<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/NCT03293524</url>
  </required_header>
  <id_info>
    <org_study_id>GS-LHON-CLIN-05</org_study_id>
    <secondary_id>2017-002187-40</secondary_id>
    <nct_id>NCT03293524</nct_id>
  </id_info>
  <brief_title>Efficacy &amp; Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year</brief_title>
  <acronym>REFLECT</acronym>
  <official_title>Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected With G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>GenSight Biologics</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>GenSight Biologics</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 goal of this clinical trial is to assess the safety and efficacy of GS010, a gene&#xD;
      therapy, in improving the retina functional &amp; structural outcomes in subjects with LHON due&#xD;
      to the G11778A ND4 mitochondrial mutation when vision loss duration is present up to one&#xD;
      year.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      GS-LHON-CLIN-05 is a Phase III, global, multi-center randomized, double-masked for the&#xD;
      primary analysis, placebo-controlled, clinical study. As LHON is a neurodegenerative disease,&#xD;
      the goal is to administer GS010 as soon as possible upon confirmation of the LHON diagnosis&#xD;
      and the causative mutation.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">March 12, 2018</start_date>
  <completion_date type="Anticipated">June 30, 2024</completion_date>
  <primary_completion_date type="Anticipated">June 30, 2024</primary_completion_date>
  <phase>Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>Yes</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Best-Corrected Visual Acuity (BCVA) reported using Log of the Minimal Angle of Resolution (LogMAR) - 1 year</measure>
    <time_frame>at 1.5 Year post baseline treatment</time_frame>
    <description>The primary efficacy endpoint will be the change from baseline (Visit 2) BCVA reported with LogMAR at 1.5 years post-treatment in second affected/not yet affected eyes of ND4 LHON subjects with vision loss up to one year. The change from baseline (Visit 2) in second affected/not yet affected eyes receiving GS010 and placebo will be the primary response of interest. LogMAR BCVA will be used to represent BCVA.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Best-Corrected Visual Acuity (BCVA) reported with LogMAR - 2 years</measure>
    <time_frame>at 1.5-Year and 2-Years post baseline treatment</time_frame>
    <description>Change from baseline in LogMAR BCVA at each timepoint of the follow-up period and at 2 years post-treatment.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Responder Analysis</measure>
    <time_frame>at 1.5-Year and 2-Years post baseline treatment</time_frame>
    <description>Response status at each timepoint of the follow-up period and at 2 years post-treatment. Definitions of responder eyes include:&#xD;
Eyes whose LogMAR BCVA improves (i.e. decreases) by ≥ 0.3 LogMAR (equivalent to a gain of ≥ 15 ETDRS letters) compared to baseline.&#xD;
Eyes whose LogMAR BCVA does not increase (i.e. worsen) by ≥ 0.3 LogMAR (equivalent to eyes that lose ≤ 15 ETDRS letters) compared to baseline.&#xD;
Eyes whose LogMAR visual acuity is &lt; 1.0 (i.e. better than LogMAR 1.0, equivalent to better than Snellen acuity of 20/200).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Spectral-Domain - Optical Coherence Tomography (SD-OCT) parameter</measure>
    <time_frame>at 1.5-Year and 2-Years post baseline treatment</time_frame>
    <description>Parameters measured with SD-OCT over time and at 1.5 and 2-years post baseline treatment. The change from baseline will be the response. Analysis will be performed mainly for second affected/not yet affected eyes treated with GS010 versus placebo and also for first affected eyes receiving GS010 as well</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Humphrey Visual Field (HVF) parameter</measure>
    <time_frame>at 1.5-Year and 2-Years post baseline treatment</time_frame>
    <description>Parameters measured with HVF 30-2 over time and at 1.5 and 2-years post baseline treatment. The change from baseline will be the response. Analysis will be performed mainly for second affected/not yet affected eyes treated with GS010 versus placebo and also for first affected eyes receiving GS010 as well</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pelli Robson Low Vision Contrast Sensitivity parameter</measure>
