<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/NCT04472130</url>
  </required_header>
  <id_info>
    <org_study_id>CRE-2019.371</org_study_id>
    <nct_id>NCT04472130</nct_id>
  </id_info>
  <brief_title>Neurodegenerative Diseases Registry</brief_title>
  <acronym>NDD Registry</acronym>
  <official_title>Biomarkers in Neurodegenerative Diseases</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Vincent Mok</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Chinese University of Hong Kong</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      With the increase in life expectancy of our population due to advancement of medical&#xD;
      diagnosis and treatments, the incidence of age dependent neurodegenerative diseases&#xD;
      increased, including Alzheimer's disease (AD), parkinsonian syndromes (PS), small vessel&#xD;
      disease (SVD) and motor neuron disease (MND). In spite of the progress of knowing the&#xD;
      pathogenesis of various neurodegenerative diseases at molecular and genetic level, they are&#xD;
      still very incompletely understood and often cause diagnostic and therapeutic challenges to&#xD;
      physicians. Due to the overlapping presentation and similar brain pathology, especially in&#xD;
      the early stage of the diseases, it is difficult to differentiate idiopathic Parkinson's&#xD;
      disease (iPD) from atypical parkinsonian syndromes, such as multiple system atrophy (MSA) and&#xD;
      progressive supranuclear palsy (PSP). Similarly, distinguishing AD from other dementia&#xD;
      syndromes including frontotemporal dementia (FTD), dementia with Lewy Bodies (DLB),&#xD;
      corticobasal degeneration (CBD) and vascular dementia can be difficult. It is necessary to&#xD;
      develop accurate and comprehensive diagnostic tests to properly prognosticate the diseases,&#xD;
      start treatments in early stage of the diseases and maximize the accuracy of drug trials for&#xD;
      more effective preventive and therapeutic measures for these neurodegenerative diseases.&#xD;
&#xD;
      Therefore, the registry aims to generate a large database of cognitive, behavioral, lifestyle&#xD;
      and psychological information of the subjects who suffered from neurodegenerative diseases,&#xD;
      as well as to examine the genetic basis of neurodegenerative diseases to help decode the&#xD;
      pathogenic mechanisms of the diseases. The registry may provide important information to&#xD;
      understand symptom development of the neurodegenerative diseases, in which may help&#xD;
      physicians to diagnose the diseases more accurately and provide better treatment plans.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a cohort study. It involves baseline, 1st follow up visit and 2nd follow up visit. At&#xD;
      baseline visit, all participants will go through a list of assessments and questionnaires and&#xD;
      blood taking. Follow-up visit(s) will be scheduled every one to two years, in which the same&#xD;
      set of assessments and questionnaires will be administered.&#xD;
&#xD;
        1. Clinical assessments and questionnaires&#xD;
&#xD;
           Different clinical assessments would be administered depending on the group that the&#xD;
           participant belongs to:&#xD;
&#xD;
             -  Hoehn and Yahr Stage and the Unified Parkinson's Disease Rating Scale (UPDRS) for&#xD;
                iPD, PSP and SVD patients with parkinsonism features&#xD;
&#xD;
             -  Unified MSA Rating Scale (UMSARS) for MSA&#xD;
&#xD;
             -  Levodopa Equivalent Dosage for medication burden for parkinsonian syndromes&#xD;
                patients&#xD;
&#xD;
             -  Amyotrophic lateral sclerosis functional rating scale revised (ALSFRS-R), body&#xD;
                weight and forced vital capacity (FVC) for MND group&#xD;
&#xD;
             -  Montreal Cognitive Assessment Hong Kong version (HK-MoCA), Olfactory Identification&#xD;
                Test (OIT) and Farnsworth-Munsell 100 Hue test for all groups&#xD;
&#xD;
           Video taking would be administered to record participants' eye movement if necessary.&#xD;
           For example, video of eye movement is useful to rate MSA patients' ocular motor&#xD;
           dysfunction, such as gaze-evoke nystagmus.&#xD;
&#xD;
           Patients with parkinsonian syndromes will fill in a set of questionnaires, including&#xD;
           demographic information, medical history, history and current medications, wearing-off&#xD;
           questionnaire, impulsiveness questionnaire, Buss-Perry Aggression Questionnaire (BPAQ),&#xD;
           rapid eye movement sleep behavior disorder questionnaire (RBDQ), Epworth Sleepiness&#xD;
           Scale (ESS), Morningness-Eveningness Questionnaire (MEQ), Insomnia Severity Index (ISI),&#xD;
           Beck's Scale for Suicide Ideation (BSSI), Scales for Outcomes in Parkinson's&#xD;
           Disease-Autonomic questionnaire (SCOPA-AUT), Hospital Anxiety and Depression Scale&#xD;
           (HADS), Patient Health Questionnaire (PHQ9), lifestyle and life history, and occupation&#xD;
           history.&#xD;
&#xD;
        2. Blood sampling Blood taking would be carried out at Prince of Wales Hospital and will be&#xD;
           processed and transported to the laboratory according to standard procedure.&#xD;
&#xD;
           Venous blood samples are collected into 6 EDTA tubes and 1 Heparin tube. The volume of&#xD;
           total blood samples will not exceed 23ml. Serum is obtained within 1 hour by&#xD;
           centrifugation at 3,000 rpm for 10 min and stored at -70°C until laboratory evaluation&#xD;
           for proteomics, SERS and other biochemical and genetics studies.&#xD;
&#xD;
        3. Sub-studies Selected participants in the cohort groups, especially those with early&#xD;
           disease onset and/or familial cases, would proceed to sub-studies which include brain&#xD;
           MRI, brain PET, lumbar puncture and/or skin biopsy. Subjects are voluntary to join one&#xD;
           or more sub-studies.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">October 9, 2019</start_date>
  <completion_date type="Anticipated">August 2024</completion_date>
  <primary_completion_date type="Anticipated">August 2024</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>4 Years</target_duration>
  <primary_outcome>
    <measure>the score change in Unified Parkinson's Disease Rating Scale (UPDRS)</measure>
    <time_frame>baseline visit, 2 years after baseline visit, 4 years after baseline visit</time_frame>
    <description>The Unified Parkinson's Disease Rating Scale (UPDRS) would be administered to assess disease severity for iPD, PSP and SVD patients with parkinsonism features</description>
  </primary_outcome>
  <primary_outcome>
    <measure>the score change in Unified MSA Rating Scale (UMSARS)</measure>
    <time_frame>baseline visit, 2 years after baseline visit, 4 years after baseline visit</time_frame>
