<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/NCT02232477</url>
  </required_header>
  <id_info>
    <org_study_id>MIRC-002 (100)</org_study_id>
    <nct_id>NCT02232477</nct_id>
  </id_info>
  <brief_title>Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I</brief_title>
  <official_title>An Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>agnes chen</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>University of Minnesota</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>UCSF Benioff Children's Hospital Oakland</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>University of California, Los Angeles</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>The Ryan Foundation</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Rare Diseases Clinical Research Network</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>National Institute of Neurological Disorders and Stroke (NINDS)</agency>
      <agency_class>NIH</agency_class>
    </collaborator>
  </sponsors>
  <source>Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a five-year extension study of the pilot study, &quot;Intrathecal Enzyme Replacement for&#xD;
      Cognitive Decline in MPS I&quot;. Participants must have completed the pilot study to participate&#xD;
      in this study.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Termination of study by investigator related to COVID pandemic.&#xD;
  </why_stopped>
  <start_date>May 2014</start_date>
  <completion_date type="Actual">October 1, 2020</completion_date>
  <primary_completion_date type="Actual">October 1, 2020</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>Hopkins Verbal Learning Test</measure>
    <time_frame>5 years</time_frame>
    <description>Mean intra-subject change in memory score on the Hopkins Verbal Learning Test between baseline/screening for MIRC-002 and the subject's final visit</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">6</enrollment>
  <condition>Mucopolysaccharidosis I</condition>
  <condition>Cognitive Decline</condition>
  <arm_group>
    <arm_group_label>Open-label treatment</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>laronidase 1.74 mg IT q 3 months for five years</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Intrathecal recombinant human alpha iduronidase</intervention_name>
    <description>Intrathecal recombinant human alpha iduronidase every 3 months</description>
    <arm_group_label>Open-label treatment</arm_group_label>
    <other_name>Aldurazyme</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. The subject has completed the MIRC-002 study of intrathecal enzyme replacement therapy&#xD;
             for cognitive decline in mucopolysaccharidosis I&#xD;
&#xD;
          2. Age six years or older.&#xD;
&#xD;
          3. Subject and/or guardian willing and able to provide written informed consent.&#xD;
&#xD;
          4. Negative urine pregnancy test at screening (non-sterile females of child-bearing&#xD;
             potential only)&#xD;
&#xD;
          5. Currently using two acceptable methods of birth control as determined by the&#xD;
             investigator and willing to continue to use acceptable birth control during their&#xD;
             participation in the study (non-sterile females of child-bearing potential who are&#xD;
             sexually active only)&#xD;
&#xD;
          6. Willing and able to comply with study procedures. For example, the subjects must be&#xD;
             able to complete written and computer-based testing. The subjects must be able to lie&#xD;
             still in the MRI scanner for at least 40 minutes without sedation.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. The subject has undergone hematopoietic stem cell transplantation&#xD;
&#xD;
          2. Recent initiation of intravenous Aldurazyme® therapy with less than 6 months of&#xD;
             therapy. Subjects who have been receiving Aldurazyme® therapy for more than 6 months,&#xD;
             and those who have never received Aldurazyme® therapy, will be allowed to enroll&#xD;
&#xD;
          3. Pregnant or lactating, or considering pregnancy&#xD;
&#xD;
          4. Receipt of an investigational drug or procedure other than intrathecal Aldurazyme®&#xD;
             within 30 days of enrollment&#xD;
&#xD;
          5. A condition, medical or other, that prevents participation in the study, including&#xD;
             severe auditory or visual impairment, significant lumbar pathology, lumbar catheter,&#xD;
             or recent major surgery within 6 weeks that would preclude their ability to&#xD;
             participate.&#xD;
&#xD;
          6. Infusion reactions to intravenous or intrathecal Aldurazyme® therapy that are&#xD;
             life-threatening or require emergent intervention such as epinephrine, cardiopulmonary&#xD;
             resuscitation, or hospitalization&#xD;
&#xD;
          7. The subject has severely impaired spinal CSF flow, demonstrated by failure of&#xD;
             appearance of radionuclide in the basal cisterns by 4 hours after intra-lumbar&#xD;
             administration.&#xD;
&#xD;
          8. The subject has a coagulopathy, as identified by a platelet count of less than 50,000,&#xD;
             an INR of 1.5 or greater, or a PTT that is 1.5 times the upper limit of normal for the&#xD;
             laboratory from which it was drawn.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center</name>
      <address>
        <city>Torrance</city>
        <state>California</state>
        <zip>90509</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>October 2020</verification_date>
  <study_first_submitted>September 3, 2014</study_first_submitted>
  <study_first_submitted_qc>September 3, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 5, 2014</study_first_posted>
  <last_update_submitted>October 5, 2020</last_update_submitted>
  <last_update_submitted_qc>October 5, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">October 8, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center</investigator_affiliation>
    <investigator_full_name>agnes chen</investigator_full_name>
    <investigator_title>Principal Investigator</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Mucopolysaccharidoses</mesh_term>
    <mesh_term>Mucopolysaccharidosis I</mesh_term>
    <mesh_term>Cognitive Dysfunction</mesh_term>
  </condition_browse>
  <pending_results>
    <submitted>August 11, 2023</submitted>
    <returned>September 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/NCT02232477
org study id: MIRC-002 (100)
nct id: NCT02232477
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
collaborator: 
has dmc: Yes
This is a five-year extension study of the pilot study, "Intrathecal Enzyme Replacement for
      Cognitive Decline in MPS I". Participants must have completed the pilot study to participate
      in this study.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Hopkins Verbal Learning Test
time frame: 5 years
description: Mean intra-subject change in memory score on the Hopkins Verbal Learning Test between baseline/screening for MIRC-002 and the subject's final visit
arm group label: Open-label treatment
arm group type: Experimental
description: laronidase 1.74 mg IT q 3 months for five years
intervention type: Drug
intervention name: Intrathecal recombinant human alpha iduronidase
description: Intrathecal recombinant human alpha iduronidase every 3 months
arm group label: Open-label treatment
other name: Aldurazyme
criteria: 
gender: All
minimum age: 6 Years
maximum age: N/A
healthy volunteers: No
facility: 
country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
investigator full name: agnes chen
investigator title: Principal Investigator
mesh term: Mucopolysaccharidoses
mesh term: Mucopolysaccharidosis I
mesh term: Cognitive Dysfunction
submitted: August 11, 2023
returned: September 6, 2023