<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/NCT04416178</url>
  </required_header>
  <id_info>
    <org_study_id>SCDGEN</org_study_id>
    <secondary_id>5U01HL133996</secondary_id>
    <nct_id>NCT04416178</nct_id>
  </id_info>
  <brief_title>Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders</brief_title>
  <official_title>Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>St. Jude Children's Research Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>National Heart, Lung, and Blood Institute (NHLBI)</agency>
      <agency_class>NIH</agency_class>
    </collaborator>
  </sponsors>
  <source>St. Jude Children's Research Hospital</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>
      The primary objectives of this prospective mixed-method interview study are to use&#xD;
      semi-structured interviews in parents of sickle cell disease (SCD) patients to describe&#xD;
      parental attitudes of research involving genomic sequencing, including concerns about&#xD;
      participation and expectations from researchers and to use surveys to quantitatively measure&#xD;
      genetic/genomic knowledge, trust in health care provider, and literacy/numeracy ability in&#xD;
      parents of children with SCD and adolescents with SCD.&#xD;
&#xD;
      Investigators hope to use the results of the planned surveys and interviews to reduce the&#xD;
      risk of misunderstanding about DNA and genetic research and build strong relationships&#xD;
      between SCD families and researchers in the future, and to design educational information and&#xD;
      study materials that will help parents with children with SCD understand important details&#xD;
      about DNA and genetic research.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      There is a critical gap in knowledge regarding the attitudes, beliefs, and expectations of&#xD;
      parents around clinical research trials involving genomic sequencing of children with sickle&#xD;
      cell disease (SCD). SCD primarily affects children of African American (Black) race;&#xD;
      institutionally we have found differences in enrollment on genomic sequencing trials (G4K&#xD;
      (NCT02530658), PG4KDS) with patients identifying as black more likely to decline enrollment.&#xD;
      Enrollment on SCCRIP (NCT02098863), a biobank study for children with SCD is high (92.3%),&#xD;
      indicating that potential genomic research does not appear to concern many families with SCD.&#xD;
      Given the rising prevalence of clinical research involving genomic sequencing in pediatric&#xD;
      SCD, coupled with the increasing likelihood that sequencing will be required for enrollment&#xD;
      on therapeutic drug or gene therapy trials, there is a clear need for research to better&#xD;
      understand stakeholder concerns and expectations around genomic sequencing in this&#xD;
      population.&#xD;
&#xD;
      Parents of children with SCD and adolescent patients will be approached to complete a short&#xD;
      survey during a routine clinic visit, in the medicine room or in-patient. Survey questions&#xD;
      will be administered at the time of the informed consent conversation. Those who agree will&#xD;
      be given a paper survey or an ipad to complete survey items which focus on genetic/genomic&#xD;
      knowledge, trust in health care provider, and literacy/numeracy ability in parents of&#xD;
      children with SCD and adolescents with SCD. Participants also have the option to have&#xD;
      questions read to them or they can take the survey on paper. Completion of the survey is&#xD;
      expected to take &lt; 30 minutes. Patient and parent can complete surveys simultaneously.&#xD;
&#xD;
      Of parents completing surveys, a subset will be approached for a private (in person or&#xD;
      virtual) semi-structured interview. Participants willing to be interviewed will be&#xD;
      interviewed at either the same study visit or at a future visit if this is more convenient&#xD;
      for the participant. The interview guide (member of the study team) will ask questions&#xD;
      designed to first assess parental perceptions about clinical research then begin to focus on&#xD;
      parental attitudes, beliefs, and expectations around research involving clinical genomics.&#xD;
      Interviews will be conducted on-site at St. Jude Children's Research Hospital in a private,&#xD;
      quiet area. The interview should last 30-60 minutes and will be audio recorded.&#xD;
&#xD;
      Some survey or interview study visits may occur remotely.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">December 17, 2020</start_date>
  <completion_date type="Anticipated">February 2024</completion_date>
  <primary_completion_date type="Anticipated">February 2024</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>Use of semi-structured interviews in parents of SCD patients to qualitatively describe parental attitudes of research involving genomic sequencing, including concerns about participation and expectations from researchers</measure>