    <description>The Unified MSA Rating Scale (UMSARS) would be administered to assess disease severity for patients with MSA</description>
  </primary_outcome>
  <primary_outcome>
    <measure>the score change in Montreal Cognitive Assessment Hong Kong version (HK-MoCA)</measure>
    <time_frame>baseline visit, 2 years after baseline visit, 4 years after baseline visit</time_frame>
    <description>Montreal Cognitive Assessment Hong Kong version (HK-MoCA) is used to assess participants' cognitive functions for all groups</description>
  </primary_outcome>
  <number_of_groups>9</number_of_groups>
  <enrollment type="Anticipated">1400</enrollment>
  <condition>Neurodegenerative Diseases</condition>
  <condition>Parkinson Disease</condition>
  <condition>Multiple System Atrophy</condition>
  <condition>Progressive Supranuclear Palsy</condition>
  <arm_group>
    <arm_group_label>Early idiopathic Parkinson's Disease</arm_group_label>
    <description>200 patients with iPD based on Movement Disorder Society clinical diagnostic criteria for Parkinson's disease with disease onset less than 5 years</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Non-early idiopathic Parkinson's Disease</arm_group_label>
    <description>200 patients with iPD based on Movement Disorder Society clinical diagnostic criteria for Parkinson's disease with disease onset more than 5 years</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Multiple System Atrophy</arm_group_label>
    <description>100 patients with Multiple System Atrophy based on Second consensus statement on the diagnosis of MSA</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Progressive Supranuclear Palsy</arm_group_label>
    <description>100 patients with Progressive Supranuclear Palsy based on Clinical research criteria for diagnosis of PSP</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Alzheimer's Disease</arm_group_label>
    <description>100 patients with Alzheimer's Disease by Diagnostic and Statistical Manual of Mental disorder, Fifth edition (DSM-5) criteria</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Motor Neuron Disease</arm_group_label>
    <description>200 patients with Motor Neuron Diseases by revised El Escorial criteria or Awaji ALS criteria</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Small Vessel Disease</arm_group_label>
    <description>200 patients with cerebral Small Vessel Diseases</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Frontotemporal Dementia</arm_group_label>
    <description>100 patients with Frontotemporal Dementia by International consensus criteria for behavioral variant FTD (FTDC) or Primary Progressive Aphasia by Gorno-Tempini</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Healthy Control</arm_group_label>
    <description>200 age and sex matched healthy controls</description>
  </arm_group>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Plasma, blood cells, cerebrospinal fluid&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Subjects will be recruited in neurology outpatient clinic in Prince of Wales Hospital, Hong&#xD;
        Kong, who meet the inclusion and exclusion criteria listed above.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Age should be between 18-80 years old.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patients with ongoing central nervous system infection and/or acute stroke or active&#xD;
             brain tumor.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>80 Years</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Vincent Mok, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Chinese University of Hong Kong</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Elyia Han, Bachelor</last_name>
    <phone>+852 2697 5027</phone>
    <email>elyiahan@cuhk.edu.hk</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Pauline Kwan, Master</last_name>
    <phone>+852 2635 2160</phone>
    <email>paulinekwan@cuhk.edu.hk</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Chinese University of Hong Kong</name>
      <address>
        <city>Hong Kong</city>
        <country>Hong Kong</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Elyia Han, Bachelor</last_name>
      <phone>+852 26975027</phone>
      <email>elyiahan@cuhk.edu.hk</email>
    </contact>
  </location>
  <location_countries>
    <country>Hong Kong</country>
  </location_countries>
  <verification_date>July 2021</verification_date>
  <study_first_submitted>June 25, 2020</study_first_submitted>
  <study_first_submitted_qc>July 14, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 15, 2020</study_first_posted>
  <last_update_submitted>July 26, 2021</last_update_submitted>
  <last_update_submitted_qc>July 26, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">July 28, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Chinese University of Hong Kong</investigator_affiliation>
    <investigator_full_name>Vincent Mok</investigator_full_name>
    <investigator_title>Professor</investigator_title>
  </responsible_party>
  <keyword>Neurodegenerative Diseases</keyword>
  <keyword>Parkinson Disease</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Parkinson Disease</mesh_term>
    <mesh_term>Neurodegenerative Diseases</mesh_term>
    <mesh_term>Multiple System Atrophy</mesh_term>
    <mesh_term>Shy-Drager Syndrome</mesh_term>
    <mesh_term>Supranuclear Palsy, Progressive</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/NCT04472130
org study id: CRE-2019.371
nct id: NCT04472130
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
With the increase in life expectancy of our population due to advancement of medical
      diagnosis and treatments, the incidence of age dependent neurodegenerative diseases
      increased, including Alzheimer's disease (AD), parkinsonian syndromes (PS), small vessel
      disease (SVD) and motor neuron disease (MND). In spite of the progress of knowing the
      pathogenesis of various neurodegenerative diseases at molecular and genetic level, they are
      still very incompletely understood and often cause diagnostic and therapeutic challenges to
      physicians. Due to the overlapping presentation and similar brain pathology, especially in
      the early stage of the diseases, it is difficult to differentiate idiopathic Parkinson's
      disease (iPD) from atypical parkinsonian syndromes, such as multiple system atrophy (MSA) and
      progressive supranuclear palsy (PSP). Similarly, distinguishing AD from other dementia
      syndromes including frontotemporal dementia (FTD), dementia with Lewy Bodies (DLB),
      corticobasal degeneration (CBD) and vascular dementia can be difficult. It is necessary to
      develop accurate and comprehensive diagnostic tests to properly prognosticate the diseases,
      start treatments in early stage of the diseases and maximize the accuracy of drug trials for
      more effective preventive and therapeutic measures for these neurodegenerative diseases.

