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/NCT06030648</url>
    </required_header>
    <id_info>
      <org_study_id>2022LS001</org_study_id>
      <nct_id>NCT06030648</nct_id>
    </id_info>
    <brief_title>MT2022-01: MSCs for ALD</brief_title>
    <official_title>Intravenous Administration of Mesenchymal Stem Cells (IV-MSC) for the Treatment of Cerebral Adrenoleukodystrophy (cALD)</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Masonic Cancer Center, University of Minnesota</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Masonic Cancer Center, University of Minnesota</source>
    <oversight_info>
      <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
    </oversight_info>
    <brief_summary>
      <textblock>
        This is a single-institution study to evaluate the use of intravenously administered&#xD;
        allogeneic, 3rd party mesenchymal stem cells (IV-MSC) in patients with active, cerebral&#xD;
        adrenoleukodystrophy (CALD), as a bridge to hematopoietic stem cell transplant or gene&#xD;
        therapy, or in patients who are too advanced for gene therapy or HSCT. The intervention will&#xD;
        occur in the time between diagnosis of active CALD and transplant which is currently 8-12&#xD;
        weeks.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Recruiting</overall_status>
    <start_date type="Actual">August 28, 2023</start_date>
    <completion_date type="Anticipated">May 1, 2028</completion_date>
    <primary_completion_date type="Anticipated">May 1, 2025</primary_completion_date>
    <phase>Phase 1</phase>
    <study_type>Interventional</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <allocation>Non-Randomized</allocation>
      <intervention_model>Parallel Assignment</intervention_model>
      <primary_purpose>Treatment</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).</measure>
      <time_frame>6 months</time_frame>
      <description>Safety and tolerability of cerebral adrenoleukodystrophy (cALD) patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC) assessed by incidence of adverse events.</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).</measure>
      <time_frame>6 months</time_frame>
      <description>Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI.</description>
    </secondary_outcome>
    <number_of_arms>2</number_of_arms>
    <enrollment type="Anticipated">10</enrollment>
    <condition>Cerebral Adrenoleukodystrophy</condition>
    <arm_group>
      <arm_group_label>IV-MSC for cALD (Early Disease/Bridge Therapy)</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>IV-MSC for cALD (Advanced Disease)</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT.</description>
    </arm_group>
    <intervention>
      <intervention_type>Biological</intervention_type>
      <intervention_name>Mesenchymal stem cells (IV-MSC)</intervention_name>
      <description>Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion.</description>
      <arm_group_label>IV-MSC for cALD (Advanced Disease)</arm_group_label>
      <arm_group_label>IV-MSC for cALD (Early Disease/Bridge Therapy)</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  age ≥ 3 years&#xD;
  &#xD;
            -  diagnosis of ALD, as established by elevation of very long chain fatty acid levels or&#xD;
               gene mutation&#xD;
  &#xD;
            -  evidence of active cerebral disease as determined by the presence of gadolinium&#xD;
               enhancement&#xD;
  &#xD;
            -  ALD MRI (Loes) score ≥ 1&#xD;
  &#xD;
            -  Patients who have not received prior gene therapy or transplant&#xD;
  &#xD;
            -  Life expectancy of &gt; 6 months as determined by the enrolling researcher&#xD;
  &#xD;
            -  Have adequate organ function confirmed by laboratory values obtained within 28 days&#xD;
               prior to enrollment&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Inability to undergo sedation or MRI studies for any reason&#xD;
  &#xD;
            -  Other concurrent life-threatening disease (life expectancy &lt;6 months) or eligible for&#xD;
               hospice care&#xD;
  &#xD;
            -  Known or suspected hypersensitivity to human serum albumin, diphenhydramine,&#xD;
               acetaminophen, methylprednisolone, or any of the components of IMS001.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>3 Years</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_contact>
      <last_name>Troy C Lund, MD</last_name>
      <phone>+1 612-626-2778</phone>
      <email>lundx072@umn.edu</email>
    </overall_contact>
    <location>
      <facility>
        <name>Masonic Cancer Center, University of Minnesota</name>
        <address>
          <city>Minneapolis</city>
          <state>Minnesota</state>
          <zip>55455</zip>
          <country>United States</country>
        </address>
      </facility>
      <status>Recruiting</status>
      <contact>
        <last_name>Troy C Lund, MD</last_name>
        <phone>612-626-2778</phone>
        <email>lundx072@umn.edu</email>
      </contact>
    </location>
    <location_countries>
      <country>United States</country>
    </location_countries>
    <verification_date>September 2023</verification_date>
    <study_first_submitted>August 31, 2023</study_first_submitted>
    <study_first_submitted_qc>September 7, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">September 11, 2023</study_first_posted>
    <last_update_submitted>September 7, 2023</last_update_submitted>
    <last_update_submitted_qc>September 7, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">September 11, 2023</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>Adrenoleukodystrophy</mesh_term>
    </condition_browse>
    <!-- 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/NCT06030648
  org study id: 2022LS001
  nct id: NCT06030648
  lead sponsor: 
  is fda regulated drug: Yes
  is fda regulated device: No
  This is a single-institution study to evaluate the use of intravenously administered
        allogeneic, 3rd party mesenchymal stem cells (IV-MSC) in patients with active, cerebral
        adrenoleukodystrophy (CALD), as a bridge to hematopoietic stem cell transplant or gene
        therapy, or in patients who are too advanced for gene therapy or HSCT. The intervention will
        occur in the time between diagnosis of active CALD and transplant which is currently 8-12
        weeks.
  allocation: Non-Randomized
  intervention model: Parallel Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Safety and tolerability of intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
  time frame: 6 months
  description: Safety and tolerability of cerebral adrenoleukodystrophy (cALD) patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC) assessed by incidence of adverse events.
  measure: Incidence of radiographic response in patients receiving intravenously-administered allogeneic, third-party mesenchymal stem cells (IV-MSC).
  time frame: 6 months
  description: Radiographic response is defined as the resolution of intravenous gadolinium contrast enhancement on brain MRI.
  arm group label: IV-MSC for cALD (Early Disease/Bridge Therapy)
  arm group type: Experimental
  description: Patients with active, cerebral adrenoleukodystrophy (cALD) as a bridge to hematopoietic stem cell transplant or gene therapy.
  arm group label: IV-MSC for cALD (Advanced Disease)
  arm group type: Experimental
  description: Patients with active, cerebral adrenoleukodystrophy (cALD) who are too advanced for gene therapy or HSCT.
  intervention type: Biological
  intervention name: Mesenchymal stem cells (IV-MSC)
  description: Patients will receive 1 infusion (1 x 106 cells/kg on day 0) then be followed for 8 weeks from the infusion.
  arm group label: IV-MSC for cALD (Advanced Disease)
  arm group label: IV-MSC for cALD (Early Disease/Bridge Therapy)
  criteria: 
  gender: All
  minimum age: 3 Years
  maximum age: N/A
  healthy volunteers: No
  last name: Troy C Lund, MD
  phone: +1 612-626-2778
  email: lundx072@umn.edu
  facility: 
  status: Recruiting
  contact: 
  country: United States
  responsible party type: Sponsor
  mesh term: Adrenoleukodystrophy