    <time_frame>at 1.5-Year and 2-Years post baseline treatment</time_frame>
    <description>Parameters measured with Pelli Robson Low Vision Contrast Sensitivity over time and at 1.5 and 2-years post baseline treatment. The change from baseline will be the response. Analysis will be performed mainly for second affected/not yet affected eyes treated with GS010 versus placebo and also for first affected eyes receiving GS010 as well</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Quality of Life: Visual Functioning Questionnaire-25</measure>
    <time_frame>at 1.5-Year and 2-Years post baseline treatment</time_frame>
    <description>Visual Functioning Questionnaire-25 at 1.5 and 2-years post-treatment</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Quality of Life: 36-Item Short Form Health Survey, version 2 Questionnaire</measure>
    <time_frame>at 1.5-Year and 2-Years post baseline treatment</time_frame>
    <description>36-Item Short Form Health Survey, version 2 Questionnaire at 1 and 2-years post-treatment.</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Adverse events (AEs) and serious adverse events (SAEs)</measure>
    <time_frame>up to 2-Years post baseline treatment</time_frame>
    <description>Adverse events (AEs) and serious adverse events (SAEs), including those that are treatment-emergent and non-treatment-emergent, throughout the study period and at each study visit. Incidence and severity of systemic and local (ocular) AEs and SAEs will be determined at each clinical site and for the entire study cohort.</description>
  </other_outcome>
  <other_outcome>
    <measure>Physical examinations</measure>
    <time_frame>At 2-Years post baseline treatment</time_frame>
    <description>Results of physical examinations</description>
  </other_outcome>
  <other_outcome>
    <measure>Electrocardiograms</measure>
    <time_frame>At 2-Years post baseline treatment</time_frame>
    <description>Results of Electrocardiograms (ECGs)</description>
  </other_outcome>
  <other_outcome>
    <measure>Laboratory results</measure>
    <time_frame>At 2-Years post baseline treatment</time_frame>
    <description>Results of laboratory tests from blood collection</description>
  </other_outcome>
  <other_outcome>
    <measure>Immune response evaluations</measure>
    <time_frame>Up to 2-Years post baseline treatment</time_frame>
    <description>Results of immune response evaluations&#xD;
Time course of the humoral immune response measured with Neutralizing Antibodies (Nab) against Adeno-associated virus vector 2 (AAV2)&#xD;
Time course of the cellular immune response against AAV2</description>
  </other_outcome>
  <other_outcome>
    <measure>Blood Bio-dissemination of AAV2 Vector DNA</measure>
    <time_frame>up to 4 weeks post-treatment</time_frame>
    <description>Results of bio-dissemination testing up to 4-weeks post-treatment</description>
  </other_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">90</enrollment>
  <condition>Leber Hereditary Optic Neuropathy</condition>
  <arm_group>
    <arm_group_label>Treatment Arm 1</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Subjects will be randomized to treatment arm 1 or treatment arm 2 in a 1:1 allocation. Subjects in treatment arm 1 will receive intravitreal GS010 in both eyes.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Treatment Arm 2</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Subjects will be randomized to treatment arm 1 or treatment arm 2 in a 1:1 allocation. Subjects in treatment arm 2 will receive GS010 in one eye and placebo intravitreal injection in the other eye.</description>
  </arm_group>
  <intervention>
    <intervention_type>Genetic</intervention_type>
    <intervention_name>GS010</intervention_name>
    <description>GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). GS010 will be administrated via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) as a single baseline intravitreal injection.</description>
    <arm_group_label>Treatment Arm 1</arm_group_label>
    <arm_group_label>Treatment Arm 2</arm_group_label>
