xml: <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/NCT06018558</url>
    </required_header>
    <id_info>
      <org_study_id>OCU410-101</org_study_id>
      <nct_id>NCT06018558</nct_id>
    </id_info>
    <brief_title>Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy</brief_title>
    <official_title>A Phase 1/2 Study to Assess the Safety And Efficacy Of OCU410 For Geographic Atrophy Secondary To Dry Age-Related Macular Degeneration</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Ocugen</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Ocugen</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>
        This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy&#xD;
        Secondary to Dry Age-Related Macular Degeneration (AMD).&#xD;
  &#xD;
        This is a multicenter study, which will be conducted in two phases and will enroll up to a&#xD;
        total of 63 subjects.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        Name of Sponsor/Company:&#xD;
  &#xD;
        Ocugen, Inc. 11 Great Valley Parkway Malvern, PA 19355&#xD;
  &#xD;
        Name of Investigational Product: OCU410&#xD;
  &#xD;
        Name of Active Ingredient:&#xD;
  &#xD;
        Adeno-associated viral vector 5 human RORA (AAV5-hRORA) Protocol Number: OCU410-101 Phase:&#xD;
        1/2 Country: US&#xD;
  &#xD;
        Title of Study:&#xD;
  &#xD;
        A Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy&#xD;
        Secondary to Dry Age-Related Macular Degeneration.&#xD;
  &#xD;
        Study Center(s): Approximately five clinical study centers in the US.&#xD;
  &#xD;
        Background:&#xD;
  &#xD;
        Age-related Macular Degeneration (AMD) is an ocular disease where macular degenerative&#xD;
        occurs. AMD manifests in two forms, Dry (nonexudative, atrophic) AMD and Wet (exudative,&#xD;
        neovascular) AMD. Geographic atrophy (GA) is an advanced stage of dry AMD that affects nearly&#xD;
        1 million people in the US and 5 million people worldwide, with its prevalence increasing&#xD;
        exponentially with age. It leads to progressive and irreversible loss of visual function due&#xD;
        to the growth of atrophic lesions that destroy the retinal cells responsible for vision.&#xD;
  &#xD;
        OCU410 Product Information:&#xD;
  &#xD;
        Ocugen, Inc., has developed a proprietary modifier gene therapy platform, OCU410, as the&#xD;
        second agent in a novel class of NHR-based gene modifier therapy for patients with dry AMD.&#xD;
        The proposed indication for OCU410 (AAV5-hRORA) is for the treatment of GA secondary to dry&#xD;
        AMD. The drug product is a sterile ophthalmic suspension for subretinal injection. OCU410&#xD;
        therapy regulates gene pathways contributing to GA by restoring homeostasis in the eye and&#xD;
        thereby serving as a therapeutic candidate for dry AMD. The modifier gene therapy platform is&#xD;
        a new way of addressing a genetic disease arising through a multitude of genetic mutations in&#xD;
        various genes but leading to the same end result (phenotype) of a diseased condition.&#xD;
  &#xD;
        This study will be conducted in two phases enrolling up to 63 subjects. Treated subjects will&#xD;
        receive a single subretinal injection of OCU410 in the study eye.&#xD;
  &#xD;
        Phase 1 is a multicenter, open-label, dose-ranging/dose-escalating study with a 3+3 design&#xD;
        enrolling up to 18 subjects.&#xD;
  &#xD;
        Phase 2 is a randomized dose-expansion cohort in which 45 subjects will be randomized in a&#xD;
        1:1:1 ratio in to one of the 2 treatment arms or the untreated control arm.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Recruiting</overall_status>
    <start_date type="Actual">August 23, 2023</start_date>
    <completion_date type="Anticipated">May 30, 2026</completion_date>
    <primary_completion_date type="Anticipated">March 30, 2026</primary_completion_date>
    <phase>Phase 1/Phase 2</phase>
    <study_type>Interventional</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <allocation>Randomized</allocation>
      <intervention_model>Sequential Assignment</intervention_model>
      <intervention_model_description>Study will be conducted in 2 phases:&#xD;
  Phase 1 will be 3+3 design will be used for sequential dose-escalation cohorts in which subjects will receive a single subretinal injection of OCU410 in the study eye.&#xD;
  Phase 2 will be a dose-expansion phase of the study, where the subjects will be randomized in 1:1:1</intervention_model_description>
      <primary_purpose>Treatment</primary_purpose>
      <masking>Single (Outcomes Assessor)</masking>
      <masking_description>The following team members will be masked:&#xD;
  Bio-Statistician, Data Programmer, Imaging Reading Center Team, Head of Clinical Development and Medical Affairs.</masking_description>
    </study_design_info>
    <primary_outcome>
      <measure>Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>The primary endpoint is safety, determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in anatomy of ocular structures using Slit Lamp Biomicroscopy</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>We will use Slit-lamp Biomicroscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in anatomy of ocular structures using Indirect ophthalmoscopy</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>We will use Indirect ophthalmoscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change from baseline in BCVA (Best Corrected Visual Acuity)</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>Visual function of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score. A higher score represents better vision.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in Low Luminance Visual Acuity</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>Measured by letter score. A higher score represents better vision</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Change in the Intraocular Pressure (mmHg)</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>Measured by applanation or rebound tonometry with confirmation with Goldmann tonometer if IOP is outside normal range (8-21mmHg).</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>Humoral and cellular immune response</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>Blood samples will be collected for the assessment. The secondary safety endpoints include change from baseline in Humoral and cellular immune response in response to OCU410 administration</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Shedding of viral vector</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>Blood samples will be collected for the assessment to determine AAV vector shedding in systemic circulation after OCU410 administration</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Laboratory parameters including serum chemistry and hematology</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>Blood samples will be collected for the assessment to determine a change from baseline after OCU410 administration.</description>
    </secondary_outcome>
    <other_outcome>
      <measure>Structural Outcome: Change Using Qualitative and quantitative assessments of autofluorescence pattern (FAF)</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>Changes in the intensity of FAF will be evaluated from the baseline measurements, to assess the loss of retinal layers.</description>
    </other_outcome>
    <other_outcome>
