<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/NCT04469894</url>
  </required_header>
  <id_info>
    <org_study_id>NNPDF-BUR001</org_study_id>
    <nct_id>NCT04469894</nct_id>
  </id_info>
  <brief_title>Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick</brief_title>
  <official_title>Understanding Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick Disease Through the Eyes of Patients and Families</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>National Niemann-Pick Disease Foundation</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Engage Health Inc.</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
    <collaborator>
      <agency>Icahn School of Medicine at Mount Sinai</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>National Niemann-Pick Disease Foundation</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>
      This study is a US based qualitative PRO research study to document the health insurance&#xD;
      literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired&#xD;
      care, services and medications for patients.&#xD;
&#xD;
      The outcome of this research will be used to inform various other workstreams as NNPDF works&#xD;
      to assist families.&#xD;
&#xD;
      The core research objectives are to understand the following from Niemann-Pick patients and&#xD;
      their families in the US&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This study is a US based qualitative PRO research study to document the health insurance&#xD;
      literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired&#xD;
      care, services and medications for patients.&#xD;
&#xD;
      The outcome of this research will be used to inform various other workstreams as NNPDF works&#xD;
      to assist families.&#xD;
&#xD;
      The core research objectives are to understand the following from Niemann-Pick patients and&#xD;
      their families in the US;&#xD;
&#xD;
        -  Level of health insurance literacy through assessment of health insurance terminology,&#xD;
           and self-reported insights regarding information seeking, document literacy and&#xD;
           cognitive skills&#xD;
&#xD;
        -  If they have healthcare insurance, and if not, why not&#xD;
&#xD;
        -  Types (including name) of insurance by which Niemann-Pick families are covered&#xD;
&#xD;
        -  Information regarding Medicaid Waivers&#xD;
&#xD;
        -  Attributes of insurance plans including, but not limited to;&#xD;
&#xD;
             -  Deductible amounts for individual and family&#xD;
&#xD;
             -  Out of pocket maximums for individual and family&#xD;
&#xD;
             -  Insurance premiums&#xD;
&#xD;
             -  Associated Health Savings Accounts&#xD;
&#xD;
             -  Associated Cost Sharing requirements&#xD;
&#xD;
             -  Services, drugs and assistive devices covered&#xD;
&#xD;
        -  What the perceived out-of-pocket costs are for Niemann-Pick families (annually)&#xD;
&#xD;
        -  When out-of-pocket maximums are perceived to be met for Niemann-Pick families&#xD;
&#xD;
        -  Patient and HCP experience in terms of challenges with accessing desired care and&#xD;
           coverage&#xD;
&#xD;
             -  Where are families experiencing challenges with accessing desired care and&#xD;
                coverage? (drugs, assistive devices, services) Is it different for the different&#xD;
                phenotypes of the disease?&#xD;
&#xD;
             -  What is the challenge? (financial, time, stress, others)&#xD;
&#xD;
             -  What is the life impact of that challenge? (work and educational impact, lack or&#xD;
                delay of treatment, others)&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">June 15, 2020</start_date>
  <completion_date type="Actual">September 21, 2020</completion_date>
  <primary_completion_date type="Actual">September 21, 2020</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>Health Insurance Literacy</measure>
    <time_frame>July 2020- September</time_frame>
    <description>Health insurance literacy will be measured by a score on a brief quiz regarding health insurance key terms as well as self-ratings regarding various aspects of the participants' ability to navigate and understand insurance</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Health Insurance Coverage</measure>
    <time_frame>July 2020- September</time_frame>
    <description>Health insurance coverage will be measured by the different multiple choice and open-ended questions based on the services available under the participants' insurance plan as well as their reported difficulty with navigating their plan and obtaining what they need for Niemann-Pick</description>
  </primary_outcome>
  <number_of_groups>7</number_of_groups>
  <enrollment type="Actual">76</enrollment>
  <condition>Niemann-Pick Diseases</condition>
  <arm_group>
    <arm_group_label>Niemann-Pick Type A</arm_group_label>
