<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/NCT04437368</url>
  </required_header>
  <id_info>
    <org_study_id>GT005-02</org_study_id>
    <nct_id>NCT04437368</nct_id>
  </id_info>
  <brief_title>EXPLORE: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005</brief_title>
  <acronym>EXPLORE</acronym>
  <official_title>EXPLORE: A Phase II, Outcomes Assessor-masked, Multicentre, Randomised Study to Evaluate the Safety and Efficacy of Two Doses of GT005 Administered as a Single Subretinal Injection in Subjects With Geographic Atrophy Secondary to Age-related Macular Degeneration</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Gyroscope Therapeutics Limited</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Novartis Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Gyroscope Therapeutics Limited</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 purpose of this clinical study is to evaluate the safety and efficacy of two doses of&#xD;
      GT005 administered as a single subretinal injection in subjects with geographic atrophy&#xD;
      secondary to age-related macular degeneration (AMD).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a Phase 2, outcomes assessor-masked, multicentre, randomised study to evaluate the&#xD;
      safety and efficacy of two doses of GT005 administered as a single subretinal injection in&#xD;
      subjects with GA secondary to AMD.&#xD;
&#xD;
      The trial includes a screening period of up to 8 weeks followed by a 96-week study period.&#xD;
&#xD;
      Subjects will be randomised to one of two arms: GT005 or the untreated control group.&#xD;
&#xD;
      Part 1 is conducted in a genetically selective sub-group of patients with GA secondary to&#xD;
      AMD.&#xD;
&#xD;
      Part 2 is conducted in a non-genetically selective sub-group of patients with GA secondary to&#xD;
      AMD.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Terminated for interim analysis demonstrating futility (trial highly unlikely to meet efficacy&#xD;
    outcome). The trial is not ending early because of medical problems or concerns.&#xD;
  </why_stopped>
  <start_date type="Actual">July 14, 2020</start_date>
  <completion_date type="Actual">August 24, 2023</completion_date>
  <primary_completion_date type="Actual">August 24, 2023</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <intervention_model_description>This is a Phase 2, outcomes assessor-masked multicentre, randomised study to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with GA secondary to AMD.</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
    <masking_description>The overall objectives of the study are to evaluate the safety and efficacy (anatomical and functional visual outcomes) of two doses of GT005 in genetically defined subjects with GA due to AMD.</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Progression of geographic atrophy</measure>
    <time_frame>48 weeks</time_frame>
    <description>The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Progression of geographic atrophy</measure>
    <time_frame>96 weeks</time_frame>
    <description>The change from baseline through Week 96 in GA area as measured by fundus autofluorescence (FAF)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluation of the safety and tolerability of GT005</measure>
    <time_frame>96 weeks</time_frame>
    <description>Frequency of treatment emergent adverse events (AEs) through Week 96</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluation of the effect of GT005 on retinal anatomical measures</measure>
    <time_frame>96 weeks</time_frame>
    <description>Change in retinal morphology on multimodal imaging through Week 96</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluation of the effect of GT005 on functional measures</measure>
    <time_frame>96 weeks</time_frame>
    <description>Change in BCVA Score via the early treatment for diabetic retinopathy (ETDRS) chart through Week 96</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluation of the effect of GT005 on functional measures</measure>
    <time_frame>96 weeks</time_frame>
    <description>Change in low luminance difference (LLD) via the ETDRS chart through Week 96</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluation of the effect of GT005 on visual function</measure>
    <time_frame>96 weeks</time_frame>
    <description>Change in reading performance as assessed by Minnesota low-vision reading test (MNRead) Chart through Week 96</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluation of the effect of GT005 on visual function</measure>
    <time_frame>96 weeks</time_frame>
    <description>Change in functional reading independence (FRI) index through Week 96</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluation of the effect of GT005 on patient-reported outcomes</measure>
    <time_frame>96 weeks</time_frame>
    <description>Change in quality of life measured on the Visual Functioning Questionnaire-25 (VFQ-25) through Week 96</description>
  </secondary_outcome>
  <number_of_arms>5</number_of_arms>
  <enrollment type="Actual">98</enrollment>
  <condition>Dry Age-related Macular Degeneration</condition>
  <arm_group>
    <arm_group_label>Part 1 - GT005 Low Dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Approximately 25 subjects are planned, with subjects randomised to GT005 Low Dose.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Part 1 - GT005 High Dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Approximately 25 subjects are planned, with subjects randomised to GT005 High Dose.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Part 1 - Untreated control</arm_group_label>
