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/NCT06031259</url>
    </required_header>
    <id_info>
      <org_study_id>TAK-609-3001</org_study_id>
      <secondary_id>2023-504127-90</secondary_id>
      <nct_id>NCT06031259</nct_id>
    </id_info>
    <brief_title>Extension Study of Idursulfase-IT Along With Elaprase in Adults With Hunter Syndrome and Cognitive Impairment</brief_title>
    <official_title>An Open-Label Extension for Subjects in Studies HGT-HIT-046 and SHP609-302 Evaluating Long-Term Safety of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Subjects With Hunter Syndrome and Cognitive Impairment</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Takeda</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Takeda</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>
      <is_us_export>Yes</is_us_export>
    </oversight_info>
    <brief_summary>
      <textblock>
        The study is an extension of two previous studies (HGT-HIT-046 [NCT01506141] and SHP609-302&#xD;
        [NCT02412787]). Participants must have completed one of the previous studies. The main aim of&#xD;
        this study is to collect more information about the safety of the treatments, idursulfase-IT&#xD;
        and elaprase, in adults with Hunter syndrome and cognitive impairment. Participants will&#xD;
        receive the same treatment as in the previous studies.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        The drug being tested in this study is called idursulfase. Idursulfase is being tested for&#xD;
        long term safety in participants with Hunter syndrome and cognitive impairment.&#xD;
  &#xD;
        The study will enroll up to approximately 8 patients. Participants will receive&#xD;
        idursulfase-IT via intrathecal drug delivery device (IDDD) once monthly along with elaprase&#xD;
        intravenous (IV) infusion, weekly.&#xD;
  &#xD;
        This multi-center trial will be conducted in France and Canada. The overall time to&#xD;
        participate in this study is approximately 10.4 years.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">January 1, 2024</start_date>
    <completion_date type="Anticipated">June 1, 2034</completion_date>
    <primary_completion_date type="Anticipated">January 1, 2034</primary_completion_date>
    <phase>Phase 2/Phase 3</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>Number of Participants with Adverse Events (AEs) by Type and Severity</measure>
      <time_frame>Up to approximately 10.4 years</time_frame>
      <description>An adverse event (AE) means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. The severity of AEs will be determined per the investigator's assessment.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Number of Participants with AEs Related to Idursulfase-IT</measure>
      <time_frame>Up to approximately 10.4 years</time_frame>
      <description>An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to idursulfase-IT will be reported in this outcome measure.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Number of Participants with AEs Related to the IDDD</measure>
      <time_frame>Up to approximately 10.4 years</time_frame>
      <description>An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to the IDDD will be reported in this outcome measure.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Number of Participants with AEs Related to Device Surgical Procedure</measure>
      <time_frame>Up to approximately 10.4 years</time_frame>
      <description>An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to device surgical procedure will be reported in this outcome measure.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Number of Participants with AEs Related to IT Administration Process</measure>
      <time_frame>Up to approximately 10.4 years</time_frame>
      <description>An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to IT administration process will be reported in this outcome measure.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Number of Participants with AEs Related to IV Elaprase Infusion</measure>
      <time_frame>Up to approximately 10.4 years</time_frame>
      <description>An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to IV elaprase infusion will be reported in this outcome measure.</description>
    </primary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">8</enrollment>
    <condition>Hunter Syndrome</condition>
    <arm_group>
      <arm_group_label>Idursulfase-IT</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Participants will receive idursulfase-IT once monthly and weekly IV infusions of elaprase at the dose used in previous studies (HGT-HIT-045/SHP609-302) via IDDD until benefit is no longer derived from the treatment, or treatment is no longer tolerable, or up to approximately 10.4 years.</description>
    </arm_group>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Idursulfase-IT</intervention_name>
      <description>Idursulfase-IT intrathecally via IDDD.</description>
      <arm_group_label>Idursulfase-IT</arm_group_label>
    </intervention>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Elaprase</intervention_name>
      <description>Elaprase IV infusion.</description>
      <arm_group_label>Idursulfase-IT</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            1. The participant must have completed end of study assessments in studies HGT-HIT-046&#xD;
               [NCT01506141] or SHP609-302 [NCT02412787] and received a clinical benefit from&#xD;
               idursulfase-IT in the opinion of the investigator.&#xD;
  &#xD;
            2. The participant, or participant's legally designated representative, must have been&#xD;
               informed of the nature of this open-label extension and must have voluntarily signed&#xD;
               an Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved&#xD;
               informed consent form after all relevant aspects of the study have been explained and&#xD;
