<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/NCT03232203</url>
  </required_header>
  <id_info>
    <org_study_id>STRIM-001</org_study_id>
    <nct_id>NCT03232203</nct_id>
  </id_info>
  <brief_title>Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)</brief_title>
  <official_title>Evaluation of Referring HCPs' and Parents'/Carers' Understanding of Specific Risks Associated With Strimvelis™ Treatment</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Orchard Therapeutics</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Orchard Therapeutics</source>
  <oversight_info>
    <has_dmc>No</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>
      STRIMVELIS is a medicinal product that restores adenosine deaminase (ADA) function in&#xD;
      hematopoietic cell lineages, thereby preventing impaired immune function. STRIMVELIS is&#xD;
      indicated for the treatment of patients with ADA- severe combined immunodeficiency (SCID),&#xD;
      for whom suitable human leukocyte antigen (HLA)-matched related stem cell donor is not&#xD;
      available. The objective of this study is to evaluate the effectiveness of routine and&#xD;
      additional risk minimization measures by assessing the understanding of referring health care&#xD;
      providers (HCPs) and parents/carers (hereby referred as participants) with regard to the&#xD;
      specific risks associated with STRIMVELIS. In this cross-sectional study, surveys will be&#xD;
      provided to referring HCPs and parents/carers of children approximately six months after&#xD;
      treatment with STRIMVELIS. The study will recruit for approximately two years or until a&#xD;
      maximum of 10 referring HCPs and 10 parents/carers have completed their respective surveys,&#xD;
      whichever occurs first.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">April 12, 2018</start_date>
  <completion_date type="Actual">June 25, 2021</completion_date>
  <primary_completion_date type="Actual">June 25, 2021</primary_completion_date>
  <study_type>Observational [Patient Registry]</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Cross-Sectional</time_perspective>
  </study_design_info>
  <target_duration>2 Years</target_duration>
  <primary_outcome>
    <measure>Proportion of HCPs providing the correct response</measure>
    <time_frame>Up to 2 years</time_frame>
    <description>A series of questions concerning the specific risks associated with STRIMVELIS will be asked during survey. Data from all survey respondents will be analyzed and reported as descriptive statistics.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Proportion of Parents/Carers providing the correct response</measure>
    <time_frame>Up to 2 years</time_frame>
    <description>A series of questions concerning the specific risks associated with STRIMVELIS will be asked during survey. Data from all survey respondents will be analyzed and reported as descriptive statistics.</description>
  </primary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Actual">16</enrollment>
  <condition>Severe Combined Immunodeficiency Due to ADA Deficiency</condition>
  <arm_group>
    <arm_group_label>Health care providers</arm_group_label>
    <description>A HCP survey instrument of approximately 20 questions will be provided. Survey questions will be based on the STRIMVELIS summary of product characteristics and educational materials</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Parent/carer</arm_group_label>
    <description>A parent/carer survey instrument of approximately 20 questions will be provided. Survey questions will be based on the STRIMVELIS Patient Information Leaflet and educational materials</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>STRIMVELIS</intervention_name>
    <description>It is the Autologous cluster of differentiation (CD) 34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA complementary Deoxyribonucleic acid (cDNA) sequence. HCP who have previously referred a patient for STRIMVELIS treatment or a parent's/carer's child who previously received treatment with STRIMVELIS will be recruited to the study</description>
    <arm_group_label>Health care providers</arm_group_label>
    <arm_group_label>Parent/carer</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Parents/carers and referring HCPs of patients enrolled within this registry or patients&#xD;
        treated with STRIMVELIS outside of the registry will be contacted separately and asked to&#xD;
        participate in their respective surveys evaluating the effectiveness of risk minimization&#xD;
        measures.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  HCPs or HCPs' close family members may not have been employees of Orchard,&#xD;
             Pharmaceutical Product Development, LLC (PPD), the Food and Drug Administration (FDA),&#xD;
             or the European Medicines Agency (EMA).&#xD;
&#xD;