    <description>Also referred to as Infantile Neurovisceral ASMD</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Niemann-Pick Type A/B</arm_group_label>
    <description>Also referred to as Intermediate form or Chronic Neurovisceral ASMD</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Niemann-Pick Type B</arm_group_label>
    <description>Also referred to as Chronic Visceral ASMD</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Niemann-Pick Type C (Early Infantile)</arm_group_label>
    <description>Onset at less than 2 years of age</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Niemann-Pick Type C (Late Infantile)</arm_group_label>
    <description>Neurodegenerative form (late-infantile) onset at 2-6 years of age</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Niemann-Pick Type C (Juvenile)</arm_group_label>
    <description>Neurodegenerative form (juvenile) onset at 6-15 years of age</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Niemann-Pick Type C (Adult)</arm_group_label>
    <description>Psychiatric neurodegenerative form (adult) onset at greater than 15 years of age</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Within the United States, patients or the parent/caregiver of patients who have been&#xD;
        diagnosed with any of the 7 known subtypes of Niemann-Pick disease&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Participant must be a person with Niemann-Pick disease who is 18 years or older or The&#xD;
             parent/legal guardian of a person with Niemann-Pick disease. Please note: Parents&#xD;
             whose child has passed on are able to participate in the RSVP. Parents whose child has&#xD;
             passed on in the last two years are also able to participate in the interview. Please&#xD;
             note that only one family member will be eligible to complete the RSVP and interview&#xD;
             for one family.&#xD;
&#xD;
          -  Confirmed diagnosis of Niemann-Pick disease, confirmed by membership in the NNPDF or&#xD;
             by provision of a proof of disease form&#xD;
&#xD;
          -  Able to read, write and communicate in English&#xD;
&#xD;
          -  Able to grant informed consent&#xD;
&#xD;
          -  Willing to complete a survey and RSVP, and to participate in a 30-minute telephone&#xD;
             interview&#xD;
&#xD;
          -  Ability to view or receive a document from the interviewer before or during the&#xD;
             interview (web browser, ability to receive a text, fax or document by mail)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        - Inability to meet any of the above 6 criteria&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>George A Diaz, MD, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Icahn School of Medicine at Mount Sinai</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Justin Hopkin, MD</last_name>
    <role>Study Chair</role>
    <affiliation>National Niemann-Pick Disease Board Chair</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Icahn School of Medicine at Mount Sinai</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10029</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>October 2020</verification_date>
  <study_first_submitted>July 10, 2020</study_first_submitted>
  <study_first_submitted_qc>July 10, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 14, 2020</study_first_posted>
  <last_update_submitted>October 5, 2020</last_update_submitted>
  <last_update_submitted_qc>October 5, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">October 6, 2020</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>Pick Disease of the Brain</mesh_term>
    <mesh_term>Aphasia, Primary Progressive</mesh_term>
    <mesh_term>Frontotemporal Dementia</mesh_term>
    <mesh_term>Niemann-Pick Diseases</mesh_term>
    <mesh_term>Niemann-Pick Disease, Type A</mesh_term>
    <mesh_term>Niemann-Pick Disease, Type C</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>The data for each question in the study will be analyzed and reported by Engage Health using descriptive statistics as appropriate for the nature of the data collected-frequency distributions, cross tabulations and measures of central tendency and dispersion.</ipd_description>
    <ipd_info_type>Study Protocol</ipd_info_type>
    <ipd_info_type>Statistical Analysis Plan (SAP)</ipd_info_type>
    <ipd_info_type>Informed Consent Form (ICF)</ipd_info_type>
    <ipd_info_type>Analytic Code</ipd_info_type>
    <ipd_time_frame>September 2020</ipd_time_frame>
  </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/NCT04469894
org study id: NNPDF-BUR001
nct id: NCT04469894
lead sponsor: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This study is a US based qualitative PRO research study to document the health insurance
      literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired
      care, services and medications for patients.