    <other_name>Lenadogene nolparvovec</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>The placebo is a BSS, sterile, apyrogenic solution and used for ocular surgery. The placebo will be administered via intravitreal injection in a volume of 90 μL.</description>
    <arm_group_label>Treatment Arm 2</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Main Selection Criteria:&#xD;
&#xD;
          -  Age 15 years or older on the date of signed informed consent.&#xD;
&#xD;
          -  Clinically manifested vision loss due to ND4 LHON, to any extent, in at least one eye.&#xD;
&#xD;
          -  Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion&#xD;
             Visit (Visit 2).&#xD;
&#xD;
        Main Non-Selection Criteria:&#xD;
&#xD;
          -  Contraindication to intravitreal injection in any eye.&#xD;
&#xD;
          -  Subjects refusing to discontinue idebenone.&#xD;
&#xD;
          -  Previous vitrectomy in either eye.&#xD;
&#xD;
          -  Narrow angle in any eye contra-indicating pupillary dilation.&#xD;
&#xD;
          -  Presence of known/documented mutations, other than the G11778A ND4 LHON-causing&#xD;
             mutation, which are known to cause pathology of the optic nerve, retina or afferent&#xD;
             visual system.&#xD;
&#xD;
          -  History of recurrent uveitis (idiopathic or immune-related) or active ocular&#xD;
             inflammation.&#xD;
&#xD;
        Main Inclusion Criteria:&#xD;
&#xD;
          -  Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion&#xD;
             Visit (Visit 2).&#xD;
&#xD;
          -  Each eye of the subject must maintain at least Hand Motion (HM) visual acuity, as&#xD;
             defined by the study's SOP for visual acuity testing.&#xD;
&#xD;
          -  Documented results of genotyping showing the presence of the G11778A mutation in the&#xD;
             ND4 gene and the absence of the other primary LHON-associated mutations (ND1 or ND6)&#xD;
             in the subject's mitochondrial DNA.&#xD;
&#xD;
        Main Exclusion Criteria:&#xD;
&#xD;
          -  Light Perception (LP) or No Light Perception (NLP) visual acuity in any eye, as&#xD;
             defined by the study's standard operating procedure (SOP) for visual acuity testing.&#xD;
&#xD;
          -  Presence of active infectious conjunctivitis, keratitis, scleritis or endophthalmitis&#xD;
             in either eye.&#xD;
&#xD;
          -  Presence of alcoholism, alcohol dependence, or alcohol or drug abuse (excluding&#xD;
             nicotine).&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>15 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Nancy Newman, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Emory University Hospital Atlanta, Georgia, United States, 30322</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Doheny Eye Center UCLA Pasadena</name>
      <address>
        <city>Pasadena</city>
        <state>California</state>
        <zip>91105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Colorado Health Eye Center</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Emory Healthcare - The Emory Clinic</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Massachusetts Eye and Ear Infirmary</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Icahn School of Medicine at Mount Sinai</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10029</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Wills Eye Institute - Ocular Oncology Service</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19107</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vanderbilt Eye Institute</name>
      <address>
        <city>Nashville</city>
        <state>Tennessee</state>
        <zip>37232</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitair Ziekenhuis Gent</name>
      <address>
        <city>Gent</city>
        <zip>9000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>CHNO Les Quinze Vingts</name>
      <address>
        <city>Paris</city>
        <zip>75012</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IRCCS Istituto delle Scienze Neurologiche di Bologna UOC Clinica Neurologica</name>
      <address>
        <city>Bologna</city>
        <zip>40139</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Ramon y Cajal</name>
      <address>
        <city>Madrid</city>
        <zip>28034</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Taipei Veterans General Hospital</name>
      <address>
        <city>Taipei</city>
