High Dose AAV - CNGB3</title>
          <description>Subretinal administration of a single high dose of adeno-associated virus AAV-CNGB3; AAV-CNGB3: AAV gene therapy for defects in CNGB3 gene</description>
        </group>
        <group group_id="E5">
          <title>Total</title>
          <description>Safety Analysis Set</description>
        </group>
      </group_list>
      <serious_events>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Eye disorders</title>
            <event_list>
              <event>
                <sub_title>Uveitis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Pregnancy, puerperium and perinatal conditions</title>
            <event_list>
              <event>
                <sub_title>Ectopic pregnancy</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="1" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="12" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="5" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="23" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Eye disorders</title>
            <event_list>
              <event>
                <sub_title>Conjunctival haemorrhage</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="10" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="5" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="20" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Lenticular opacities</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="7" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="3" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="15" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Visual acuity reduced</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="7" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="5" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="15" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Subretinal fluid</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="4" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="3" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="9" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Foreign body sensation in eyes</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="7" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="8" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Ocular discomfort</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="5" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="3" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="8" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Chorioretinal folds</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="4" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="2" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="6" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Retinal haemorrhage</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="2" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="6" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Conjunctival hyperaemia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="3" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="4" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Eye inflammation</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="4" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Maculopathy</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="3" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="4" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Retinal depigmentation</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="4" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Retinal oedema</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="2" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="4" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Retinal tear</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="3" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="4" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Eye pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Photophobia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Vitreous floaters</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Conjunctival oedema</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Corneal disorder</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Macular fibrosis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Retinal disorder</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Retinal pigment epitheliopathy</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Uveitis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="2" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Rhinitis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="4" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="5" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Postoperative wound infection</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Intraocular pressure increased</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="2" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="6" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="3" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="6" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Visual field defect</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="4" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="5" subjects_at_risk="23"/>
              </event>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="4" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Rash</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="12"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="23"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Program Manager</name_or_title>
      <organization>MeiraGTx</organization>
      <phone>0044 (0)20 3866 4320</phone>
      <email>CNGB3@meiragtx.com</email>
    </point_of_contact>
  </clinical_results>
</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/NCT03001310
org study id: MGT006
secondary id: 2016-002290-35
nct id: NCT03001310
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
A clinical trial of AAV - CNGB3 retinal gene therapy for patients with achromatopsia
CNGB3 retinal gene therapy for patients with achromatopsia
allocation: Non-Randomized
intervention model: Sequential Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: 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
description: 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)
measure: Improvements in Visual Function as Assessed by Visual Acuity
time frame: 6 months
description: 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.
measure: Improvements in Retinal Function as Assessed by Static Perimetry
time frame: 6 months
description: Change from baseline to Week 24 in mean retinal sensitivity in the treated eye. The direction of improvement is an increase in sensitivity.
measure: Quality of Life Measured by QoL Questionnaires in Children and Adolescents
time frame: 6 months
description: 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.
measure: Quality of Life Measured by QoL Questionnaires in Adults
time frame: 6 Months
description: 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.
arm group label: Biological-Low dose AAV - CNGB3
arm group type: Experimental
description: Subretinal administration of a single low dose of AAV - CNGB3
arm group label: Biological-Medium dose AAV - CNGB3
arm group type: Experimental
description: Subretinal administration of a single intermediate dose of AAV - CNGB3
arm group label: Biological-High dose AAV - CNGB3
arm group type: Experimental
description: Subretinal administration of a single high dose of AAV - CNGB3
intervention type: Biological
intervention name: AAV - CNGB3
description: Comparison of different dosages of AAV-CNGB3
arm group label: Biological-High dose AAV - CNGB3
arm group label: Biological-Low dose AAV - CNGB3
arm group label: Biological-Medium dose AAV - CNGB3
criteria: 
gender: All
minimum age: 3 Years
maximum age: N/A
healthy volunteers: No
last name: James Bainbridge, Prof
role: Principal Investigator
affiliation: Chief Investigator
facility: 
facility: 
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Color Vision Defects
sharing ipd: No
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 