    <time_frame>Day 1, or at a future visit (up to approximately 1 year)</time_frame>
    <description>Interviews will be audio recorded, transcribed verbatim and analyzed using semantic content analysis to identify common themes</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Use of surveys to quantitatively measure genetic/genomic knowledge, trust in health care provider/researchers, and literacy/numeracy ability in parents of children with SCD and adolescents with SCD.</measure>
    <time_frame>Day 1</time_frame>
    <description>Patients and parents' demographic characteristics will be collected from the electronic medical record (EMR). Participants will complete various survey instruments designed to measure knowledge and attitudes around genetic testing and biobanks, self-reported literacy and numeracy, and trust in providers. Data will be analyzed quantitatively using descriptive statistics, generalized linear regression models and generalized estimation equations.</description>
  </primary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Actual">300</enrollment>
  <condition>Sickle Cell Disease</condition>
  <arm_group>
    <arm_group_label>Parents of children with SCD</arm_group_label>
    <description>Parent of child with HbSS, HbS/ β0thalassemia, or HbSC aged 12 months to 18 years at study initiation</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Adolescents with SCD</arm_group_label>
    <description>Patient aged 13-18 with HbSS, HbS/ β0thalassemia, or HbSC</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Those who meet the Eligibility criteria&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Parent of child with HbSS, HbS/ β0thalassemia, or HbSC aged 12 months to 18 years at&#xD;
             study initiation, irrespective of clinical severity or patient aged 13-18 with&#xD;
             aforementioned SCD genotype.&#xD;
&#xD;
          -  Informed consent from parent or legal guardian and assent of adolescent participant.&#xD;
&#xD;
          -  Has been previously approached for SCRIPP.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Participants who are unable to converse fluently in English will be excluded.&#xD;
             (Permanent)&#xD;
&#xD;
          -  Condition or chronic illness, which in the opinion of the PI/Co-I, makes participation&#xD;
             unsafe or untenable (i.e. cognitive impairment, concurrent acute morbidity).&#xD;
             Participant may be re-evaluated.&#xD;
&#xD;
          -  Inability or unwillingness of research participant or legal guardian/representative to&#xD;
             give written informed consent.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>13 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Liza M. Johnson, MD, MPH, MSB</last_name>
    <role>Principal Investigator</role>
    <affiliation>St. Jude Children's Research Hospital</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>St. Jude Children's Research Hospital</name>
      <address>
        <city>Memphis</city>
        <state>Tennessee</state>
        <zip>38105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://stjude.org</url>
    <description>St. Jude Children's Research Hospital</description>
  </link>
  <link>
    <url>http://www.stjude.org/protocols</url>
    <description>Clinical Trials Open at St. Jude</description>
  </link>
  <verification_date>August 2023</verification_date>
  <study_first_submitted>May 27, 2020</study_first_submitted>
  <study_first_submitted_qc>June 1, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 4, 2020</study_first_posted>
  <last_update_submitted>August 18, 2023</last_update_submitted>
  <last_update_submitted_qc>August 18, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">August 21, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Sickle Cell Disease</keyword>
  <keyword>Sickle Cell Anemia</keyword>
  <keyword>Hemoglobin SC Disease</keyword>
  <keyword>Hemoglobin SS Disease</keyword>
  <keyword>Hemoglobin S beta zero thalassemia</keyword>
  <keyword>Adolescent</keyword>
  <keyword>Parent</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Anemia, Sickle Cell</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/NCT04416178
org study id: SCDGEN
secondary id: 5U01HL133996
nct id: NCT04416178
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The primary objectives of this prospective mixed-method interview study are to use
      semi-structured interviews in parents of sickle cell disease (SCD) patients to describe
      parental attitudes of research involving genomic sequencing, including concerns about
      participation and expectations from researchers and to use surveys to quantitatively measure
      genetic/genomic knowledge, trust in health care provider, and literacy/numeracy ability in
      parents of children with SCD and adolescents with SCD.