      The outcome of this research will be used to inform various other workstreams as NNPDF works
      to assist families.

      The core research objectives are to understand the following from Niemann-Pick patients and
      their families in the US
This study is a US based qualitative PRO research study to document the health insurance
      literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired
      care, services and medications for patients.

      The outcome of this research will be used to inform various other workstreams as NNPDF works
      to assist families.

      The core research objectives are to understand the following from Niemann-Pick patients and
      their families in the US;

        -  Level of health insurance literacy through assessment of health insurance terminology,
           and self-reported insights regarding information seeking, document literacy and
           cognitive skills

        -  If they have healthcare insurance, and if not, why not

        -  Types (including name) of insurance by which Niemann-Pick families are covered

        -  Information regarding Medicaid Waivers

        -  Attributes of insurance plans including, but not limited to;

             -  Deductible amounts for individual and family

             -  Out of pocket maximums for individual and family

             -  Insurance premiums

             -  Associated Health Savings Accounts

             -  Associated Cost Sharing requirements

             -  Services, drugs and assistive devices covered

        -  What the perceived out-of-pocket costs are for Niemann-Pick families (annually)

        -  When out-of-pocket maximums are perceived to be met for Niemann-Pick families

        -  Patient and HCP experience in terms of challenges with accessing desired care and
           coverage

             -  Where are families experiencing challenges with accessing desired care and
                coverage? (drugs, assistive devices, services) Is it different for the different
                phenotypes of the disease?

             -  What is the challenge? (financial, time, stress, others)

             -  What is the life impact of that challenge? (work and educational impact, lack or
                delay of treatment, others)
observational model: Cohort
time perspective: Prospective
measure: Health Insurance Literacy
time frame: July 2020- September
description: Health insurance literacy will be measured by a score on a brief quiz regarding health insurance key terms as well as self-ratings regarding various aspects of the participants' ability to navigate and understand insurance
measure: Health Insurance Coverage
time frame: July 2020- September
description: Health insurance coverage will be measured by the different multiple choice and open-ended questions based on the services available under the participants' insurance plan as well as their reported difficulty with navigating their plan and obtaining what they need for Niemann-Pick
arm group label: Niemann-Pick Type A
description: Also referred to as Infantile Neurovisceral ASMD
arm group label: Niemann-Pick Type A/B
description: Also referred to as Intermediate form or Chronic Neurovisceral ASMD
arm group label: Niemann-Pick Type B
description: Also referred to as Chronic Visceral ASMD
arm group label: Niemann-Pick Type C (Early Infantile)
description: Onset at less than 2 years of age
arm group label: Niemann-Pick Type C (Late Infantile)
description: Neurodegenerative form (late-infantile) onset at 2-6 years of age
arm group label: Niemann-Pick Type C (Juvenile)
description: Neurodegenerative form (juvenile) onset at 6-15 years of age
arm group label: Niemann-Pick Type C (Adult)
description: Psychiatric neurodegenerative form (adult) onset at greater than 15 years of age
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: George A Diaz, MD, PhD
role: Principal Investigator
affiliation: Icahn School of Medicine at Mount Sinai
last name: Justin Hopkin, MD
role: Study Chair
affiliation: National Niemann-Pick Disease Board Chair
facility: 
country: United States
responsible party type: Sponsor
mesh term: Pick Disease of the Brain
mesh term: Aphasia, Primary Progressive
mesh term: Frontotemporal Dementia
mesh term: Niemann-Pick Diseases
mesh term: Niemann-Pick Disease, Type A
mesh term: Niemann-Pick Disease, Type C
sharing ipd: Yes
ipd description: The data for each question in the study will be analyzed and reported by Engage Health using descriptive statistics as appropriate for the nature of the data collected-frequency distributions, cross tabulations and measures of central tendency and dispersion.
ipd info type: Study Protocol
ipd info type: Statistical Analysis Plan (SAP)
ipd info type: Informed Consent Form (ICF)
ipd info type: Analytic Code
ipd time frame: September 2020