      <measure>Changes in National Eye Institute Visual Function Questionnaire 25 (NEI-VFQ25)</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>The National Eye Institute Visual Function Questionnaire 25 (NEI-VFQ25) questionnaires will be completed to assess the impact of vision on quality of subject's life.</description>
    </other_outcome>
    <other_outcome>
      <measure>Change From Baseline in Mean Threshold Sensitivity (MAIA)</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>Mean threshold sensitivity of all points will be determined to assess the macular functional response and determine GA progression.</description>
    </other_outcome>
    <other_outcome>
      <measure>Change from Baseline in drusen volume using SD-OCT</measure>
      <time_frame>12 months (Screening to 12 months post OCU410 administration)</time_frame>
      <description>Measurement of change in drusen volume will be determined using Spectral Domain OCT measurements.</description>
    </other_outcome>
    <number_of_arms>6</number_of_arms>
    <enrollment type="Anticipated">63</enrollment>
    <condition>Geographic Atrophy</condition>
    <arm_group>
      <arm_group_label>Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL):</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Low Dose (2.5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the low dose concentration.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL):</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Medium Dose (5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the medium dose concentration.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL):</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>High Dose (1.5×10E11 vg/mL): Subjects will receive a subretinal injection in the high dose concentration.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Maximum tolerated dose (MTD) from Phase 1: Subjects will receive a subretinal injection in the MTD concentration.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Subjects will receive a subretinal injection of OCU410 in a Lower Dose concentration.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Control Arm</arm_group_label>
      <arm_group_type>No Intervention</arm_group_type>
      <description>No Intervention Control Arm: Subject will not receive any active study intervention</description>
    </arm_group>
    <intervention>
      <intervention_type>Genetic</intervention_type>
      <intervention_name>OCU410</intervention_name>
      <description>Subretinal administration of OCU410</description>
      <arm_group_label>Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm</arm_group_label>
      <arm_group_label>Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm</arm_group_label>
      <arm_group_label>Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL):</arm_group_label>
      <arm_group_label>Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL):</arm_group_label>
      <arm_group_label>Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL):</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            1. Subjects 50 years of age or older.&#xD;
  &#xD;
            2. BCVA of approximately 24 letters or more using Early Treatment Diabetic Retinopathy&#xD;
               Study (ETDRS) chart (20/320 Snellen equivalent).&#xD;
  &#xD;
            3. Fundus autofluorescence (FAF) imaging shows:&#xD;
  &#xD;
                 1. Total GA area ≥2.5 and ≤17.5 mm2 (1 and 7 disk areas [DA], respectively)&#xD;
  &#xD;
                 2. If GA is multifocal, at least one focal lesion must be ≥1.25 mm2 (0.5 DA), with&#xD;
                    the overall aggregate area of GA&#xD;
  &#xD;
                 3. The entire GA lesion must be completely visualized on the macula-centered image&#xD;
                    and must be able to be imaged in its entirety, and not contiguous with any areas&#xD;
                    of peripapillary atrophy&#xD;
  &#xD;
                 4. Absence of any pattern of hyper-autofluorescence in the junctional zone of GA&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            1. Previous treatment with a gene-therapy or cell therapy product&#xD;
  &#xD;
            2. Previous treatment with any investigational drug or device within one year. The&#xD;
               history of any investigational product with a washout period of up to six months will&#xD;
               be evaluated on a case-by-case basis.&#xD;
  &#xD;
            3. Previous treatment with Syfovre (Pegcetacoplan injection)&#xD;
  &#xD;
            4. GA due to causes other than AMD such as Stargardt disease, cone rod dystrophy or toxic&#xD;
               maculopathies like Plaquenil maculopathy. however, benign conditions of the vitreous&#xD;
               or peripheral retina are not exclusionary (i.e., pavingstone degeneration).&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>50 Years</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Huma Qamar, MD, MPH, CMI</last_name>
      <role>Study Director</role>
      <affiliation>Ocugen</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Umair Qazi, MD, MPH</last_name>
      <phone>202 817 0787</phone>
      <email>umair.qazi@ocugen.com</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Roshan George, MD, MPH</last_name>
      <phone>(845) 664-1505</phone>
      <email>roshan.george@ocugen.com</email>
    </overall_contact_backup>
    <location>
      <facility>
        <name>Retina Consultants of Texas</name>
        <address>
          <city>Bellaire</city>
          <state>Texas</state>
          <zip>77401</zip>
          <country>United States</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Rebbecca Taing</last_name>
        <phone>800-833-5921</phone>
        <email>rebbecca.taing@retinaconsultantstexas.com</email>
      </contact>
      <investigator>
        <last_name>Charles Wykoff, M.D; Ph.D</last_name>
        <role>Principal Investigator</role>
      </investigator>
    </location>
    <location>
      <facility>
        <name>Retina Foundation of the Southwest</name>
        <address>
          <city>Dallas</city>
          <state>Texas</state>
          <zip>75231</zip>
          <country>United States</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Kimberly Cummings, CCRC</last_name>
        <phone>214-363-3911</phone>
        <phone_ext>128</phone_ext>
        <email>evasquez@retinafoundation.org</email>
      </contact>
      <investigator>
        <last_name>Karl Csaky, M.D; Ph.D</last_name>
        <role>Principal Investigator</role>
      </investigator>
    </location>
    <location_countries>
      <country>United States</country>
    </location_countries>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>June 30, 2023</study_first_submitted>
    <study_first_submitted_qc>August 25, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 30, 2023</study_first_posted>
    <last_update_submitted>August 25, 2023</last_update_submitted>
    <last_update_submitted_qc>August 25, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 30, 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>Geographic Atrophy</mesh_term>
      <mesh_term>Atrophy</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT06018558
  org study id: OCU410-101
  nct id: NCT06018558
  lead sponsor: 
  has dmc: Yes
  is fda regulated drug: Yes
  is fda regulated device: No
  This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy
        Secondary to Dry Age-Related Macular Degeneration (AMD).
  
