<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/NCT03285126</url>
  </required_header>
  <id_info>
    <org_study_id>Pro00085445</org_study_id>
    <nct_id>NCT03285126</nct_id>
  </id_info>
  <brief_title>Pompe Gene Therapy- Screening for Eligibility</brief_title>
  <official_title>Screening for Eligibility to Participate in a Clinical Trial of Gene Therapy for Late-onset Pompe Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Duke University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Duke University</source>
  <oversight_info>
    <has_dmc>No</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 this study is to determine eligibility for the future clinical trial of gene&#xD;
      therapy in adults with late-onset Pompe disease.&#xD;
&#xD;
      This screening protocol will enroll up to 20 adults patients (&gt;=18 yo) with late-onset Pompe&#xD;
      disease. Study assessments include review of medical history, vital signs, physical&#xD;
      examination, muscle function testing, lung function testing, blood and urine collection,&#xD;
      serum pregnancy test and ECG.&#xD;
&#xD;
      The study results will be collected to perform descriptive statistical analysis and used to&#xD;
      determine eligibility for the proposed clinical trial of gene therapy in the future.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">November 1, 2017</start_date>
  <completion_date type="Actual">June 29, 2019</completion_date>
  <primary_completion_date type="Actual">June 29, 2019</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Eligibility for gene therapy.</measure>
    <time_frame>Up to 2 days</time_frame>
    <description>The purpose of this study is to determine eligibility for the proposed clinical trial of gene therapy in adults with late-onset Pompe disease. Eligibility is measure by a composite score of blood tests, Electrocardiogram, Pulmonary Function Tests, and Muscle Status Testing.</description>
  </primary_outcome>
  <enrollment type="Actual">19</enrollment>
  <condition>Pompe Disease (Late-onset)</condition>
  <eligibility>
    <study_pop>
      <textblock>
        Adults with late on-set Pompe Disease.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria&#xD;
&#xD;
          -  Diagnosis of Pompe disease by blood or skin fibroblast GAA assay and two pathogenic&#xD;
             variants in the GAA gene.&#xD;
&#xD;
          -  Age: at least 18 years at enrollment.&#xD;
&#xD;
          -  Subjects are capable of giving written consent.&#xD;
&#xD;
          -  Receiving ERT at a stable dose for at least 104 weeks.&#xD;
&#xD;
        Exclusion Criteria&#xD;
&#xD;
          -  Any condition that would interfere with participation in the study as determined by&#xD;
             the principal investigator.&#xD;
&#xD;
          -  Pregnancy or nursing mothers.&#xD;
&#xD;
          -  History of active hepatitis B, hepatitis C, or cirrhosis&#xD;
&#xD;
          -  Receiving any investigational agent.&#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>Dwight Koeberl</last_name>
    <role>Principal Investigator</role>
    <affiliation>Duke University</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Duke University Medical Center</name>
      <address>
        <city>Durham</city>
        <state>North Carolina</state>
        <zip>27701</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>June 2019</verification_date>
  <study_first_submitted>September 13, 2017</study_first_submitted>
  <study_first_submitted_qc>September 13, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 15, 2017</study_first_posted>
  <last_update_submitted>July 1, 2019</last_update_submitted>
  <last_update_submitted_qc>July 1, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">July 2, 2019</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>Glycogen Storage Disease Type II</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/NCT03285126
org study id: Pro00085445
nct id: NCT03285126
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The purpose of this study is to determine eligibility for the future clinical trial of gene
      therapy in adults with late-onset Pompe disease.

      This screening protocol will enroll up to 20 adults patients (>=18 yo) with late-onset Pompe
      disease. Study assessments include review of medical history, vital signs, physical
      examination, muscle function testing, lung function testing, blood and urine collection,
      serum pregnancy test and ECG.

      The study results will be collected to perform descriptive statistical analysis and used to
      determine eligibility for the proposed clinical trial of gene therapy in the future.
observational model: Case-Only
time perspective: Prospective
measure: Eligibility for gene therapy.
time frame: Up to 2 days
description: The purpose of this study is to determine eligibility for the proposed clinical trial of gene therapy in adults with late-onset Pompe disease. Eligibility is measure by a composite score of blood tests, Electrocardiogram, Pulmonary Function Tests, and Muscle Status Testing.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Dwight Koeberl
role: Principal Investigator
affiliation: Duke University
facility: 
country: United States
responsible party type: Sponsor
mesh term: Glycogen Storage Disease Type II