      Investigators hope to use the results of the planned surveys and interviews to reduce the
      risk of misunderstanding about DNA and genetic research and build strong relationships
      between SCD families and researchers in the future, and to design educational information and
      study materials that will help parents with children with SCD understand important details
      about DNA and genetic research.
There is a critical gap in knowledge regarding the attitudes, beliefs, and expectations of
      parents around clinical research trials involving genomic sequencing of children with sickle
      cell disease (SCD). SCD primarily affects children of African American (Black) race;
      institutionally we have found differences in enrollment on genomic sequencing trials (G4K
      (NCT02530658), PG4KDS) with patients identifying as black more likely to decline enrollment.
      Enrollment on SCCRIP (NCT02098863), a biobank study for children with SCD is high (92.3%),
      indicating that potential genomic research does not appear to concern many families with SCD.
      Given the rising prevalence of clinical research involving genomic sequencing in pediatric
      SCD, coupled with the increasing likelihood that sequencing will be required for enrollment
      on therapeutic drug or gene therapy trials, there is a clear need for research to better
      understand stakeholder concerns and expectations around genomic sequencing in this
      population.

      Parents of children with SCD and adolescent patients will be approached to complete a short
      survey during a routine clinic visit, in the medicine room or in-patient. Survey questions
      will be administered at the time of the informed consent conversation. Those who agree will
      be given a paper survey or an ipad to complete survey items which focus on genetic/genomic
      knowledge, trust in health care provider, and literacy/numeracy ability in parents of
      children with SCD and adolescents with SCD. Participants also have the option to have
      questions read to them or they can take the survey on paper. Completion of the survey is
      expected to take < 30 minutes. Patient and parent can complete surveys simultaneously.

      Of parents completing surveys, a subset will be approached for a private (in person or
      virtual) semi-structured interview. Participants willing to be interviewed will be
      interviewed at either the same study visit or at a future visit if this is more convenient
      for the participant. The interview guide (member of the study team) will ask questions
      designed to first assess parental perceptions about clinical research then begin to focus on
      parental attitudes, beliefs, and expectations around research involving clinical genomics.
      Interviews will be conducted on-site at St. Jude Children's Research Hospital in a private,
      quiet area. The interview should last 30-60 minutes and will be audio recorded.

      Some survey or interview study visits may occur remotely.
observational model: Cohort
time perspective: Prospective
measure: Use of semi-structured interviews in parents of SCD patients to qualitatively describe parental attitudes of research involving genomic sequencing, including concerns about participation and expectations from researchers
time frame: Day 1, or at a future visit (up to approximately 1 year)
description: Interviews will be audio recorded, transcribed verbatim and analyzed using semantic content analysis to identify common themes
measure: Use of surveys to quantitatively measure genetic/genomic knowledge, trust in health care provider/researchers, and literacy/numeracy ability in parents of children with SCD and adolescents with SCD.
time frame: Day 1
description: Patients and parents' demographic characteristics will be collected from the electronic medical record (EMR). Participants will complete various survey instruments designed to measure knowledge and attitudes around genetic testing and biobanks, self-reported literacy and numeracy, and trust in providers. Data will be analyzed quantitatively using descriptive statistics, generalized linear regression models and generalized estimation equations.
arm group label: Parents of children with SCD
description: Parent of child with HbSS, HbS/ β0thalassemia, or HbSC aged 12 months to 18 years at study initiation
arm group label: Adolescents with SCD
description: Patient aged 13-18 with HbSS, HbS/ β0thalassemia, or HbSC
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 13 Years
maximum age: N/A
healthy volunteers: Accepts Healthy Volunteers
last name: Liza M. Johnson, MD, MPH, MSB
role: Principal Investigator
affiliation: St. Jude Children's Research Hospital
facility: 
country: United States
url: http://stjude.org
description: St. Jude Children's Research Hospital
url: http://www.stjude.org/protocols
description: Clinical Trials Open at St. Jude
responsible party type: Sponsor
mesh term: Anemia, Sickle Cell