        This is a multicenter study, which will be conducted in two phases and will enroll up to a
        total of 63 subjects.
  Name of Sponsor/Company:
  
        Ocugen, Inc. 11 Great Valley Parkway Malvern, PA 19355
  
        Name of Investigational Product: OCU410
  
        Name of Active Ingredient:
  
        Adeno-associated viral vector 5 human RORA (AAV5-hRORA) Protocol Number: OCU410-101 Phase:
        1/2 Country: US
  
        Title of Study:
  
        A Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy
        Secondary to Dry Age-Related Macular Degeneration.
  
        Study Center(s): Approximately five clinical study centers in the US.
  
        Background:
  
        Age-related Macular Degeneration (AMD) is an ocular disease where macular degenerative
        occurs. AMD manifests in two forms, Dry (nonexudative, atrophic) AMD and Wet (exudative,
        neovascular) AMD. Geographic atrophy (GA) is an advanced stage of dry AMD that affects nearly
        1 million people in the US and 5 million people worldwide, with its prevalence increasing
        exponentially with age. It leads to progressive and irreversible loss of visual function due
        to the growth of atrophic lesions that destroy the retinal cells responsible for vision.
  
        OCU410 Product Information:
  
        Ocugen, Inc., has developed a proprietary modifier gene therapy platform, OCU410, as the
        second agent in a novel class of NHR-based gene modifier therapy for patients with dry AMD.
        The proposed indication for OCU410 (AAV5-hRORA) is for the treatment of GA secondary to dry
        AMD. The drug product is a sterile ophthalmic suspension for subretinal injection. OCU410
        therapy regulates gene pathways contributing to GA by restoring homeostasis in the eye and
        thereby serving as a therapeutic candidate for dry AMD. The modifier gene therapy platform is
        a new way of addressing a genetic disease arising through a multitude of genetic mutations in
        various genes but leading to the same end result (phenotype) of a diseased condition.
  
        This study will be conducted in two phases enrolling up to 63 subjects. Treated subjects will
        receive a single subretinal injection of OCU410 in the study eye.
  
