<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/NCT03227042</url>
  </required_header>
  <id_info>
    <org_study_id>250-902</org_study_id>
    <nct_id>NCT03227042</nct_id>
  </id_info>
  <brief_title>A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)</brief_title>
  <official_title>A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Allievex Corporation</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Allievex Corporation</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>
      This is a natural history study for children up to 18 years of age who have been diagnosed&#xD;
      with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B).&#xD;
      Mucopolysaccharidosis type IIIB is a severe neurodegenerative disorder. The information&#xD;
      gathered from this trial may help inform the design and interpretation of subsequent&#xD;
      interventional studies. No clinical intervention or study drug is provided by Allievex in&#xD;
      this study.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">November 16, 2017</start_date>
  <completion_date type="Anticipated">December 2025</completion_date>
  <primary_completion_date type="Anticipated">October 2025</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>Neurocognitive function</measure>
    <time_frame>Baseline + every 24 weeks for up to 240 weeks</time_frame>
    <description>A neurodevelopmental assessment will be performed using standardized developmental tests to provide quantifiable measures of neurocognitive function.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Behavioral function</measure>
    <time_frame>Baseline + every 24 weeks for up to 240 weeks</time_frame>
    <description>Disease-related behaviors will be assessed using an MPS IIIB specific behavior rating scale.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Quality of Life Tests</measure>
    <time_frame>Baseline + every 24 weeks for up to 240 weeks</time_frame>
    <description>Multiple QoL tools will be used to capture physical, mental, and social well-being of the patient as well as to examine the impact of the patient's disease on the parent/guardian and family.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Sleep habits</measure>
    <time_frame>Baseline + every 24 weeks for up to 240 weeks</time_frame>
    <description>Patient sleep habits will be assessed using Children's Sleep Habits Questionnaires (CSHQ).</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Disease-specific Biomarkers</measure>
    <time_frame>Baseline + every 24 weeks for up to 240 weeks</time_frame>
    <description>Urine sample for glycosaminoglycans (GAGs) and creatinine.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Biochemical, Molecular, Cellular and Genetic Markers of Disease Burden</measure>
    <time_frame>Once (at baseline visit)</time_frame>
    <description>Blood and urine samples will be used to evaluate biochemical, molecular cellular, and genetic/genomic aspects of MPS IIIB.</description>
  </primary_outcome>
  <enrollment type="Actual">44</enrollment>
  <condition>Mucopolysaccharidosis Type IIIB</condition>
  <eligibility>
    <study_pop>
      <textblock>
        Males and Females with a documented diagnosis of MPSIIIB&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Have deficient NAGLU enzyme activity at Baseline. Blood for NAGLU enzyme activity will&#xD;
             be collected and analyzed centrally.&#xD;
&#xD;
          -  Is up to 18 years of age&#xD;
&#xD;
          -  Written informed consent from parent or legal guardian and assent from subject, if&#xD;
             required&#xD;
&#xD;
          -  Has the ability to comply with protocol requirements, in the opinion of the&#xD;
             investigator&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Has another neurological illness that may have caused cognitive decline (e.g., trauma,&#xD;
             meningitis, or hemorrhage) before study entry&#xD;
&#xD;
          -  Has received stem cell, gene therapy, or enzyme replacement therapy for MPS IIIB&#xD;
&#xD;
          -  Has received any investigational medication within 30 days prior to the Baseline visit&#xD;
             or is scheduled to receive any investigational drug during the course of the study&#xD;
&#xD;
          -  Has a medical condition or extenuating circumstance that, in the opinion of the&#xD;
             investigator, might compromise the subject's ability to comply with protocol&#xD;
             requirements, the subject's wellbeing or safety, or the interpretability of the&#xD;
             subject's clinical data.&#xD;
&#xD;
          -  Is currently participating in another natural history study&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>18 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Medical Director, MD</last_name>
    <role>Study Director</role>
    <affiliation>Allievex Medical Monitor</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>UCSF Benioff Children's Hospital Oakland</name>
      <address>
        <city>Oakland</city>
        <state>California</state>