    <arm_group_type>No Intervention</arm_group_type>
    <description>Approximately 25 subjects are planned, with subjects randomised to untreated control.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Part 2 - GT005 Low Dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Approximately 116 subjects are planned, with subjects randomised to Part 2 - GT005 Low Dose.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Part 2 - Untreated control</arm_group_label>
    <arm_group_type>No Intervention</arm_group_type>
    <description>Approximately 54 subjects are planned, with subjects randomised to untreated control.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>GT005; Low Dose</intervention_name>
    <description>The study will test two doses of GT005: Low Dose and High Dose.</description>
    <arm_group_label>Part 1 - GT005 Low Dose</arm_group_label>
    <arm_group_label>Part 2 - GT005 Low Dose</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>GT005; High Dose</intervention_name>
    <description>The study will test two doses of GT005: Low Dose and High Dose.</description>
    <arm_group_label>Part 1 - GT005 High Dose</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Able and willing to give written informed consent&#xD;
&#xD;
          2. Age ≥55 years&#xD;
&#xD;
          3. Have a clinical diagnosis of GA secondary to AMD in the study eye, as determined by&#xD;
             the Investigator, and a diagnosis of AMD in the contralateral eye (except if the&#xD;
             subject is monocular)&#xD;
&#xD;
          4. Have GA lesion(s) total size between or equal to 1.25mm2 to 17.5mm2 in the study eye&#xD;
&#xD;
          5. The GA lesion(s) in the study eye must reside completely within the FAF image&#xD;
&#xD;
          6. Up to 25% of the enrolled study population are permitted to have CNV in the fellow&#xD;
             eye, defined as either:&#xD;
&#xD;
               1. Non-exudative/sub-clinical fellow eye CNV identified at Screening, or&#xD;
&#xD;
               2. Known history of fellow eye CNV with either ≥2 years since diagnosis or with no&#xD;
                  active treatment required in 6 months prior to Screening&#xD;
&#xD;
          7. Have a BCVA of 24 letters (6/95 and 20/320 Snellen acuity equivalent) or better, using&#xD;
             ETDRS charts, in the study eye&#xD;
&#xD;
          8. Part 1 Only: Subjects carrying a CFI rare variant genotype (minor allele frequency of&#xD;
             ≤1%) previously associated with low serum CFI or subjects carrying an unreported CFI&#xD;
             rare variant genotype that have tested to have a low serum CFI&#xD;
&#xD;
          9. Able to attend all study visits and complete the study procedures&#xD;
&#xD;
         10. Women of child-bearing potential must have a negative pregnancy test within 2 weeks&#xD;
             prior to randomisation. A pregnancy test is not required for postmenopausal women&#xD;
             (defined as being at least 12 consecutive months without menses) or those surgically&#xD;
             sterilised (those having a bilateral tubal ligation/bilateral salpingectomy, bilateral&#xD;
             tubal occlusive procedure, hysterectomy, or bilateral oophorectomy)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Subjects who have a clinical diagnosis of Stargardt Disease or other retinal&#xD;
             dystrophies, confirmed by the central reading centre&#xD;
&#xD;
          2. Have a history, or evidence, of CNV in the study eye&#xD;
&#xD;
          3. Presence of moderate/severe or worse non-proliferative diabetic retinopathy in the&#xD;
             study eye&#xD;
&#xD;
          4. Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in the&#xD;
             study eye&#xD;
&#xD;
          5. History of intraocular surgery in the study eye within 12 weeks prior to Screening&#xD;
             (Visit 1). Yttrium aluminium garnet capsulotomy is permitted if performed &gt;10 weeks&#xD;
             prior to Visit 1&#xD;
&#xD;
          6. Have clinically significant cataract that may require surgery during the study period&#xD;
             in the study eye&#xD;
&#xD;
          7. Presence of moderate to severe glaucomatous optic neuropathy in the study eye;&#xD;
             uncontrolled IOP despite the use of two or more topical agents; a history of&#xD;
             glaucoma-filtering or valve surgery is also excluded&#xD;
&#xD;
          8. Axial myopia of greater than -8 dioptres in the study eye&#xD;
&#xD;
          9. Have any other significant ocular or non-ocular medical or psychiatric condition&#xD;
             which, in the opinion of the Investigator, may either put the subject at risk or may&#xD;
             influence the results of the study&#xD;
&#xD;
         10. Have a contraindication to specified protocol corticosteroid regimen&#xD;
&#xD;
         11. Have received any investigational and/or approved product(s) for the treatment of GA&#xD;
             within the past 6 months, or 5 half-lives (whichever is longer) other than nutritional&#xD;
             supplements such as the age-related eye disease study (AREDS) formula in the study eye&#xD;
             or systemically&#xD;
&#xD;
         12. Have received a gene or cell therapy at any time&#xD;
&#xD;
         13. Are unwilling to use two forms of contraception (one of which being a barrier method)&#xD;
