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/NCT05973630</url>
    </required_header>
    <id_info>
      <org_study_id>ATA-003-GSAR</org_study_id>
      <nct_id>NCT05973630</nct_id>
    </id_info>
    <brief_title>ATA-200 Dose-escalation Gene Therapy Trial in Patients With LGMDR5</brief_title>
    <official_title>A Phase 1-2, Open-label, Dose Escalation Study to Evaluate the Safety of 2 Doses of Intravenous ATA-200, an Adeno-associated Viral Vector Carrying the Human Gamma-sarcoglycan Gene, in Patients With Gamma-sarcoglycanopathy (Limb-girdle Muscular Dystrophy LGMDR5)</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Atamyo Therapeutics</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Atamyo Therapeutics</source>
    <oversight_info>
      <has_dmc>Yes</has_dmc>
      <is_fda_regulated_drug>No</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
    </oversight_info>
    <brief_summary>
      <textblock>
        The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single&#xD;
        intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy&#xD;
        type 2c/R5 (LGMD R5). Patients will be treated sequentially in 2 dose-cohorts.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        This is a multicenter Phase 1b assessing the safety and tolerability of 2 doses of ATA-200&#xD;
        for the treatment of LGMDR5.&#xD;
  &#xD;
        The dose escalation phase will enroll ambulant patients with LGMDR5. Two dose cohorts (C1)&#xD;
        and (C2) will be enrolled sequentially and enrollment will be staggered with at least 4-week&#xD;
        interval between 2 patient treatments. An initial cohort C1 of three (3) patients will&#xD;
        receive a potentially effective dose corresponding to the minimum effective dose (MED) in&#xD;
        preclinical studies.&#xD;
  &#xD;
        Enrollment of three (3) patients in the 2nd higher dose cohort C2 (with a 7-fold safety&#xD;
        margin relative to the highest safe dose in the GLP toxicology study) will be initiated&#xD;
        following review of the one-month safety data post-administration in cohort C1 by an&#xD;
        independent Data Safety Monitoring Board (DSMB).&#xD;
  &#xD;
        Time point of primary interest for safety evaluation and dose selection for future studies is&#xD;
        at 6 months.&#xD;
  &#xD;
        All subjects will be followed up for an additional 4.5 years after completion of the&#xD;
        evaluation period.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">January 1, 2024</start_date>
    <completion_date type="Anticipated">June 30, 2030</completion_date>
    <primary_completion_date type="Anticipated">December 31, 2025</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>Non-Randomized</allocation>
      <intervention_model>Sequential Assignment</intervention_model>
      <intervention_model_description>Dose escalation study</intervention_model_description>
      <primary_purpose>Treatment</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Incidence of adverse events</measure>
      <time_frame>0-6 months</time_frame>
      <description>Collection of adverse events at each visit</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Incidence of treatment-emergent adverse events,</measure>
      <time_frame>0-6 months</time_frame>
      <description>Collection of adverse events at each visit</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Incidence of serious adverse events</measure>
      <time_frame>0-6 months</time_frame>
      <description>Collection of adverse events at each visits</description>
    </primary_outcome>
    <number_of_arms>2</number_of_arms>
    <enrollment type="Anticipated">6</enrollment>
    <condition>LGMD2C</condition>
    <arm_group>
      <arm_group_label>Cohort 1</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>ATA-200 Dose level 1: 1.0E+14 vg/Kg, solution for injection, single IV infusion over 2h</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Cohort 2</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>ATA-200 Dose level 2: 3.0E+14 vg/Kg, solution for injection, single IV infusion over 2h</description>
    </arm_group>
    <intervention>
      <intervention_type>Biological</intervention_type>
      <intervention_name>ATA-200</intervention_name>
      <description>single intravenous infusion</description>
      <arm_group_label>Cohort 1</arm_group_label>
      <arm_group_label>Cohort 2</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Confirmed diagnosis of LGMDR5 before age of 10, based on clinical presentation and&#xD;
               genotyping&#xD;
  &#xD;
            -  Ambulant male or female patients aged 6 to less than 12 years of age at screening&#xD;
  &#xD;
            -  Able to perform the 10-meter walk test (10MWT) in less than 15 sec and to rise from&#xD;
               chair with or without arm support&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Detectable neutralizing antibodies against AAV8&#xD;
  &#xD;
            -  Cardiomyopathy with left ventricular ejection fraction (LVEF) &lt; 50%&#xD;
  &#xD;
            -  Respiratory assistance&#xD;
  &#xD;
            -  Concomitant medical condition that might interfere with LGMDR5 evolution&#xD;
  &#xD;
            -  Acute illness within 4 weeks of anticipated IMP administration&#xD;
  &#xD;
            -  Current participation in another clinical trial with investigational medicinal product&#xD;
  &#xD;
            -  Previous participation in gene and cell therapy trials&#xD;
  &#xD;
            -  Any condition that would contraindicate immunosuppressant treatment&#xD;
  &#xD;
            -  Presence of any permanent items (e.g., metal braces) precluding undergoing MRI&#xD;
  &#xD;
            -  Any vaccination 1 month prior to planned IMP administration&#xD;
  &#xD;
            -  Serology consistent with HIV exposure or active hepatitis B or C infection&#xD;
  &#xD;
            -  Grade 2 or higher lab abnormalities for liver function tests, creatinine, hemogram and&#xD;
               coagulation&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>6 Years</minimum_age>
      <maximum_age>12 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <verification_date>July 2023</verification_date>
    <study_first_submitted>July 24, 2023</study_first_submitted>
    <study_first_submitted_qc>August 1, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 3, 2023</study_first_posted>
    <last_update_submitted>August 1, 2023</last_update_submitted>
    <last_update_submitted_qc>August 1, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 3, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <!-- 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/NCT05973630
  org study id: ATA-003-GSAR
  nct id: NCT05973630
  lead sponsor: 
  has dmc: Yes
  is fda regulated drug: No
  is fda regulated device: No
  The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single
        intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy
        type 2c/R5 (LGMD R5). Patients will be treated sequentially in 2 dose-cohorts.
  This is a multicenter Phase 1b assessing the safety and tolerability of 2 doses of ATA-200
        for the treatment of LGMDR5.
  