        Phase 1 is a multicenter, open-label, dose-ranging/dose-escalating study with a 3+3 design
        enrolling up to 18 subjects.
  
        Phase 2 is a randomized dose-expansion cohort in which 45 subjects will be randomized in a
        1:1:1 ratio in to one of the 2 treatment arms or the untreated control arm.
  allocation: Randomized
  intervention model: Sequential Assignment
  intervention model description: Study will be conducted in 2 phases:
  Phase 1 will be 3+3 design will be used for sequential dose-escalation cohorts in which subjects will receive a single subretinal injection of OCU410 in the study eye.
  Phase 2 will be a dose-expansion phase of the study, where the subjects will be randomized in 1:1:1
  primary purpose: Treatment
  masking: Single (Outcomes Assessor)
  masking description: The following team members will be masked:
  Bio-Statistician, Data Programmer, Imaging Reading Center Team, Head of Clinical Development and Medical Affairs.
  measure: Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: The primary endpoint is safety, determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
  measure: Change in anatomy of ocular structures using Slit Lamp Biomicroscopy
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: We will use Slit-lamp Biomicroscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.
  measure: Change in anatomy of ocular structures using Indirect ophthalmoscopy
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: We will use Indirect ophthalmoscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.
  measure: Change from baseline in BCVA (Best Corrected Visual Acuity)
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: Visual function of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score. A higher score represents better vision.
  measure: Change in Low Luminance Visual Acuity
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: Measured by letter score. A higher score represents better vision
  measure: Change in the Intraocular Pressure (mmHg)
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: Measured by applanation or rebound tonometry with confirmation with Goldmann tonometer if IOP is outside normal range (8-21mmHg).
  measure: Humoral and cellular immune response
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: Blood samples will be collected for the assessment. The secondary safety endpoints include change from baseline in Humoral and cellular immune response in response to OCU410 administration
  measure: Shedding of viral vector
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: Blood samples will be collected for the assessment to determine AAV vector shedding in systemic circulation after OCU410 administration
  measure: Laboratory parameters including serum chemistry and hematology
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: Blood samples will be collected for the assessment to determine a change from baseline after OCU410 administration.
  measure: Structural Outcome: Change Using Qualitative and quantitative assessments of autofluorescence pattern (FAF)
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: Changes in the intensity of FAF will be evaluated from the baseline measurements, to assess the loss of retinal layers.
  measure: Changes in National Eye Institute Visual Function Questionnaire 25 (NEI-VFQ25)
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: The National Eye Institute Visual Function Questionnaire 25 (NEI-VFQ25) questionnaires will be completed to assess the impact of vision on quality of subject's life.
  measure: Change From Baseline in Mean Threshold Sensitivity (MAIA)
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: Mean threshold sensitivity of all points will be determined to assess the macular functional response and determine GA progression.
  measure: Change from Baseline in drusen volume using SD-OCT
  time frame: 12 months (Screening to 12 months post OCU410 administration)
  description: Measurement of change in drusen volume will be determined using Spectral Domain OCT measurements.
  arm group label: Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL):
  arm group type: Experimental
  description: Low Dose (2.5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the low dose concentration.
  arm group label: Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL):
  arm group type: Experimental
  description: Medium Dose (5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the medium dose concentration.
  arm group label: Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL):
  arm group type: Experimental
  description: High Dose (1.5×10E11 vg/mL): Subjects will receive a subretinal injection in the high dose concentration.
  arm group label: Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm
  arm group type: Experimental
  description: Maximum tolerated dose (MTD) from Phase 1: Subjects will receive a subretinal injection in the MTD concentration.
  arm group label: Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm
  arm group type: Experimental
  description: Subjects will receive a subretinal injection of OCU410 in a Lower Dose concentration.
  arm group label: Control Arm
  arm group type: No Intervention
  description: No Intervention Control Arm: Subject will not receive any active study intervention
  intervention type: Genetic
  intervention name: OCU410
  description: Subretinal administration of OCU410
  arm group label: Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm
  arm group label: Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm
  arm group label: Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL):
  arm group label: Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL):
  arm group label: Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL):
  criteria: 
  gender: All
  minimum age: 50 Years
  maximum age: N/A
  healthy volunteers: No
  last name: Huma Qamar, MD, MPH, CMI
  role: Study Director
  affiliation: Ocugen
  last name: Umair Qazi, MD, MPH
  phone: 202 817 0787
  email: umair.qazi@ocugen.com
  last name: Roshan George, MD, MPH
  phone: (845) 664-1505
  email: roshan.george@ocugen.com
  facility: 
  status: Recruiting
  contact: 
  investigator: 
  facility: 
  status: Recruiting
  contact: 
  investigator: 
  country: United States
  responsible party type: Sponsor
  mesh term: Geographic Atrophy
  mesh term: Atrophy
  sharing ipd: No