             for 90 days post-dosing, if relevant&#xD;
&#xD;
         14. Active malignancy within the past 12 months, except for: appropriately treated&#xD;
             carcinoma in situ of the cervix, non-melanoma skin carcinoma, or prostate cancer with&#xD;
             a stable prostate-specific antigen (PSA) ≥12 months&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>55 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Retinal Research Institute (retina consultants of AZ)</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85053</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Retina Associates of Southern California</name>
      <address>
        <city>Huntington Beach</city>
        <state>California</state>
        <zip>92647</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Byers Eye Institute at Stanford</name>
      <address>
        <city>Palo Alto</city>
        <state>California</state>
        <zip>94303</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Retina Consultants San Diego</name>
      <address>
        <city>Poway</city>
        <state>California</state>
        <zip>92064</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>VitreoRetinal Associates, P.A.</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32607</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Bascom Palmer Eye Institute</name>
      <address>
        <city>Miami</city>
        <state>Florida</state>
        <zip>33136</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Retina Vitreous Associates of Florida</name>
      <address>
        <city>Saint Petersburg</city>
        <state>Florida</state>
        <zip>33711</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Southeast Retina Center</name>
      <address>
        <city>Augusta</city>
        <state>Georgia</state>
        <zip>30909</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Retina Macula Associates PC</name>
      <address>
        <city>Lemont</city>
        <state>Illinois</state>
        <zip>60439</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Midwest Eye Institute Northside</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46290</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Wolfe Eye Clinic</name>
      <address>
        <city>West Des Moines</city>
        <state>Iowa</state>
        <zip>50266</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Retina Care Center</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21209</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ophthalmic Consultants of Boston (OCB)</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>VitreoRetinal Surgery, PLLC</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55435</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sierra Eye Associates</name>
      <address>
        <city>Reno</city>
        <state>Nevada</state>
        <zip>89502</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vision Research Center Eye Associates of New Mexico</name>
      <address>
        <city>Albuquerque</city>
        <state>New Mexico</state>
        <zip>87109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Columbia University Medical Center</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10032</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Retina Associates of Western New York</name>
      <address>
        <city>Rochester</city>
        <state>New York</state>
        <zip>14620</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cincinnati Eye Institute</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45242</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cleveland Clinic</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44195</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oregon Retina</name>
      <address>
        <city>Eugene</city>
        <state>Oregon</state>
        <zip>97401</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Casey Eye Institute</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Erie Retinal Surgery, INC</name>
      <address>
        <city>Erie</city>
        <state>Pennsylvania</state>
        <zip>16507</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mid Atlantic Retina</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19107</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Southeastern Retina Associates, PC</name>
      <address>
        <city>Knoxville</city>
        <state>Tennessee</state>
        <zip>37922</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Charles Retina Institute</name>
      <address>
        <city>Memphis</city>
        <state>Tennessee</state>
        <zip>38138</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Austin Research Center for Retina, PLLC</name>
      <address>
        <city>Austin</city>
        <state>Texas</state>
        <zip>78705</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Retina Consultants of Houston-TMC</name>
      <address>
        <city>Bellaire</city>
        <state>Texas</state>
        <zip>77401</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Texas Retina Associates</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75231</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Retinal Consultants of San Antonio</name>
      <address>
        <city>San Antonio</city>
        <state>Texas</state>
        <zip>78240</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Department of Ophthalmology UW Medicine</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98104-2499</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>West Virginia University</name>