        <zip>11217</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Moorfields Eye Hospital</name>
      <address>
        <city>London</city>
        <state>Greater London</state>
        <zip>EC1V 2PD</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Belgium</country>
    <country>France</country>
    <country>Italy</country>
    <country>Spain</country>
    <country>Taiwan</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://www.gensight-biologics.com</url>
    <description>GenSight Biologics website</description>
  </link>
  <verification_date>August 2022</verification_date>
  <study_first_submitted>September 19, 2017</study_first_submitted>
  <study_first_submitted_qc>September 21, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 26, 2017</study_first_posted>
  <last_update_submitted>August 2, 2022</last_update_submitted>
  <last_update_submitted_qc>August 2, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">August 3, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Heredity Optic Atrophy</keyword>
  <keyword>Leber Hereditary Optic Atrophy</keyword>
  <keyword>Leber Hereditary Optic Neuropathy</keyword>
  <keyword>LHON</keyword>
  <keyword>Eye Diseases</keyword>
  <keyword>Hereditary Eye Diseases</keyword>
  <keyword>Inherited retinal dystrophies or degeneration</keyword>
  <keyword>Inborn Genetic Disease</keyword>
  <keyword>Gene Therapy</keyword>
  <keyword>Intravitreal Injections</keyword>
  <keyword>Mitochondrial Disease</keyword>
  <keyword>AAV2 Vectors</keyword>
  <keyword>Nervous System Diseases</keyword>
  <keyword>Neurodegenerative Disease</keyword>
  <keyword>Heredodegenerative Disorders of the Nervous System</keyword>
  <keyword>Atrophy</keyword>
  <keyword>Pathological Conditions, Anatomical</keyword>
  <keyword>Anesthetics</keyword>
  <keyword>Central Nervous System Depressants</keyword>
  <keyword>Physiological Effects of Drugs</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Optic Nerve Diseases</mesh_term>
    <mesh_term>Optic Atrophy, Hereditary, Leber</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/NCT03293524
org study id: GS-LHON-CLIN-05
secondary id: 2017-002187-40
nct id: NCT03293524
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The goal of this clinical trial is to assess the safety and efficacy of GS010, a gene
      therapy, in improving the retina functional & structural outcomes in subjects with LHON due
      to the G11778A ND4 mitochondrial mutation when vision loss duration is present up to one
      year.
GS-LHON-CLIN-05 is a Phase III, global, multi-center randomized, double-masked for the
      primary analysis, placebo-controlled, clinical study. As LHON is a neurodegenerative disease,
      the goal is to administer GS010 as soon as possible upon confirmation of the LHON diagnosis
      and the causative mutation.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: Best-Corrected Visual Acuity (BCVA) reported using Log of the Minimal Angle of Resolution (LogMAR) - 1 year
time frame: at 1.5 Year post baseline treatment
description: The primary efficacy endpoint will be the change from baseline (Visit 2) BCVA reported with LogMAR at 1.5 years post-treatment in second affected/not yet affected eyes of ND4 LHON subjects with vision loss up to one year. The change from baseline (Visit 2) in second affected/not yet affected eyes receiving GS010 and placebo will be the primary response of interest. LogMAR BCVA will be used to represent BCVA.
measure: Best-Corrected Visual Acuity (BCVA) reported with LogMAR - 2 years
time frame: at 1.5-Year and 2-Years post baseline treatment
description: Change from baseline in LogMAR BCVA at each timepoint of the follow-up period and at 2 years post-treatment.
measure: Responder Analysis
time frame: at 1.5-Year and 2-Years post baseline treatment
description: Response status at each timepoint of the follow-up period and at 2 years post-treatment. Definitions of responder eyes include:
Eyes whose LogMAR BCVA improves (i.e. decreases) by ≥ 0.3 LogMAR (equivalent to a gain of ≥ 15 ETDRS letters) compared to baseline.
Eyes whose LogMAR BCVA does not increase (i.e. worsen) by ≥ 0.3 LogMAR (equivalent to eyes that lose ≤ 15 ETDRS letters) compared to baseline.
Eyes whose LogMAR visual acuity is < 1.0 (i.e. better than LogMAR 1.0, equivalent to better than Snellen acuity of 20/200).
