<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/NCT03271840</url>
  </required_header>
  <id_info>
    <org_study_id>pro00074669</org_study_id>
    <nct_id>NCT03271840</nct_id>
  </id_info>
  <brief_title>Registry for Primary Ciliary Dyskinesia</brief_title>
  <official_title>Registry for Primary Ciliary Dyskinesia: : Systematic Data Collection on Incidence, Clinical Presentation, Treatment and Course of the Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University of Alberta</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>University of Alberta</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>
      Primary Ciliary Dyskinesia (PCD) is a rare disease, which means that any single PCD center&#xD;
      has experience with a limited number of patients. PCD Registry is the collection of data&#xD;
      about PCD from many centers and countries who treat children with PCD. Collecting data about&#xD;
      PCD increase the knowledge on PCD, better describe the course of the disease, and help to&#xD;
      better understand the progression of the disease and be used to develop new treatments.&#xD;
&#xD;
      In the PCD registry of Alberta, important information about PCD such as time of diagnosis,&#xD;
      symptoms, and tests which led to the diagnosis, state of health at diagnosis, the progression&#xD;
      of lung function, the occurrence of severe infections, tests and treatments data will be&#xD;
      collected from the patients' medical records.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      In order to better characterize the clinical course, and improve the diagnosis and treatment&#xD;
      of PCD, the investigators propose to establish a provincial PCD registry in Alberta. This&#xD;
      registry will be securely linked to the currently existing International Registry for PCD and&#xD;
      will allow more researchers to use the information to do research on PCD.&#xD;
&#xD;
      The investigators will collect demographic data: current age, sex, age, diagnostic findings&#xD;
      and symptoms at diagnosis; in addition, clinical data about PCD which are recorded during the&#xD;
      past and future clinical visits and encounters (e.g. lung function, weight, height,&#xD;
      infections, treatments, and complications) will also be collected.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">August 26, 2017</start_date>
  <completion_date type="Actual">November 25, 2021</completion_date>
  <primary_completion_date type="Actual">November 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>Prospective</time_perspective>
  </study_design_info>
  <target_duration>10 Years</target_duration>
  <primary_outcome>
    <measure>Age of diagnosis</measure>
    <time_frame>2017-2030</time_frame>
    <description>Years</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Lung Function</measure>
    <time_frame>2017-2030</time_frame>
    <description>% predicted for age</description>
  </primary_outcome>
  <enrollment type="Actual">12</enrollment>
  <condition>Primary Ciliary Dyskinesia</condition>
  <eligibility>
    <study_pop>
      <textblock>
        Patients with PCD&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Clinical diagnosis of PCD&#xD;
&#xD;
          -  Abnormal findings in at least two of the following tests:&#xD;
&#xD;
        High-frequency video microscopic finding, transmission electron microscopy finding,&#xD;
        immunofluorescence finding, low nasal NO concentration/production, demonstration of&#xD;
        biallelic disease-causing mutations by genotyping.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        • Failure or unwillingness to give written informed consent.&#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>Israel Amirav, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Alberta</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of Alberta</name>
      <address>
        <city>Edmonton</city>
        <state>Alberta</state>
        <zip>T6G2C6</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
  </location_countries>
  <verification_date>June 2023</verification_date>
  <study_first_submitted>August 26, 2017</study_first_submitted>
  <study_first_submitted_qc>August 31, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 5, 2017</study_first_posted>
  <last_update_submitted>June 5, 2023</last_update_submitted>
  <last_update_submitted_qc>June 5, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 6, 2023</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>Ciliary Motility Disorders</mesh_term>
    <mesh_term>Dyskinesias</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </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/NCT03271840
org study id: pro00074669
nct id: NCT03271840
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
Primary Ciliary Dyskinesia (PCD) is a rare disease, which means that any single PCD center
      has experience with a limited number of patients. PCD Registry is the collection of data
      about PCD from many centers and countries who treat children with PCD. Collecting data about
      PCD increase the knowledge on PCD, better describe the course of the disease, and help to
      better understand the progression of the disease and be used to develop new treatments.

      In the PCD registry of Alberta, important information about PCD such as time of diagnosis,
      symptoms, and tests which led to the diagnosis, state of health at diagnosis, the progression
      of lung function, the occurrence of severe infections, tests and treatments data will be
      collected from the patients' medical records.
In order to better characterize the clinical course, and improve the diagnosis and treatment
      of PCD, the investigators propose to establish a provincial PCD registry in Alberta. This
      registry will be securely linked to the currently existing International Registry for PCD and
      will allow more researchers to use the information to do research on PCD.

      The investigators will collect demographic data: current age, sex, age, diagnostic findings
      and symptoms at diagnosis; in addition, clinical data about PCD which are recorded during the
      past and future clinical visits and encounters (e.g. lung function, weight, height,
      infections, treatments, and complications) will also be collected.
observational model: Cohort
time perspective: Prospective
measure: Age of diagnosis
time frame: 2017-2030
description: Years
measure: Lung Function
time frame: 2017-2030
description: % predicted for age
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Israel Amirav, MD
role: Principal Investigator
affiliation: University of Alberta
facility: 
country: Canada
responsible party type: Sponsor
mesh term: Ciliary Motility Disorders
mesh term: Dyskinesias
sharing ipd: Undecided