<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/NCT04419233</url>
  </required_header>
  <id_info>
    <org_study_id>232SM402</org_study_id>
    <nct_id>NCT04419233</nct_id>
  </id_info>
  <brief_title>Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection</brief_title>
  <acronym>PANDA</acronym>
  <official_title>A Multicenter, Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection When Used in Routine Medical Practice in China</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Biogen</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Biogen</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The primary objective of this study is to evaluate the safety of nusinersen sodium injection&#xD;
      in the postmarketing setting in China.The secondary objectives are to collect data on the&#xD;
      efficacy and the pharmacokinetics of nusinersen sodium injection in the post-marketing&#xD;
      setting in China.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">November 18, 2020</start_date>
  <completion_date type="Anticipated">January 27, 2025</completion_date>
  <primary_completion_date type="Anticipated">May 31, 2024</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Only</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)</measure>
    <time_frame>Day 0 up to End of Treatment (2 Years)</time_frame>
    <description>An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or is a medically important event.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percentage of Participants who Achieved World Health Organization (WHO) Motor Milestone</measure>
    <time_frame>Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)</time_frame>
    <description>WHO Multicentre Growth Reference Study (MGRS), a component to assess gross motor development and is usually assessed in terms of age of achievement of motor milestones. Six distinct gross motor milestones criteria are as follows: sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Hammersmith Infant Neurological Examination (HINE) Section 2 Scores</measure>
    <time_frame>Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)</time_frame>
    <description>Section 2 of the HINE is used to assess motor milestones of the participants. It is composed of 8 motor milestone categories: voluntary grasp, ability to kick in supine position, head control, rolling, sitting, crawling, standing, and walking.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants with Ventilatory Support</measure>
    <time_frame>Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Plasma Concentrations of Nusinersen Sodium Injection</measure>
    <time_frame>Pre-dose and multiple time points post-dose on Day 0, post-dose on Day 28 and pre-dose every 4 months up to end of treatment (2 years)</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Cerebrospinal Fluid (CSF) Concentrations of Nusinersen Sodium Injection</measure>
    <time_frame>pre-dose on Day 0, Day 63, and Every 4 Months Up to End of Treatment (2 years)</time_frame>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">50</enrollment>
  <condition>Muscular Atrophy, Spinal</condition>
  <arm_group>
    <arm_group_label>All Participants</arm_group_label>
    <description>Participants with 5q SMA and who were prescribed with nusinersen sodium injection in China according to the local marketing authorization.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Nusinersen Sodium Injection</intervention_name>
    <description>Administered as specified in the treatment arm.</description>
    <arm_group_label>All Participants</arm_group_label>
    <other_name>BIIB058</other_name>
    <other_name>ISIS 396443</other_name>
    <other_name>Spinraza</other_name>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Participants who are prescribed nusinersen sodium injection in China according to the local&#xD;
        marketing authorization (including documentation of 5q SMA diagnosis), agree to the&#xD;
        collection and use of data as specified in the protocol, and meet the eligibility criteria.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Key Inclusion Criteria:&#xD;
&#xD;
          -  Ability of the participant or his/her legally authorized representative (e.g., parent&#xD;
             or legal guardian), as appropriate and applicable, to understand the purpose and risks&#xD;
             of the study and provide signed and dated informed consent and authorization to use&#xD;
             protected health information in accordance with national and local subject privacy&#xD;
             regulations.&#xD;
&#xD;
          -  The decision to newly prescribe nusinersen sodium injection according to the local&#xD;
             marketing authorization, including documentation of 5q SMA diagnosis, has been made by&#xD;
             the Investigator and agreed to by the participant (or legally authorized&#xD;
             representative)&#xD;
&#xD;
        Key Exclusion Criteria:&#xD;
&#xD;
          -  Hypersensitivity to the active substance or any of the excipients of nusinersen sodium&#xD;
             injection.&#xD;
&#xD;
          -  Current or past administration of nusinersen sodium injection, in either a commercial&#xD;
             or a clinical study setting.&#xD;
&#xD;
          -  Other unspecified reasons that, in the opinion of the Investigator or the Sponsor,&#xD;