measure: Spectral-Domain - Optical Coherence Tomography (SD-OCT) parameter
time frame: at 1.5-Year and 2-Years post baseline treatment
description: Parameters measured with SD-OCT over time and at 1.5 and 2-years post baseline treatment. The change from baseline will be the response. Analysis will be performed mainly for second affected/not yet affected eyes treated with GS010 versus placebo and also for first affected eyes receiving GS010 as well
measure: Humphrey Visual Field (HVF) parameter
time frame: at 1.5-Year and 2-Years post baseline treatment
description: Parameters measured with HVF 30-2 over time and at 1.5 and 2-years post baseline treatment. The change from baseline will be the response. Analysis will be performed mainly for second affected/not yet affected eyes treated with GS010 versus placebo and also for first affected eyes receiving GS010 as well
measure: Pelli Robson Low Vision Contrast Sensitivity parameter
time frame: at 1.5-Year and 2-Years post baseline treatment
description: Parameters measured with Pelli Robson Low Vision Contrast Sensitivity over time and at 1.5 and 2-years post baseline treatment. The change from baseline will be the response. Analysis will be performed mainly for second affected/not yet affected eyes treated with GS010 versus placebo and also for first affected eyes receiving GS010 as well
measure: Quality of Life: Visual Functioning Questionnaire-25
time frame: at 1.5-Year and 2-Years post baseline treatment
description: Visual Functioning Questionnaire-25 at 1.5 and 2-years post-treatment
measure: Quality of Life: 36-Item Short Form Health Survey, version 2 Questionnaire
time frame: at 1.5-Year and 2-Years post baseline treatment
description: 36-Item Short Form Health Survey, version 2 Questionnaire at 1 and 2-years post-treatment.
measure: Adverse events (AEs) and serious adverse events (SAEs)
time frame: up to 2-Years post baseline treatment
description: Adverse events (AEs) and serious adverse events (SAEs), including those that are treatment-emergent and non-treatment-emergent, throughout the study period and at each study visit. Incidence and severity of systemic and local (ocular) AEs and SAEs will be determined at each clinical site and for the entire study cohort.
measure: Physical examinations
time frame: At 2-Years post baseline treatment
description: Results of physical examinations
measure: Electrocardiograms
time frame: At 2-Years post baseline treatment
description: Results of Electrocardiograms (ECGs)
measure: Laboratory results
time frame: At 2-Years post baseline treatment
description: Results of laboratory tests from blood collection
measure: Immune response evaluations
time frame: Up to 2-Years post baseline treatment
description: Results of immune response evaluations
Time course of the humoral immune response measured with Neutralizing Antibodies (Nab) against Adeno-associated virus vector 2 (AAV2)
Time course of the cellular immune response against AAV2
measure: Blood Bio-dissemination of AAV2 Vector DNA
time frame: up to 4 weeks post-treatment
description: Results of bio-dissemination testing up to 4-weeks post-treatment
arm group label: Treatment Arm 1
arm group type: Experimental
description: Subjects will be randomized to treatment arm 1 or treatment arm 2 in a 1:1 allocation. Subjects in treatment arm 1 will receive intravitreal GS010 in both eyes.
arm group label: Treatment Arm 2
arm group type: Placebo Comparator
description: Subjects will be randomized to treatment arm 1 or treatment arm 2 in a 1:1 allocation. Subjects in treatment arm 2 will receive GS010 in one eye and placebo intravitreal injection in the other eye.
intervention type: Genetic
intervention name: GS010
description: GS010 is a recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4). GS010 will be administrated via intravitreal injection containing 9E10 viral genomes in 90μL balanced salt solution (BSS) as a single baseline intravitreal injection.
arm group label: Treatment Arm 1
arm group label: Treatment Arm 2
other name: Lenadogene nolparvovec
intervention type: Drug
intervention name: Placebo
description: The placebo is a BSS, sterile, apyrogenic solution and used for ocular surgery. The placebo will be administered via intravitreal injection in a volume of 90 μL.
arm group label: Treatment Arm 2
criteria: 
gender: All
minimum age: 15 Years
maximum age: N/A
healthy volunteers: No
last name: Nancy Newman, MD
role: Principal Investigator
affiliation: Emory University Hospital Atlanta, Georgia, United States, 30322
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Belgium
country: France
country: Italy
country: Spain
country: Taiwan
country: United Kingdom
country: United States
url: http://www.gensight-biologics.com
description: GenSight Biologics website
responsible party type: Sponsor
mesh term: Optic Nerve Diseases
mesh term: Optic Atrophy, Hereditary, Leber