      <address>
        <city>Morgantown</city>
        <state>West Virginia</state>
        <zip>26506</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The University of Melbourne - The Centre for Eye Research Australia (CERA)</name>
      <address>
        <city>Melbourne E.</city>
        <state>Victoria</state>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sydney Hospital and Sydney Eye Hospital</name>
      <address>
        <city>Sydney</city>
        <zip>2000</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre Paradis Monticelli</name>
      <address>
        <city>Marseille</city>
        <state>Alpes-Cote d'Azur</state>
        <zip>13008</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>CHU Hôpital F. Mitterrand</name>
      <address>
        <city>Dijon</city>
        <state>Bourgogne-Franche-Comté</state>
        <zip>21079</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>CHU de Nantes - Hôtel-Dieu</name>
      <address>
        <city>Nantes</city>
        <state>Pays De La Loire</state>
        <zip>44000</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitaetsklinikum Schleswig-Holstein Campus Lübeck</name>
      <address>
        <city>Lübeck</city>
        <state>Schleswig-Holstein</state>
        <zip>23538</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitaetsklinikum Bonn</name>
      <address>
        <city>Bonn</city>
        <zip>53127</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Internationale Innovative Ophthalmochirurgie</name>
      <address>
        <city>Düsseldorf</city>
        <zip>40549</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>St. Franziskus-Hospital</name>
      <address>
        <city>Münster</city>
        <zip>48145</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitatsklinikum Tübingen</name>
      <address>
        <city>Tübingen</city>
        <zip>72076</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Stichting Radboud Universitair Medisch Centrum</name>
      <address>
        <city>Nijmegen</city>
        <zip>6525 GA</zip>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oftalmika Spolka z ograniczona odpowiedzialnoscia</name>
      <address>
        <city>Bydgoszcz</city>
        <zip>85-631</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitari General de Catalunya</name>
      <address>
        <city>Sant Cugat Del Vallès</city>
        <state>Barcelona</state>
        <zip>08195</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinica Universidad de Navarra - Pamplona</name>
      <address>
        <city>Pamplona</city>
        <state>Navarra</state>
        <zip>31008</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital La Arruzafa</name>
      <address>
        <city>Córdoba</city>
        <zip>14012</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinica Baviera</name>
      <address>
        <city>Madrid</city>
        <zip>28046</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Clinica Oftalvist Valencia</name>
      <address>
        <city>Valencia</city>
        <zip>46100</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Bristol Eye Hospital</name>
      <address>
        <city>Bristol</city>
        <zip>BS1 2LX</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>St.Paul's Eye Unit</name>
      <address>
        <city>Liverpool</city>
        <zip>L7 8XP</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Moorfields Eye Hospital - NHS Foundation Trust</name>
      <address>
        <city>London</city>
        <zip>EC1V 2PD</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Retina Clinic London</name>
      <address>
        <city>London</city>
        <zip>W1G 7LB</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sheffield Teaching Hospitals NHS Foundation Trust</name>
      <address>
        <city>Sheffield</city>
        <zip>S10 2SB</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sunderland Eye Infirmary</name>
      <address>
        <city>Sunderland</city>
        <zip>SR2 9HP</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>France</country>
    <country>Germany</country>
    <country>Netherlands</country>
    <country>Poland</country>
    <country>Spain</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>September 2023</verification_date>
  <study_first_submitted>June 3, 2020</study_first_submitted>
  <study_first_submitted_qc>June 17, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 18, 2020</study_first_posted>
  <last_update_submitted>September 13, 2023</last_update_submitted>
  <last_update_submitted_qc>September 13, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">September 15, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Geographic atrophy</keyword>
  <keyword>Retinal disease</keyword>
  <keyword>Eye disease</keyword>
  <keyword>Retinal degeneration</keyword>
  <keyword>Macular atrophy</keyword>
  <keyword>Dry age-related macular degeneration</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Macular Degeneration</mesh_term>
    <mesh_term>Geographic Atrophy</mesh_term>
  </condition_browse>
  <!-- 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/NCT04437368
org study id: GT005-02
nct id: NCT04437368
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this clinical study is to evaluate the safety and efficacy of two doses of
      GT005 administered as a single subretinal injection in subjects with geographic atrophy
      secondary to age-related macular degeneration (AMD).
