<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/NCT04476862</url>
  </required_header>
  <id_info>
    <org_study_id>190-501</org_study_id>
    <nct_id>NCT04476862</nct_id>
  </id_info>
  <brief_title>Cerliponase Alfa Observational Study in the US</brief_title>
  <official_title>Cerliponase Alfa Observational Study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>BioMarin Pharmaceutical</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>BioMarin Pharmaceutical</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>Yes</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a multicenter, observational study for patients with a confirmed diagnosis of&#xD;
      neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who&#xD;
      intend to be or are currently being treated with cerliponase alfa. Patients receiving or&#xD;
      expected to receive cerliponase alfa within 60 days of signing the informed consent form&#xD;
      (ICF) may be eligible to enroll in the study, assuming all regulatory requirements for sites&#xD;
      that have agreed to participate and protocol inclusion criteria are met. Data may be&#xD;
      collected for all or some of the assessments as outlined in the protocol, dependent upon the&#xD;
      clinic's and/or individual patient's standard of care.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">August 19, 2020</start_date>
  <completion_date type="Anticipated">August 24, 2030</completion_date>
  <primary_completion_date type="Anticipated">August 24, 2030</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Safety surveillance of cerliponase alfa</measure>
    <time_frame>10 years</time_frame>
    <description>To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Hypersensitivity</measure>
    <time_frame>10 years</time_frame>
    <description>To further assess the occurrence of serious hypersensitivity reactions (including anaphylaxis), serious cardiovascular adverse events, and serious device-related complications.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Severe SAE impact on patient's motor and language functions</measure>
    <time_frame>10 years</time_frame>
    <description>To evaluate the effects of Grade III or higher serious adverse events (SAEs) on patient performance on the CLN2 clinical rating scale (motor and language domains).</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">35</enrollment>
  <condition>Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2</condition>
  <arm_group>
    <arm_group_label>Cerliponase alfa patients</arm_group_label>
    <description>Patients who are currently on or plan to start taking cerliponase alfa within 60 days of signing the study informed consent form.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Cerliponase Alfa</intervention_name>
    <description>Commercially available product provided to patient by participating clinic site.</description>
    <arm_group_label>Cerliponase alfa patients</arm_group_label>
    <other_name>Brineura</other_name>
  </intervention>
  <intervention>
    <intervention_type>Device</intervention_type>
    <intervention_name>Administration Kit</intervention_name>
    <description>Commercially available administration kit provided to the patient by participating clinic site.</description>
    <arm_group_label>Cerliponase alfa patients</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        CLN2 patients who are currently taking or intend to take cerliponase alfa within 60 days of&#xD;
        signing the informed consent form.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Diagnosed with CLN2 disease.&#xD;
&#xD;
          2. Currently receiving or plan to begin treatment with cerliponase alfa.&#xD;
&#xD;
          3. Written informed consent/assent obtained.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        1. Currently receiving treatment in another investigational device or drug study.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Medical Monitor, MD</last_name>
    <role>Study Director</role>
    <affiliation>BioMarin Pharmaceutical</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Trial Specialist</last_name>
    <phone>+1.651.523.0310</phone>
    <email>medinfo@bmrn.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>Arkansas Children's Hospital</name>
      <address>
        <city>Little Rock</city>
        <state>Arkansas</state>
        <zip>72205</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Kapil Arya, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Orange County</name>
      <address>
        <city>Orange</city>
        <state>California</state>
        <zip>92868</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Raymond Wang, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Colorado</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Scott Demarest, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Children's National Hospital</name>
      <address>
        <city>Washington</city>
        <state>District of Columbia</state>
        <zip>20010</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Andrea Gropman, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Advent Health</name>
      <address>
        <city>Orlando</city>
        <state>Florida</state>
        <zip>32803</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Ki Hyeong Lee</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Children's Healthcare of Atlanta</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30329</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Stephanie Keller</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Rush University Medical Center</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60612</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Elizabeth Berry-Kravis</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Boston Children's Hospital,</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Terminated</status>
  </location>
  <location>
    <facility>
      <name>Children's Hospital Minnesota</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55404</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Vikas Bhambhani, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>NYU Langone Medical Center</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10017</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Not yet recruiting</status>
    <investigator>
      <last_name>Nicholas Abreu, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Mt. Sinai School of Medicine</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10029</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Jayesh Ganesh, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Rochester Medical Center</name>
      <address>
        <city>Rochester</city>
        <state>New York</state>
        <zip>14642</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Jennifer Vermilion, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Nationwide Children's Hospital</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43130</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Emily De Los Reyes, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>University of Oklahoma Health Sciences Center</name>
      <address>
        <city>Oklahoma City</city>
        <state>Oklahoma</state>
        <zip>73104</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Terminated</status>
  </location>
  <location>
    <facility>
      <name>Texas Children's Hospital</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Gary Clark</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Seattle Children's Hospital</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98145-5005</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Irene Chang</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>February 2023</verification_date>
  <study_first_submitted>July 15, 2020</study_first_submitted>
  <study_first_submitted_qc>July 15, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 20, 2020</study_first_posted>
  <last_update_submitted>February 22, 2023</last_update_submitted>
  <last_update_submitted_qc>February 22, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">February 23, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Batten's Disease</keyword>
  <keyword>CLN2</keyword>
  <keyword>TPP1 enzyme</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neuronal Ceroid-Lipofuscinoses</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</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/NCT04476862
org study id: 190-501
nct id: NCT04476862
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: Yes
This is a multicenter, observational study for patients with a confirmed diagnosis of
      neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who
      intend to be or are currently being treated with cerliponase alfa. Patients receiving or
      expected to receive cerliponase alfa within 60 days of signing the informed consent form
      (ICF) may be eligible to enroll in the study, assuming all regulatory requirements for sites
      that have agreed to participate and protocol inclusion criteria are met. Data may be
      collected for all or some of the assessments as outlined in the protocol, dependent upon the
      clinic's and/or individual patient's standard of care.
observational model: Cohort
time perspective: Prospective
measure: Safety surveillance of cerliponase alfa
time frame: 10 years
description: To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).
measure: Hypersensitivity
time frame: 10 years
description: To further assess the occurrence of serious hypersensitivity reactions (including anaphylaxis), serious cardiovascular adverse events, and serious device-related complications.
measure: Severe SAE impact on patient's motor and language functions
time frame: 10 years
description: To evaluate the effects of Grade III or higher serious adverse events (SAEs) on patient performance on the CLN2 clinical rating scale (motor and language domains).
arm group label: Cerliponase alfa patients
description: Patients who are currently on or plan to start taking cerliponase alfa within 60 days of signing the study informed consent form.
intervention type: Drug
intervention name: Cerliponase Alfa
description: Commercially available product provided to patient by participating clinic site.
arm group label: Cerliponase alfa patients
other name: Brineura
intervention type: Device
intervention name: Administration Kit
description: Commercially available administration kit provided to the patient by participating clinic site.
arm group label: Cerliponase alfa patients
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Medical Monitor, MD
role: Study Director
affiliation: BioMarin Pharmaceutical
last name: Trial Specialist
phone: +1.651.523.0310
email: medinfo@bmrn.com
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Terminated
facility: 
status: Recruiting
investigator: 
facility: 
status: Not yet recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Terminated
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
country: United States
responsible party type: Sponsor
mesh term: Neuronal Ceroid-Lipofuscinoses
sharing ipd: No