        <zip>94609</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Austral</name>
      <address>
        <city>Pilar</city>
        <zip>B1629AHJ</zip>
        <country>Argentina</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Murdoch Childrens Research Institute and Royal Children's Hospital</name>
      <address>
        <city>Melbourne</city>
        <state>Victoria</state>
        <zip>3052</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Medical Genetics Service/HCPA, Department of Genetics/UFRGS</name>
      <address>
        <city>Pôrto Alegre</city>
        <state>Rio Grande Do Sul</state>
        <zip>90035-903</zip>
        <country>Brazil</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Fundación Cardio Infantil - Instituto de Cardiología</name>
      <address>
        <city>Bogotá</city>
        <country>Colombia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Medical Center Hamburg-Eppendorf</name>
      <address>
        <city>Hamburg</city>
        <zip>20246</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Clínico Universitario de Santiago</name>
      <address>
        <city>Santiago de Compostela</city>
        <state>A Coruña</state>
        <zip>15706</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>MacKay Memorial Children's Hospital</name>
      <address>
        <city>Taipei</city>
        <zip>10449</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Gazi University Faculty of Medicine</name>
      <address>
        <city>Ankara</city>
        <zip>06560</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Argentina</country>
    <country>Australia</country>
    <country>Brazil</country>
    <country>Colombia</country>
    <country>Germany</country>
    <country>Spain</country>
    <country>Taiwan</country>
    <country>Turkey</country>
    <country>United States</country>
  </location_countries>
  <verification_date>October 2022</verification_date>
  <study_first_submitted>July 11, 2017</study_first_submitted>
  <study_first_submitted_qc>July 19, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">July 24, 2017</study_first_posted>
  <last_update_submitted>October 24, 2022</last_update_submitted>
  <last_update_submitted_qc>October 24, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">October 26, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Sanfilippo Syndrome Type B</keyword>
  <keyword>MPS IIIB</keyword>
  <keyword>MPS 3 B</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Mucopolysaccharidoses</mesh_term>
    <mesh_term>Mucopolysaccharidosis III</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </patient_data>
  <!-- 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/NCT03227042
org study id: 250-902
nct id: NCT03227042
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
This is a natural history study for children up to 18 years of age who have been diagnosed
      with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B).
      Mucopolysaccharidosis type IIIB is a severe neurodegenerative disorder. The information
      gathered from this trial may help inform the design and interpretation of subsequent
      interventional studies. No clinical intervention or study drug is provided by Allievex in
      this study.
observational model: Other
time perspective: Prospective
measure: Neurocognitive function
time frame: Baseline + every 24 weeks for up to 240 weeks
description: A neurodevelopmental assessment will be performed using standardized developmental tests to provide quantifiable measures of neurocognitive function.
measure: Behavioral function
time frame: Baseline + every 24 weeks for up to 240 weeks
description: Disease-related behaviors will be assessed using an MPS IIIB specific behavior rating scale.
measure: Quality of Life Tests
time frame: Baseline + every 24 weeks for up to 240 weeks
description: Multiple QoL tools will be used to capture physical, mental, and social well-being of the patient as well as to examine the impact of the patient's disease on the parent/guardian and family.
measure: Sleep habits
time frame: Baseline + every 24 weeks for up to 240 weeks
description: Patient sleep habits will be assessed using Children's Sleep Habits Questionnaires (CSHQ).
measure: Disease-specific Biomarkers
time frame: Baseline + every 24 weeks for up to 240 weeks
description: Urine sample for glycosaminoglycans (GAGs) and creatinine.
measure: Biochemical, Molecular, Cellular and Genetic Markers of Disease Burden
time frame: Once (at baseline visit)
description: Blood and urine samples will be used to evaluate biochemical, molecular cellular, and genetic/genomic aspects of MPS IIIB.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: 18 Years
healthy volunteers: No
last name: Medical Director, MD
role: Study Director
affiliation: Allievex Medical Monitor
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Argentina
country: Australia
country: Brazil
country: Colombia
country: Germany
country: Spain
country: Taiwan
country: Turkey
country: United States
responsible party type: Sponsor
mesh term: Mucopolysaccharidoses
mesh term: Mucopolysaccharidosis III
sharing ipd: Undecided