               discussed. Written consent of the participant's legally designated representative (if&#xD;
               applicable) and the participant's consent/assent, as relevant, must be obtained.&#xD;
  &#xD;
            3. The participant has continued to receive elaprase on a regular basis in studies&#xD;
               HGT-HIT-046 or SHP609-302.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            1. The participant has experienced, in the opinion of the investigator, a safety or&#xD;
               medical issue that contraindicates treatment with idursulfase-IT, including, but not&#xD;
               limited to, uncontrolled seizure disorder, bleeding disorder, and clinically relevant&#xD;
               hypertension.&#xD;
  &#xD;
            2. The participant has clinically relevant intracranial hypertension.&#xD;
  &#xD;
            3. The participant is enrolled in another clinical study, other than studies HGT-HIT-046&#xD;
               or SHP609-302, that involves clinical investigations or use of any investigational&#xD;
               product (drug or [intrathecal/spinal] device) within 30 days prior to study enrolment&#xD;
               or at any time during the study.&#xD;
        </textblock>
      </criteria>
      <gender>Male</gender>
      <gender_based>Yes</gender_based>
      <minimum_age>3 Years</minimum_age>
      <maximum_age>19 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Study Director</last_name>
      <role>Study Director</role>
      <affiliation>Takeda</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Takeda Contact</last_name>
      <phone>+1-877-825-3327</phone>
      <email>medinfoUS@takeda.com</email>
    </overall_contact>
    <location>
      <facility>
        <name>M.A.G.I.C. Clinic LTD</name>
        <address>
          <city>Calgary</city>
          <state>Alberta</state>
          <zip>T2E 7Z4</zip>
          <country>Canada</country>
        </address>
      </facility>
      <contact>
        <last_name>Site Contact</last_name>
        <phone>4039557587</phone>
        <email>Aneal.khan@ahs.ca</email>
      </contact>
      <investigator>
        <last_name>Aneal Khan</last_name>
        <role>Principal Investigator</role>
      </investigator>
    </location>
    <location>
      <facility>
        <name>University of British Columbia</name>
        <address>
          <city>Vancouver</city>
          <state>British Columbia</state>
          <zip>V6T1Z3</zip>
          <country>Canada</country>
        </address>
      </facility>
      <contact>
        <last_name>Site Contact</last_name>
        <phone>6048755905</phone>
        <email>sstockler@cw.bc.ca</email>
      </contact>
      <investigator>
        <last_name>Sylvia Stockler</last_name>
        <role>Principal Investigator</role>
      </investigator>
    </location>
    <location>
      <facility>
        <name>The Hospital for Sick Children</name>
        <address>
          <city>Toronto</city>
          <state>Ontario</state>
          <zip>M5G1X8</zip>
          <country>Canada</country>
        </address>
      </facility>
      <contact>
        <last_name>Site Contact</last_name>
        <phone>4168135753</phone>
        <email>Michal.inbar-feigenberg@sickkids.ca</email>
      </contact>
      <investigator>
        <last_name>Michal Inbar- Feigenberg</last_name>
        <role>Principal Investigator</role>
      </investigator>
    </location>
    <location>
      <facility>
        <name>Hospices Civils de Lyon</name>
        <address>
          <city>Lyon Cedex</city>
          <zip>69229</zip>
          <country>France</country>
        </address>
      </facility>
      <contact>
        <last_name>Site Contact</last_name>
        <phone>+33472129537</phone>
        <email>Nathalie.guffon-fouilhoux@chu-lyon.fr</email>
      </contact>
      <investigator>
        <last_name>Nathalie Guffon</last_name>
        <role>Principal Investigator</role>
      </investigator>
    </location>
    <location_countries>
      <country>Canada</country>
      <country>France</country>
    </location_countries>
    <verification_date>September 2023</verification_date>
    <study_first_submitted>September 4, 2023</study_first_submitted>
    <study_first_submitted_qc>September 4, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">September 11, 2023</study_first_posted>
    <last_update_submitted>September 4, 2023</last_update_submitted>
    <last_update_submitted_qc>September 4, 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>
    <keyword>Drug Therapy</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Mucopolysaccharidosis II</mesh_term>
      <mesh_term>Syndrome</mesh_term>
      <mesh_term>Cognitive Dysfunction</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>Yes</sharing_ipd>
      <ipd_description>Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.</ipd_description>
      <ipd_info_type>Study Protocol</ipd_info_type>
      <ipd_info_type>Statistical Analysis Plan (SAP)</ipd_info_type>
      <ipd_info_type>Informed Consent Form (ICF)</ipd_info_type>
      <ipd_info_type>Clinical Study Report (CSR)</ipd_info_type>
      <ipd_access_criteria>IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.</ipd_access_criteria>
      <ipd_url>https://vivli.org/ourmember/takeda/</ipd_url>
    </patient_data>
    <!-- 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/NCT06031259
  org study id: TAK-609-3001
  secondary id: 2023-504127-90
  nct id: NCT06031259
  lead sponsor: 
  has dmc: No
  is fda regulated drug: Yes
  is fda regulated device: No
  is us export: Yes
  The study is an extension of two previous studies (HGT-HIT-046 [NCT01506141] and SHP609-302
        [NCT02412787]). Participants must have completed one of the previous studies. The main aim of
        this study is to collect more information about the safety of the treatments, idursulfase-IT
        and elaprase, in adults with Hunter syndrome and cognitive impairment. Participants will
        receive the same treatment as in the previous studies.