             make the participant unsuitable for enrollment.&#xD;
&#xD;
        NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.&#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 Director</last_name>
    <role>Study Director</role>
    <affiliation>Biogen</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Research Site</name>
      <address>
        <city>Xicheng</city>
        <state>Beijing Shi</state>
        <zip>100045</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site</name>
      <address>
        <city>Fuzhou</city>
        <state>Fujian</state>
        <zip>UNK</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site</name>
      <address>
        <city>Guangzhou</city>
        <state>Guangdong</state>
        <zip>UNK</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site</name>
      <address>
        <city>Suzhou</city>
        <state>Jiangsu</state>
        <zip>UNK</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site</name>
      <address>
        <city>Changchun</city>
        <state>Jilin</state>
        <zip>130021</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site</name>
      <address>
        <city>Minhang</city>
        <state>Shanghai</state>
        <zip>201103</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site</name>
      <address>
        <city>Chengdu</city>
        <state>Sichuan</state>
        <zip>610041</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site</name>
      <address>
        <city>Beijing</city>
        <state>Xicheng</state>
        <zip>100045</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site</name>
      <address>
        <city>Hangzhou</city>
        <state>Zhejiang</state>
        <zip>310052</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <verification_date>April 2023</verification_date>
  <study_first_submitted>June 3, 2020</study_first_submitted>
  <study_first_submitted_qc>June 3, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 5, 2020</study_first_posted>
  <last_update_submitted>April 11, 2023</last_update_submitted>
  <last_update_submitted_qc>April 11, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">April 13, 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>Muscular Atrophy</mesh_term>
    <mesh_term>Muscular Atrophy, Spinal</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/</ipd_description>
    <ipd_url>https://vivli.org/</ipd_url>
  </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/NCT04419233
org study id: 232SM402
nct id: NCT04419233
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
The primary objective of this study is to evaluate the safety of nusinersen sodium injection
      in the postmarketing setting in China.The secondary objectives are to collect data on the
      efficacy and the pharmacokinetics of nusinersen sodium injection in the post-marketing
      setting in China.
observational model: Case-Only
time perspective: Prospective
measure: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
time frame: Day 0 up to End of Treatment (2 Years)
description: An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or is a medically important event.
measure: Percentage of Participants who Achieved World Health Organization (WHO) Motor Milestone
time frame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
description: WHO Multicentre Growth Reference Study (MGRS), a component to assess gross motor development and is usually assessed in terms of age of achievement of motor milestones. Six distinct gross motor milestones criteria are as follows: sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.
measure: Hammersmith Infant Neurological Examination (HINE) Section 2 Scores
time frame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
description: Section 2 of the HINE is used to assess motor milestones of the participants. It is composed of 8 motor milestone categories: voluntary grasp, ability to kick in supine position, head control, rolling, sitting, crawling, standing, and walking.
measure: Number of Participants with Ventilatory Support
time frame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
measure: Plasma Concentrations of Nusinersen Sodium Injection
time frame: Pre-dose and multiple time points post-dose on Day 0, post-dose on Day 28 and pre-dose every 4 months up to end of treatment (2 years)
measure: Cerebrospinal Fluid (CSF) Concentrations of Nusinersen Sodium Injection
time frame: pre-dose on Day 0, Day 63, and Every 4 Months Up to End of Treatment (2 years)
arm group label: All Participants
description: Participants with 5q SMA and who were prescribed with nusinersen sodium injection in China according to the local marketing authorization.
intervention type: Drug
intervention name: Nusinersen Sodium Injection
description: Administered as specified in the treatment arm.
arm group label: All Participants
other name: BIIB058
other name: ISIS 396443
other name: Spinraza
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Medical Director
role: Study Director
affiliation: Biogen
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: China
responsible party type: Sponsor
mesh term: Muscular Atrophy
mesh term: Muscular Atrophy, Spinal
sharing ipd: Yes
ipd description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
ipd url: https://vivli.org/