<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/NCT03231644</url>
  </required_header>
  <id_info>
    <org_study_id>Pro00018980.</org_study_id>
    <nct_id>NCT03231644</nct_id>
  </id_info>
  <brief_title>Fibrous Dysplasia, McCune-Albright Syndrome Patient Registry</brief_title>
  <official_title>Fibrous Dysplasia, McCune-Albright Syndrome Patient Registry</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Tovah Burstein</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Fibrous Dysplasia Foundation</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      The FD/MAS Patient Registry is an IRB-approved research study that that invites the patients&#xD;
      and families to help answer some of the biggest questions about FD/MAS by completing&#xD;
      questionnaires about their lives with FD or MAS.&#xD;
&#xD;
      Have you enrolled in the FD/MAS Patient Registry yet? Are you up-to-date on your surveys?&#xD;
      Take a trip to www.fdmasregistry.org today to learn more about the project, enroll, complete&#xD;
      your surveys, or make sure you aren't due to provide more info!&#xD;
&#xD;
      The FD/MAS Patient Registry: Your story powers research.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The FD/MAS Patient Registry is an IRB approved research project that allows patients and&#xD;
      families to share their experiences with fibrous dysplasia/McCune-Albright syndrome (FD/MAS)&#xD;
      by completing a series of surveys.&#xD;
&#xD;
      The surveys were created in collaboration with patients, parents, clinicians and researchers,&#xD;
      so that the data can be used to answer some of the most important questions about FD/MAS,&#xD;
      including:&#xD;
&#xD;
      the way the disease develops over time (its &quot;natural history&quot;), the patient experience of the&#xD;
      disease, and its impact on quality of life, how and when diagnoses are made, the scope of&#xD;
      treatments in use, what surgical techniques work best, and for whom, what other medical&#xD;
      interventions work best, and for whom, what social services and therapies are useful, the&#xD;
      costs of care to patients and their families, issues that concern patients (such as the&#xD;
      impact of pregnancy on FD, or children feeling different and facing stigma), and which&#xD;
      research questions and support programs you think are important to fund.&#xD;
&#xD;
      Participation is free and convenient for people with FD/MAS and their legal guardians. You&#xD;
      can join today at www.fdmasregistry.org.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">October 31, 2016</start_date>
  <completion_date type="Anticipated">October 2026</completion_date>
  <primary_completion_date type="Anticipated">October 2026</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>Treatment satisfaction</measure>
    <time_frame>Through study completion, an average of every 2 years</time_frame>
    <description>FACIT-Treatment Satisfaction scale</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Perceived symptoms of pain</measure>
    <time_frame>Through study completion, an average of every 2 years</time_frame>
    <description>Brief Pain Inventory</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Depression/anxiety</measure>
    <time_frame>Through study completion, an average of every 2 years</time_frame>
    <description>Hospital Anxiety Depression Scale</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Stigma</measure>
    <time_frame>Through study completion, an average of every 2 years</time_frame>
    <description>NeuroQol Pediatric and Adult Stigma short forms</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Health-related Quality of Life</measure>
    <time_frame>Through study completion, an average of every 2 years</time_frame>
    <description>SF-36,PedsQL 4.0</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Financial health</measure>
    <time_frame>Through study completion, an average of every 2 years</time_frame>
    <description>FACIT-Cost</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">600</enrollment>
  <condition>Fibrous Dysplasia</condition>
  <condition>McCune Albright Syndrome</condition>
  <condition>Mazabraud Syndrome</condition>
  <arm_group>
    <arm_group_label>FD/MAS Patients</arm_group_label>
    <description>Patients with fibrous dysplasia and/or McCune-Albright syndrome and related disorders.</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Community sample of all patients in these particular disease communities.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria any one or more of the following:&#xD;
&#xD;
          -  clinical diagnosis of fibrous dysplasia&#xD;
&#xD;
          -  clinical diagnosis of McCune-Albright syndrome&#xD;
&#xD;
          -  clinical diagnosis of Mazabraud's syndrome&#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_contact>
    <last_name>Carmel Shemmesh-Rafalowsky</last_name>
    <email>PI.Registry@fibrousdysplasia.org</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Tovah Burstein</last_name>
    <phone>603-325-2489</phone>
    <email>TBurstein@fibrousdysplasia.org</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Tovah Burstein</name>
      <address>
        <city>Bethesda</city>
        <state>Maryland</state>
        <zip>20816-2558</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Tovah Burstein, MFA</last_name>
      <phone>603-325-2489</phone>
      <email>info@fibrousdysplasia.org</email>
    </contact>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>February 2023</verification_date>
  <study_first_submitted>July 20, 2017</study_first_submitted>
  <study_first_submitted_qc>July 24, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">July 27, 2017</study_first_posted>
  <last_update_submitted>February 13, 2023</last_update_submitted>
  <last_update_submitted_qc>February 13, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">February 14, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Fibrous Dysplasia Foundation</investigator_affiliation>
    <investigator_full_name>Tovah Burstein</investigator_full_name>
    <investigator_title>Community Engagement Director</investigator_title>
  </responsible_party>
  <keyword>Fibrous Dysplasia</keyword>
  <keyword>McCune-Albright Syndrome</keyword>
  <keyword>Mazabrauds</keyword>
  <keyword>FD/MAS</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Fibrous Dysplasia of Bone</mesh_term>
    <mesh_term>Fibrous Dysplasia, Polyostotic</mesh_term>
    <mesh_term>Syndrome</mesh_term>
    <mesh_term>Hyperplasia</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/NCT03231644
org study id: Pro00018980.
nct id: NCT03231644
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
is us export: No
The FD/MAS Patient Registry is an IRB-approved research study that that invites the patients
      and families to help answer some of the biggest questions about FD/MAS by completing
      questionnaires about their lives with FD or MAS.

