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/NCT06024057</url>
    </required_header>
    <id_info>
      <org_study_id>SHGH-LX101-EX</org_study_id>
      <nct_id>NCT06024057</nct_id>
    </id_info>
    <brief_title>An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA</brief_title>
    <official_title>An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy (LX101) in Patients With Leber's Congenital Amaurosis (LCA)</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine</source>
    <oversight_info>
      <is_fda_regulated_drug>No</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
    </oversight_info>
    <brief_summary>
      <textblock>
        To evaluate the scaling clinical trail of AAV2-RPE65 gene therapy agent (LX101) in patients&#xD;
        with congenital amaurosis (LCA).&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        This study evaluated the overall safety and initial efficacy of RPE65 mutant congenital&#xD;
        amaurosis (RPE65-LCA) in subjects treated with a single, subretinal injection of LX101&#xD;
        (containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65) in the second&#xD;
        eye 1 year after treatment.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">September 1, 2023</start_date>
    <completion_date type="Anticipated">September 30, 2038</completion_date>
    <primary_completion_date type="Anticipated">September 30, 2028</primary_completion_date>
    <phase>N/A</phase>
    <study_type>Interventional</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <allocation>N/A</allocation>
      <intervention_model>Single Group Assignment</intervention_model>
      <primary_purpose>Treatment</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Local and systemic safety assessment</measure>
      <time_frame>52（+4）weeks</time_frame>
      <description>Eye and non-eye adverse events</description>
    </primary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">3</enrollment>
    <condition>To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)</condition>
    <arm_group>
      <arm_group_label>LX101</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
    </arm_group>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>LX101</intervention_name>
      <description>LX101 (Containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65 encoding human RPE65 gene).Specification: 0.2mL/bottle.Route of administration: subretinal injection, injection volume 0.3 mL/ eye</description>
      <arm_group_label>LX101</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  1) Participants in the main study &quot;Exploratory clinical study evaluating AAV2-RPE65&#xD;
               gene therapy formulation (LX101) in patients with congenital amaurosis (LCA) 2)&#xD;
               Willingness to participate in the extended study, subject and/or their guardian&#xD;
               signing a written informed consent and complying with a long-term follow-up protocol&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Have any of the following eye conditions:&#xD;
  &#xD;
                 1. Subjects with SAE in the &quot;Exploratory clinical Study Evaluating AAV2-RPE65 gene&#xD;
                    therapy agent (LX101) in patients with congenital Amaurosis (LCA)&#xD;
  &#xD;
                 2. pre-existing eye conditions (such as glaucoma, cornea, or severe lens opacity)&#xD;
                    that the investigator determines may interfere with planned surgery or with&#xD;
                    interpretation of the study endpoint;&#xD;
  &#xD;
                 3. Screening subjects who had undergone any internal eye surgery in the six months&#xD;
                    prior to the visit;&#xD;
  &#xD;
                 4. Inability or unwillingness to meet the requirements of the study;&#xD;
  &#xD;
          Have any of the following systemic conditions:&#xD;
  &#xD;
          6) Known allergy to the drug planned for use in the study; 7) Have a history of surgical&#xD;
          operations within 1 month before enrollment, and/or present unhealed wounds, ulcers,&#xD;
          fractures, etc.; Present infectious disease requiring oral, intramuscular or intravenous&#xD;
          administration; 8) Baseline laboratory values for complex systemic diseases or clinically&#xD;
          significant abnormalities; 9) Complex systemic diseases include diseases in which the&#xD;
          disease itself or the treatment of the disease can alter eye function. For example, the&#xD;
          treatment of malignant tumors can affect central nervous system function (e.g., orbital&#xD;
          radiation therapy; Leukemia with central/optic nerve involvement); 10) Subjects with a&#xD;
          history of immune-damaging diseases due to the possibility of opportunistic infection&#xD;
          (e.g., CMV retinitis); Subjects with diabetes or sickle cell disease will be excluded if&#xD;
          they have any manifestations of advanced retinopathy, such as macular edema or&#xD;
          proliferative changes. Subjects with juvenile rheumatoid arthritis have an increased risk&#xD;
          of infection due to poor postoperative wound healing; 11) Participants who cannot&#xD;
          discontinue Viagra (Sildenafil) or related compounds used to treat erectile dysfunction&#xD;
          during the study period; 12) Subjects who cannot discontinue hydroxychloroquine,&#xD;
          Chloroquine, meralide, or any related retinal toxic compound during the study; 13) Any&#xD;
          other conditions in which the potential subject is not allowed to complete a follow-up&#xD;
          examination during the study, and the investigator believes that there are conditions that&#xD;
          make the potential subject unfit for the study.&#xD;
  &#xD;
          Fertile subjects with any of the following conditions:&#xD;
  &#xD;
          14) Those who do not use effective contraceptive measures; 15) Pregnant and lactating&#xD;
          women.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>18 Years</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 29, 2023</study_first_submitted>
    <study_first_submitted_qc>August 29, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">September 5, 2023</study_first_posted>
    <last_update_submitted>August 29, 2023</last_update_submitted>
    <last_update_submitted_qc>August 29, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">September 5, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Principal Investigator</responsible_party_type>
      <investigator_affiliation>Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine</investigator_affiliation>
      <investigator_full_name>Xiaodong Sun</investigator_full_name>
      <investigator_title>Assessor</investigator_title>
    </responsible_party>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Blindness</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/NCT06024057
  org study id: SHGH-LX101-EX
  nct id: NCT06024057
  lead sponsor: 
  is fda regulated drug: No
  is fda regulated device: No
  To evaluate the scaling clinical trail of AAV2-RPE65 gene therapy agent (LX101) in patients
        with congenital amaurosis (LCA).
  This study evaluated the overall safety and initial efficacy of RPE65 mutant congenital
        amaurosis (RPE65-LCA) in subjects treated with a single, subretinal injection of LX101
        (containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65) in the second
        eye 1 year after treatment.
  allocation: N/A
  intervention model: Single Group Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Local and systemic safety assessment
  time frame: 52（+4）weeks
  description: Eye and non-eye adverse events
  arm group label: LX101
  arm group type: Experimental
  intervention type: Drug
  intervention name: LX101
  description: LX101 (Containing recombinant human adeno-associated virus serotype 2, AAV2-RPE65 encoding human RPE65 gene).Specification: 0.2mL/bottle.Route of administration: subretinal injection, injection volume 0.3 mL/ eye
  arm group label: LX101
  criteria: 
  gender: All
  minimum age: 18 Years
  maximum age: N/A
  healthy volunteers: No
  responsible party type: Principal Investigator
  investigator affiliation: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
  investigator full name: Xiaodong Sun
  investigator title: Assessor
  mesh term: Blindness
  sharing ipd: No