<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/NCT03252847</url>
  </required_header>
  <id_info>
    <org_study_id>MGT009</org_study_id>
    <nct_id>NCT03252847</nct_id>
  </id_info>
  <brief_title>Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)</brief_title>
  <official_title>An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2-.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)</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>Bionical Emas</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>
      The Phase 1 part of the study is a dose escalation of subretinal administration of AAV2/5&#xD;
      vector to assess the safety of this vector in participants with XLRP caused by mutations in&#xD;
      RPGR.&#xD;
&#xD;
      The Phase 2 part of the study is a cohort expansion of subretinal administration of AAV2/5&#xD;
      vector to assess the safety and efficacy of this vector in participants with XLRP caused by&#xD;
      mutations in RPGR.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is an open-label phase 1/2 dose-escalation and cohort expansion trial to determine the&#xD;
      safety and efficacy of subretinal administration of AAV2/5 vector in participants with XLRP&#xD;
      caused by mutations in RPGR.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">July 14, 2017</start_date>
  <completion_date type="Actual">November 18, 2021</completion_date>
  <primary_completion_date type="Actual">November 18, 2021</primary_completion_date>
  <phase>Phase 1/Phase 2</phase>
  <study_type>Interventional</study_type>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Sequential Assignment</intervention_model>
    <intervention_model_description>Biological</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 sub retinal administration of AAV2-RPGR</measure>
    <time_frame>18 months</time_frame>
    <description>Safety is defined as the absence of advanced therapy medicinal product (ATIMP)-related safety events</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Improvement in visual function</measure>
    <time_frame>18 months</time_frame>
    <description>Improvements in visual function as assessed by ocular examination</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Improvement in retinal function</measure>
    <time_frame>18 months</time_frame>
    <description>Improvements in retinal function as assessed by retinal assessement</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Improvement in Quality of Life</measure>
    <time_frame>18 months</time_frame>
    <description>•Quality of life will be measured by QoL questionnaire</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">49</enrollment>
  <condition>X-Linked Retinitis Pigmentosa</condition>
  <arm_group>
    <arm_group_label>Phase 1 (Part 1, Dose Escalation)</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants receive one of three doses of AAV2/5-RPGR</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Phase 2 (Part 2; Expansion)</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants receive one of two doses of AAV2/5-RPGR</description>
  </arm_group>
  <intervention>
    <intervention_type>Genetic</intervention_type>
    <intervention_name>AAV2/5-RPGR</intervention_name>
    <description>Single, subretinal administration of AAV2/5-RPGR</description>
    <arm_group_label>Phase 1 (Part 1, Dose Escalation)</arm_group_label>
    <arm_group_label>Phase 2 (Part 2; Expansion)</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Key inclusion Criteria:&#xD;
&#xD;
          -  Are aged 5 years or older male&#xD;
&#xD;
          -  Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI)&#xD;
&#xD;
        Key exclusion Criteria:&#xD;
&#xD;
        • Have participated in another research study involving an investigational medicinal&#xD;
        therapy for ocular disease within the last 6 months&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>5 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>University College, London</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Massachusetts Eye and Ear Institute</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kellogg Eye Center</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UPMC Eye Center</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15213</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Leeds Teaching Hospitals NHS Trust</name>
      <address>
        <city>Leeds</city>
        <zip>LS9 7TF</zip>
        <country>United Kingdom</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>November 2022</verification_date>
  <study_first_submitted>August 1, 2017</study_first_submitted>
  <study_first_submitted_qc>August 16, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 17, 2017</study_first_posted>
  <disposition_first_submitted>November 14, 2022</disposition_first_submitted>
  <disposition_first_submitted_qc>December 2, 2022</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">December 7, 2022</disposition_first_posted>
  <last_update_submitted>December 2, 2022</last_update_submitted>
  <last_update_submitted_qc>December 2, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">December 7, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>XLRP RPGR</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Retinitis</mesh_term>
    <mesh_term>Retinitis Pigmentosa</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted, but a results disposition has been submitted           -->
</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/NCT03252847
org study id: MGT009
nct id: NCT03252847
lead sponsor: 
collaborator: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The Phase 1 part of the study is a dose escalation of subretinal administration of AAV2/5
      vector to assess the safety of this vector in participants with XLRP caused by mutations in
      RPGR.

      The Phase 2 part of the study is a cohort expansion of subretinal administration of AAV2/5
      vector to assess the safety and efficacy of this vector in participants with XLRP caused by
      mutations in RPGR.
This is an open-label phase 1/2 dose-escalation and cohort expansion trial to determine the
      safety and efficacy of subretinal administration of AAV2/5 vector in participants with XLRP
      caused by mutations in RPGR.
allocation: Randomized
intervention model: Sequential Assignment
intervention model description: Biological
primary purpose: Treatment
masking: None (Open Label)
measure: Incidence of Adverse Events related to the sub retinal administration of AAV2-RPGR
time frame: 18 months
description: Safety is defined as the absence of advanced therapy medicinal product (ATIMP)-related safety events
measure: Improvement in visual function
time frame: 18 months
description: Improvements in visual function as assessed by ocular examination
measure: Improvement in retinal function
time frame: 18 months
description: Improvements in retinal function as assessed by retinal assessement
measure: Improvement in Quality of Life
time frame: 18 months
description: •Quality of life will be measured by QoL questionnaire
arm group label: Phase 1 (Part 1, Dose Escalation)
arm group type: Experimental
description: Participants receive one of three doses of AAV2/5-RPGR
arm group label: Phase 2 (Part 2; Expansion)
arm group type: Experimental
description: Participants receive one of two doses of AAV2/5-RPGR
intervention type: Genetic
intervention name: AAV2/5-RPGR
description: Single, subretinal administration of AAV2/5-RPGR
arm group label: Phase 1 (Part 1, Dose Escalation)
arm group label: Phase 2 (Part 2; Expansion)
criteria: 
gender: Male
minimum age: 5 Years
maximum age: N/A
healthy volunteers: No
last name: James Bainbridge, Prof
role: Principal Investigator
affiliation: University College, London
facility: 
facility: 
facility: 
facility: 
facility: 
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Retinitis
mesh term: Retinitis Pigmentosa
sharing ipd: No