      Have you enrolled in the FD/MAS Patient Registry yet? Are you up-to-date on your surveys?
      Take a trip to www.fdmasregistry.org today to learn more about the project, enroll, complete
      your surveys, or make sure you aren't due to provide more info!

      The FD/MAS Patient Registry: Your story powers research.
The FD/MAS Patient Registry is an IRB approved research project that allows patients and
      families to share their experiences with fibrous dysplasia/McCune-Albright syndrome (FD/MAS)
      by completing a series of surveys.

      The surveys were created in collaboration with patients, parents, clinicians and researchers,
      so that the data can be used to answer some of the most important questions about FD/MAS,
      including:

      the way the disease develops over time (its "natural history"), the patient experience of the
      disease, and its impact on quality of life, how and when diagnoses are made, the scope of
      treatments in use, what surgical techniques work best, and for whom, what other medical
      interventions work best, and for whom, what social services and therapies are useful, the
      costs of care to patients and their families, issues that concern patients (such as the
      impact of pregnancy on FD, or children feeling different and facing stigma), and which
      research questions and support programs you think are important to fund.

      Participation is free and convenient for people with FD/MAS and their legal guardians. You
      can join today at www.fdmasregistry.org.
observational model: Cohort
time perspective: Cross-Sectional
measure: Treatment satisfaction
time frame: Through study completion, an average of every 2 years
description: FACIT-Treatment Satisfaction scale
measure: Perceived symptoms of pain
time frame: Through study completion, an average of every 2 years
description: Brief Pain Inventory
measure: Depression/anxiety
time frame: Through study completion, an average of every 2 years
description: Hospital Anxiety Depression Scale
measure: Stigma
time frame: Through study completion, an average of every 2 years
description: NeuroQol Pediatric and Adult Stigma short forms
measure: Health-related Quality of Life
time frame: Through study completion, an average of every 2 years
description: SF-36,PedsQL 4.0
measure: Financial health
time frame: Through study completion, an average of every 2 years
description: FACIT-Cost
arm group label: FD/MAS Patients
description: Patients with fibrous dysplasia and/or McCune-Albright syndrome and related disorders.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Carmel Shemmesh-Rafalowsky
email: PI.Registry@fibrousdysplasia.org
last name: Tovah Burstein
phone: 603-325-2489
email: TBurstein@fibrousdysplasia.org
facility: 
status: Recruiting
contact: 
country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: Fibrous Dysplasia Foundation
investigator full name: Tovah Burstein
investigator title: Community Engagement Director
mesh term: Fibrous Dysplasia of Bone
mesh term: Fibrous Dysplasia, Polyostotic
mesh term: Syndrome
mesh term: Hyperplasia