        The dose escalation phase will enroll ambulant patients with LGMDR5. Two dose cohorts (C1)
        and (C2) will be enrolled sequentially and enrollment will be staggered with at least 4-week
        interval between 2 patient treatments. An initial cohort C1 of three (3) patients will
        receive a potentially effective dose corresponding to the minimum effective dose (MED) in
        preclinical studies.
  
        Enrollment of three (3) patients in the 2nd higher dose cohort C2 (with a 7-fold safety
        margin relative to the highest safe dose in the GLP toxicology study) will be initiated
        following review of the one-month safety data post-administration in cohort C1 by an
        independent Data Safety Monitoring Board (DSMB).
  
        Time point of primary interest for safety evaluation and dose selection for future studies is
        at 6 months.
  
        All subjects will be followed up for an additional 4.5 years after completion of the
        evaluation period.
  allocation: Non-Randomized
  intervention model: Sequential Assignment
  intervention model description: Dose escalation study
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Incidence of adverse events
  time frame: 0-6 months
  description: Collection of adverse events at each visit
  measure: Incidence of treatment-emergent adverse events,
  time frame: 0-6 months
  description: Collection of adverse events at each visit
  measure: Incidence of serious adverse events
  time frame: 0-6 months
  description: Collection of adverse events at each visits
  arm group label: Cohort 1
  arm group type: Experimental
  description: ATA-200 Dose level 1: 1.0E+14 vg/Kg, solution for injection, single IV infusion over 2h
  arm group label: Cohort 2
  arm group type: Experimental
  description: ATA-200 Dose level 2: 3.0E+14 vg/Kg, solution for injection, single IV infusion over 2h
  intervention type: Biological
  intervention name: ATA-200
  description: single intravenous infusion
  arm group label: Cohort 1
  arm group label: Cohort 2
  criteria: 
  gender: All
  minimum age: 6 Years
  maximum age: 12 Years
  healthy volunteers: No
  responsible party type: Sponsor