          -  HCPs must be licensed&#xD;
&#xD;
          -  An HCP must not have previously completed a survey regarding STRIMVELIS educational&#xD;
             materials.&#xD;
&#xD;
          -  An HCP must have previously referred a patient for STRIMVELIS treatment.&#xD;
&#xD;
          -  Parents/carers or parents'/carers' close family members may not have been employees of&#xD;
             Orchard, PPD, FDA, or EMA.&#xD;
&#xD;
          -  A parent/carer must not have previously completed a survey regarding STRIMVELIS&#xD;
             educational materials.&#xD;
&#xD;
          -  A parent's or carer's child must have previously received treatment with STRIMVELIS&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  No exclusion criteria&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Clinical Trials</last_name>
    <role>Study Director</role>
    <affiliation>Orchard Therapeutics</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)</name>
      <address>
        <city>Milan</city>
        <zip>20132</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Italy</country>
  </location_countries>
  <verification_date>February 2022</verification_date>
  <study_first_submitted>July 25, 2017</study_first_submitted>
  <study_first_submitted_qc>July 25, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">July 27, 2017</study_first_posted>
  <last_update_submitted>February 18, 2022</last_update_submitted>
  <last_update_submitted_qc>February 18, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">February 21, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Human leukocyte antigen</keyword>
  <keyword>Key Risk Message</keyword>
  <keyword>ADA-SCID</keyword>
  <keyword>Health care providers</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Severe Combined Immunodeficiency</mesh_term>
  </condition_browse>
  <!-- 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/NCT03232203
org study id: STRIM-001
nct id: NCT03232203
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
STRIMVELIS is a medicinal product that restores adenosine deaminase (ADA) function in
      hematopoietic cell lineages, thereby preventing impaired immune function. STRIMVELIS is
      indicated for the treatment of patients with ADA- severe combined immunodeficiency (SCID),
      for whom suitable human leukocyte antigen (HLA)-matched related stem cell donor is not
      available. The objective of this study is to evaluate the effectiveness of routine and
      additional risk minimization measures by assessing the understanding of referring health care
      providers (HCPs) and parents/carers (hereby referred as participants) with regard to the
      specific risks associated with STRIMVELIS. In this cross-sectional study, surveys will be
      provided to referring HCPs and parents/carers of children approximately six months after
      treatment with STRIMVELIS. The study will recruit for approximately two years or until a
      maximum of 10 referring HCPs and 10 parents/carers have completed their respective surveys,
      whichever occurs first.
observational model: Cohort
time perspective: Cross-Sectional
measure: Proportion of HCPs providing the correct response
time frame: Up to 2 years
description: A series of questions concerning the specific risks associated with STRIMVELIS will be asked during survey. Data from all survey respondents will be analyzed and reported as descriptive statistics.
measure: Proportion of Parents/Carers providing the correct response
time frame: Up to 2 years
description: A series of questions concerning the specific risks associated with STRIMVELIS will be asked during survey. Data from all survey respondents will be analyzed and reported as descriptive statistics.
arm group label: Health care providers
description: A HCP survey instrument of approximately 20 questions will be provided. Survey questions will be based on the STRIMVELIS summary of product characteristics and educational materials
arm group label: Parent/carer
description: A parent/carer survey instrument of approximately 20 questions will be provided. Survey questions will be based on the STRIMVELIS Patient Information Leaflet and educational materials
intervention type: Drug
intervention name: STRIMVELIS
description: It is the Autologous cluster of differentiation (CD) 34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA complementary Deoxyribonucleic acid (cDNA) sequence. HCP who have previously referred a patient for STRIMVELIS treatment or a parent's/carer's child who previously received treatment with STRIMVELIS will be recruited to the study
arm group label: Health care providers
arm group label: Parent/carer
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: Accepts Healthy Volunteers
last name: Clinical Trials
role: Study Director
affiliation: Orchard Therapeutics
facility: 
country: Italy
responsible party type: Sponsor
mesh term: Severe Combined Immunodeficiency