  The drug being tested in this study is called idursulfase. Idursulfase is being tested for
        long term safety in participants with Hunter syndrome and cognitive impairment.
  
        The study will enroll up to approximately 8 patients. Participants will receive
        idursulfase-IT via intrathecal drug delivery device (IDDD) once monthly along with elaprase
        intravenous (IV) infusion, weekly.
  
        This multi-center trial will be conducted in France and Canada. The overall time to
        participate in this study is approximately 10.4 years.
  allocation: N/A
  intervention model: Single Group Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: Number of Participants with Adverse Events (AEs) by Type and Severity
  time frame: Up to approximately 10.4 years
  description: An adverse event (AE) means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. The severity of AEs will be determined per the investigator's assessment.
  measure: Number of Participants with AEs Related to Idursulfase-IT
  time frame: Up to approximately 10.4 years
  description: An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to idursulfase-IT will be reported in this outcome measure.
  measure: Number of Participants with AEs Related to the IDDD
  time frame: Up to approximately 10.4 years
  description: An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to the IDDD will be reported in this outcome measure.
  measure: Number of Participants with AEs Related to Device Surgical Procedure
  time frame: Up to approximately 10.4 years
  description: An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to device surgical procedure will be reported in this outcome measure.
  measure: Number of Participants with AEs Related to IT Administration Process
  time frame: Up to approximately 10.4 years
  description: An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to IT administration process will be reported in this outcome measure.
  measure: Number of Participants with AEs Related to IV Elaprase Infusion
  time frame: Up to approximately 10.4 years
  description: An AE means any untoward medical occurrence in a participant or participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product whether or not it is related to the medicinal product. Any adverse event related to IV elaprase infusion will be reported in this outcome measure.
  arm group label: Idursulfase-IT
  arm group type: Experimental
  description: Participants will receive idursulfase-IT once monthly and weekly IV infusions of elaprase at the dose used in previous studies (HGT-HIT-045/SHP609-302) via IDDD until benefit is no longer derived from the treatment, or treatment is no longer tolerable, or up to approximately 10.4 years.
  intervention type: Drug
  intervention name: Idursulfase-IT
  description: Idursulfase-IT intrathecally via IDDD.
  arm group label: Idursulfase-IT
  intervention type: Drug
  intervention name: Elaprase
  description: Elaprase IV infusion.
  arm group label: Idursulfase-IT
  criteria: 
  gender: Male
  gender based: Yes
  minimum age: 3 Years
  maximum age: 19 Years
  healthy volunteers: No
  last name: Study Director
  role: Study Director
  affiliation: Takeda
  last name: Takeda Contact
  phone: +1-877-825-3327
  email: medinfoUS@takeda.com
  facility: 
  contact: 
  investigator: 
  facility: 
  contact: 
  investigator: 
  facility: 
  contact: 
  investigator: 
  facility: 
  contact: 
  investigator: 
  country: Canada
  country: France
  responsible party type: Sponsor
  mesh term: Mucopolysaccharidosis II
  mesh term: Syndrome
  mesh term: Cognitive Dysfunction
  sharing ipd: Yes
  ipd description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
  ipd info type: Study Protocol
  ipd info type: Statistical Analysis Plan (SAP)
  ipd info type: Informed Consent Form (ICF)
  ipd info type: Clinical Study Report (CSR)
  ipd access criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
  ipd url: https://vivli.org/ourmember/takeda/