<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/NCT03804021</url>
  </required_header>
  <id_info>
    <org_study_id>ADVM-043-03</org_study_id>
    <nct_id>NCT03804021</nct_id>
  </id_info>
  <brief_title>Long-Term Follow-up Study of ADVM-043</brief_title>
  <official_title>Post-treatment Long-term Follow-up Study of ADVM-043 Gene Therapy in Alpha-1 Antitrypsin Deficiency</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Adverum Biotechnologies, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Adverum Biotechnologies, Inc.</source>
  <oversight_info>
    <has_dmc>No</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>
      ADVM-043-03 is a long-term follow-up (LTFU) study of subjects who participated in the&#xD;
      ADVM-043-01 multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for&#xD;
      the treatment of Alpha-1 Antitrypsin (A1AT) deficiency.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      ADVM-043-03 was a multi-center, observational study without administration of the ADVM-043&#xD;
      investigational product. Subjects who participated in the ADVANCE study were enrolled into&#xD;
      this LTFU study for continued safety monitoring for a 2-year period.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">December 27, 2018</start_date>
  <completion_date type="Actual">December 6, 2021</completion_date>
  <primary_completion_date type="Actual">December 6, 2021</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Other</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Type, frequency, severity, duration, and relationship to ADVM-043 of adverse events of interest and of serious adverse events related to ADVM-043</measure>
    <time_frame>2 years</time_frame>
    <description>Safety was assessed by collection of severity and relatedness of Adverse Events to ADVM-043, Adverse Events of Interest and Serious Adverse Events related to ADVM-043</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">6</enrollment>
  <condition>Alpha-1 Antitrypsin Deficiency</condition>
  <arm_group>
    <arm_group_label>1</arm_group_label>
    <description>Patients who received ADVM-043 in a treatment protocol</description>
  </arm_group>
  <intervention>
    <intervention_type>Genetic</intervention_type>
    <intervention_name>ADVM-043</intervention_name>
    <description>Long term follow-up of subjects that received ADVM-043</description>
    <arm_group_label>1</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Patients who received ADVM-043 in a treatment protocol&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Key Inclusion Criteria:&#xD;
&#xD;
          -  The subject has A1AT deficiency and has previously received ADVM-043 gene therapy&#xD;
&#xD;
        Key Exclusion Criteria:&#xD;
&#xD;
          -  The subject is unwilling or unable to participate in all required study evaluations in&#xD;
             the long-term follow-up protocol&#xD;
&#xD;
          -  The subject is participating in another investigational treatment protocol&#xD;
&#xD;
          -  The subject is unable to understand the purpose and risks of the study or cannot&#xD;
             provide a signed and dated informed consent form (ICF)&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Charlton Strange, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Medical University of South Carolina, Charleston, SC, USA</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Mark Brantly, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Florida, Gainesville, FL, USA</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of Florida</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32610</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Medical University of South Carolina</name>
      <address>
        <city>Charleston</city>
        <state>South Carolina</state>
        <zip>29425</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>March 2022</verification_date>
  <study_first_submitted>January 11, 2019</study_first_submitted>
  <study_first_submitted_qc>January 11, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 15, 2019</study_first_posted>
  <last_update_submitted>March 16, 2022</last_update_submitted>
  <last_update_submitted_qc>March 16, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">March 31, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Alpha-1 Antitrypsin Deficiency</keyword>
  <keyword>alpha1-antitrypsin deficiency</keyword>
  <keyword>alpha 1-antitrypsin deficiency</keyword>
  <keyword>alpha1AT</keyword>
  <keyword>A1AT</keyword>
  <keyword>ADVM-043</keyword>
  <keyword>AAVrh.10halpha1AT</keyword>
  <keyword>AAVrh.10hA1AT</keyword>
  <keyword>Gene transfer vector</keyword>
  <keyword>Gene therapy</keyword>
  <keyword>Lung disease</keyword>
  <keyword>Emphysema</keyword>
  <keyword>COPD</keyword>
  <keyword>ADVANCE study</keyword>
  <keyword>Long Term Follow Up</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Alpha 1-Antitrypsin Deficiency</mesh_term>
  </condition_browse>
  <pending_results>
    <submitted>July 20, 2022</submitted>
    <returned>June 6, 2023</returned>
  </pending_results>
  <!-- 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/NCT03804021
org study id: ADVM-043-03
nct id: NCT03804021
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
ADVM-043-03 is a long-term follow-up (LTFU) study of subjects who participated in the
      ADVM-043-01 multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for
      the treatment of Alpha-1 Antitrypsin (A1AT) deficiency.
ADVM-043-03 was a multi-center, observational study without administration of the ADVM-043
      investigational product. Subjects who participated in the ADVANCE study were enrolled into
      this LTFU study for continued safety monitoring for a 2-year period.
observational model: Other
time perspective: Prospective
measure: Type, frequency, severity, duration, and relationship to ADVM-043 of adverse events of interest and of serious adverse events related to ADVM-043
time frame: 2 years
description: Safety was assessed by collection of severity and relatedness of Adverse Events to ADVM-043, Adverse Events of Interest and Serious Adverse Events related to ADVM-043
arm group label: 1
description: Patients who received ADVM-043 in a treatment protocol
intervention type: Genetic
intervention name: ADVM-043
description: Long term follow-up of subjects that received ADVM-043
arm group label: 1
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Charlton Strange, MD
role: Principal Investigator
affiliation: Medical University of South Carolina, Charleston, SC, USA
last name: Mark Brantly, MD
role: Principal Investigator
affiliation: University of Florida, Gainesville, FL, USA
facility: 
facility: 
country: United States
responsible party type: Sponsor
mesh term: Alpha 1-Antitrypsin Deficiency
submitted: July 20, 2022
returned: June 6, 2023