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/NCT06019637</url>
    </required_header>
    <id_info>
      <org_study_id>COAV101ABR01</org_study_id>
      <nct_id>NCT06019637</nct_id>
    </id_info>
    <brief_title>A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma</brief_title>
    <acronym>ARISER</acronym>
    <official_title>A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Novartis Pharmaceuticals</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Novartis</source>
    <brief_summary>
      <textblock>
        A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular&#xD;
        Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        This study is a non-interventional Post Authorization Safety Study (PASS) to evaluate&#xD;
        long-term, real-world safety data of Brazilian pediatric patients diagnosed with SMA and&#xD;
        treated with Onasemnogene Abeparvovec (Zolgensma®) for up to 15 years after the treatment.&#xD;
        This study will support the benefit-risk assessment of Onasemnogene Abeparvovec in the&#xD;
        approved indications and may also allow for detection of new safety signals and provide&#xD;
        further guidance on the management of safety risks associated with Onasemnogene Abeparvovec&#xD;
        to patients/caregivers, health care providers (HCPs) and treating physicians, as required by&#xD;
        Brazilian Health Authority ANVISA as a conditional measure for granting Zolgensma®'s&#xD;
        authorization.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">October 27, 2023</start_date>
    <completion_date type="Anticipated">August 2, 2038</completion_date>
    <primary_completion_date type="Anticipated">August 2, 2038</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>Other</time_perspective>
    </study_design_info>
    <primary_outcome>
      <measure>Incidence and severity of treatment-emergent SAEs</measure>
      <time_frame>Up to 5 years</time_frame>
      <description>Incidence and severity of treatment-emergent Serious AEs (SAEs)</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>Number of patients who experience at least one AESI and number of patients by AESI</measure>
      <time_frame>Up to 15 years</time_frame>
      <description>Adverse Event of Special Interest: thrombocytopenia, hepatotoxicity, thrombotic microangiopathy (TMA), dorsal root ganglia toxicity, cardiac AEs, new malignancies, new incidence of neurologic, autoimmune, and hematologic disorder</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of patients who have survived and have not required permanent ventilatory assistance</measure>
      <time_frame>Up to 15 years</time_frame>
      <description>Evaluation of permanent ventilatory assistance, defined as requiring invasive ventilation(tracheostomy), or respiratory assistance for 16 or more hours per day (including non-invasiveventilatory support) continuously for 14 or more days in the absence of an acute reversible illness,excluding perioperative ventilation.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Time until death or the need for permanent ventilatory assistance</measure>
      <time_frame>Up to 15 years</time_frame>
      <description>Evaluation of permanent ventilatory assistance, defined as requiring invasive ventilation(tracheostomy), or respiratory assistance for 16 or more hours per day (including non-invasiveventilatory support) continuously for 14 or more days in the absence of an acute reversible illness,excluding perioperative ventilation.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of patients who achieve each Developmental Motor Milestone</measure>
      <time_frame>Up to 15 years</time_frame>
      <description>Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Number of patients who achieve each WHO Developmental Milestone within age percentiles</measure>
      <time_frame>Up to 15 years</time_frame>
      <description>Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.</description>
    </secondary_outcome>
    <number_of_groups>1</number_of_groups>
    <enrollment type="Anticipated">50</enrollment>
    <condition>Spinal Muscular Atrophies</condition>
    <arm_group>
      <arm_group_label>Spinal Muscular Atrophy Patients</arm_group_label>
      <description>Brazilian pediatric patients with a confirmed diagnosis of Spinal Muscular Atrophy treated with Onasemnogene Abeparvovec</description>
    </arm_group>
    <intervention>
      <intervention_type>Other</intervention_type>
      <intervention_name>Onasemnogene Abeparvovec</intervention_name>
      <description>Retrospective and prospective observational study. There is no treatment allocation.</description>
      <arm_group_label>Spinal Muscular Atrophy Patients</arm_group_label>
      <other_name>Zolgensma</other_name>
    </intervention>
    <eligibility>
      <study_pop>
        <textblock>
          The study population will consist of approximately 50 Brazilian pediatric patients with SMA&#xD;
          who were treated with Onasemnogene Abeparvovec (Zolgensma®) in the commercial setting, the&#xD;
          closed MAP, or the phase IV OFELIA trial. Patients will be recruited in up to 3 centers in&#xD;
          Brazil, over one year of recruitment.&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Non-Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Subject's parent or legal guardian has provided signed eICF.&#xD;
  &#xD;
            -  Subject with SMA, genetically confirmed: with a bi-allelic mutation in the SMN1 gene,&#xD;
               and a clinical diagnosis of SMA Type 1 or up to 3 copies of the SMN2 gene.&#xD;
  &#xD;
            -  Subject treated* with Onasemnogene Abeparvovec (Zolgensma®) prior to enrolling in this&#xD;
               study.&#xD;
  &#xD;
          Subjects treated with nusinersen or risdiplam prior to Onasemnogene Abeparvovec&#xD;
          (Zolgensma®) can be enrolled if currently not receiving it.&#xD;
  &#xD;
          *Subjects can be enrolled in this study on the day treated with Onasemnogene Abeparvovec&#xD;