This is a Phase 2, outcomes assessor-masked, multicentre, randomised study to evaluate the
      safety and efficacy of two doses of GT005 administered as a single subretinal injection in
      subjects with GA secondary to AMD.

      The trial includes a screening period of up to 8 weeks followed by a 96-week study period.

      Subjects will be randomised to one of two arms: GT005 or the untreated control group.

      Part 1 is conducted in a genetically selective sub-group of patients with GA secondary to
      AMD.

      Part 2 is conducted in a non-genetically selective sub-group of patients with GA secondary to
      AMD.
allocation: Randomized
intervention model: Parallel Assignment
intervention model description: This is a Phase 2, outcomes assessor-masked multicentre, randomised study to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with GA secondary to AMD.
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
masking description: The overall objectives of the study are to evaluate the safety and efficacy (anatomical and functional visual outcomes) of two doses of GT005 in genetically defined subjects with GA due to AMD.
measure: Progression of geographic atrophy
time frame: 48 weeks
description: The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)
measure: Progression of geographic atrophy
time frame: 96 weeks
description: The change from baseline through Week 96 in GA area as measured by fundus autofluorescence (FAF)
measure: Evaluation of the safety and tolerability of GT005
time frame: 96 weeks
description: Frequency of treatment emergent adverse events (AEs) through Week 96
measure: Evaluation of the effect of GT005 on retinal anatomical measures
time frame: 96 weeks
description: Change in retinal morphology on multimodal imaging through Week 96
measure: Evaluation of the effect of GT005 on functional measures
time frame: 96 weeks
description: Change in BCVA Score via the early treatment for diabetic retinopathy (ETDRS) chart through Week 96
measure: Evaluation of the effect of GT005 on functional measures
time frame: 96 weeks
description: Change in low luminance difference (LLD) via the ETDRS chart through Week 96
measure: Evaluation of the effect of GT005 on visual function
time frame: 96 weeks
description: Change in reading performance as assessed by Minnesota low-vision reading test (MNRead) Chart through Week 96
measure: Evaluation of the effect of GT005 on visual function
time frame: 96 weeks
description: Change in functional reading independence (FRI) index through Week 96
measure: Evaluation of the effect of GT005 on patient-reported outcomes
time frame: 96 weeks
description: Change in quality of life measured on the Visual Functioning Questionnaire-25 (VFQ-25) through Week 96
arm group label: Part 1 - GT005 Low Dose
arm group type: Experimental
description: Approximately 25 subjects are planned, with subjects randomised to GT005 Low Dose.
arm group label: Part 1 - GT005 High Dose
arm group type: Experimental
description: Approximately 25 subjects are planned, with subjects randomised to GT005 High Dose.
arm group label: Part 1 - Untreated control
arm group type: No Intervention
description: Approximately 25 subjects are planned, with subjects randomised to untreated control.
arm group label: Part 2 - GT005 Low Dose
arm group type: Experimental
description: Approximately 116 subjects are planned, with subjects randomised to Part 2 - GT005 Low Dose.
arm group label: Part 2 - Untreated control
arm group type: No Intervention
description: Approximately 54 subjects are planned, with subjects randomised to untreated control.
intervention type: Drug
intervention name: GT005; Low Dose
description: The study will test two doses of GT005: Low Dose and High Dose.
arm group label: Part 1 - GT005 Low Dose
arm group label: Part 2 - GT005 Low Dose
intervention type: Drug
intervention name: GT005; High Dose
description: The study will test two doses of GT005: Low Dose and High Dose.
arm group label: Part 1 - GT005 High Dose
criteria: 
gender: All
minimum age: 55 Years
maximum age: N/A
healthy volunteers: No
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country: Australia
country: France
country: Germany
country: Netherlands
country: Poland
country: Spain
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Macular Degeneration
mesh term: Geographic Atrophy