          (Zolgensma®) or if prior medical history is available to complete all assessments&#xD;
          retrospectively, in accordance with local ethical requirements.&#xD;
  &#xD;
            -  Subject and parent/guardian are willing and able to comply with the phone contacts&#xD;
               through the course of the study&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Patients currently enrolled in any interventional clinical trial** other than the&#xD;
               phase IV OFELIA trial will be excluded from the study.&#xD;
  &#xD;
                 -  Subjects who were enrolled in a clinical trial (independently of the disease&#xD;
                    indication and interventional treatment) but are not currently enrolled, can be&#xD;
                    included in this study.&#xD;
  &#xD;
          During the follow-up, subjects who enroll any clinical trial with pharmacological&#xD;
          intervention will discontinue from this 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>Novartis Pharmaceuticals</last_name>
      <role>Study Director</role>
      <affiliation>Novartis Pharmaceuticals</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Novartis Pharmaceuticals</last_name>
      <phone>+41613241111</phone>
      <email>novartis.email@novartis.com</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Novartis Pharmaceuticals</last_name>
    </overall_contact_backup>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 25, 2023</study_first_submitted>
    <study_first_submitted_qc>August 25, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 31, 2023</study_first_posted>
    <last_update_submitted>August 25, 2023</last_update_submitted>
    <last_update_submitted_qc>August 25, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 31, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>Spinal Muscular Atrophies;</keyword>
    <keyword>Onasemnogene Abeparvovec;</keyword>
    <keyword>Brazil;</keyword>
    <keyword>NIS</keyword>
    <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>
      <mesh_term>Atrophy</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
    </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/NCT06019637
  org study id: COAV101ABR01
  nct id: NCT06019637
  lead sponsor: 
  A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular
        Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
  This study is a non-interventional Post Authorization Safety Study (PASS) to evaluate
        long-term, real-world safety data of Brazilian pediatric patients diagnosed with SMA and
        treated with Onasemnogene Abeparvovec (Zolgensma®) for up to 15 years after the treatment.
        This study will support the benefit-risk assessment of Onasemnogene Abeparvovec in the
        approved indications and may also allow for detection of new safety signals and provide
        further guidance on the management of safety risks associated with Onasemnogene Abeparvovec
        to patients/caregivers, health care providers (HCPs) and treating physicians, as required by
        Brazilian Health Authority ANVISA as a conditional measure for granting Zolgensma®'s
        authorization.
  observational model: Cohort
  time perspective: Other
  measure: Incidence and severity of treatment-emergent SAEs
  time frame: Up to 5 years
  description: Incidence and severity of treatment-emergent Serious AEs (SAEs)
  measure: Number of patients who experience at least one AESI and number of patients by AESI
  time frame: Up to 15 years
  description: Adverse Event of Special Interest: thrombocytopenia, hepatotoxicity, thrombotic microangiopathy (TMA), dorsal root ganglia toxicity, cardiac AEs, new malignancies, new incidence of neurologic, autoimmune, and hematologic disorder
  measure: Number of patients who have survived and have not required permanent ventilatory assistance
  time frame: Up to 15 years
  description: Evaluation of permanent ventilatory assistance, defined as requiring invasive ventilation(tracheostomy), or respiratory assistance for 16 or more hours per day (including non-invasiveventilatory support) continuously for 14 or more days in the absence of an acute reversible illness,excluding perioperative ventilation.
  measure: Time until death or the need for permanent ventilatory assistance
  time frame: Up to 15 years
  description: Evaluation of permanent ventilatory assistance, defined as requiring invasive ventilation(tracheostomy), or respiratory assistance for 16 or more hours per day (including non-invasiveventilatory support) continuously for 14 or more days in the absence of an acute reversible illness,excluding perioperative ventilation.
  measure: Number of patients who achieve each Developmental Motor Milestone
  time frame: Up to 15 years
  description: Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.
  measure: Number of patients who achieve each WHO Developmental Milestone within age percentiles
  time frame: Up to 15 years
  description: Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.
  arm group label: Spinal Muscular Atrophy Patients
  description: Brazilian pediatric patients with a confirmed diagnosis of Spinal Muscular Atrophy treated with Onasemnogene Abeparvovec
  intervention type: Other
  intervention name: Onasemnogene Abeparvovec
  description: Retrospective and prospective observational study. There is no treatment allocation.
  arm group label: Spinal Muscular Atrophy Patients
  other name: Zolgensma
  study pop: 
  sampling method: Non-Probability Sample
  criteria: 
  gender: All
  minimum age: N/A
  maximum age: N/A
  healthy volunteers: No
  last name: Novartis Pharmaceuticals
  role: Study Director
  affiliation: Novartis Pharmaceuticals
  last name: Novartis Pharmaceuticals
  phone: +41613241111
  email: novartis.email@novartis.com
  last name: Novartis Pharmaceuticals
  responsible party type: Sponsor
  mesh term: Muscular Atrophy
  mesh term: Muscular Atrophy, Spinal
  mesh term: Atrophy
  sharing ipd: No