      Therefore, the registry aims to generate a large database of cognitive, behavioral, lifestyle
      and psychological information of the subjects who suffered from neurodegenerative diseases,
      as well as to examine the genetic basis of neurodegenerative diseases to help decode the
      pathogenic mechanisms of the diseases. The registry may provide important information to
      understand symptom development of the neurodegenerative diseases, in which may help
      physicians to diagnose the diseases more accurately and provide better treatment plans.
This is a cohort study. It involves baseline, 1st follow up visit and 2nd follow up visit. At
      baseline visit, all participants will go through a list of assessments and questionnaires and
      blood taking. Follow-up visit(s) will be scheduled every one to two years, in which the same
      set of assessments and questionnaires will be administered.

        1. Clinical assessments and questionnaires

           Different clinical assessments would be administered depending on the group that the
           participant belongs to:

             -  Hoehn and Yahr Stage and the Unified Parkinson's Disease Rating Scale (UPDRS) for
                iPD, PSP and SVD patients with parkinsonism features

             -  Unified MSA Rating Scale (UMSARS) for MSA

             -  Levodopa Equivalent Dosage for medication burden for parkinsonian syndromes
                patients

             -  Amyotrophic lateral sclerosis functional rating scale revised (ALSFRS-R), body
                weight and forced vital capacity (FVC) for MND group

             -  Montreal Cognitive Assessment Hong Kong version (HK-MoCA), Olfactory Identification
                Test (OIT) and Farnsworth-Munsell 100 Hue test for all groups

           Video taking would be administered to record participants' eye movement if necessary.
           For example, video of eye movement is useful to rate MSA patients' ocular motor
           dysfunction, such as gaze-evoke nystagmus.

           Patients with parkinsonian syndromes will fill in a set of questionnaires, including
           demographic information, medical history, history and current medications, wearing-off
           questionnaire, impulsiveness questionnaire, Buss-Perry Aggression Questionnaire (BPAQ),
           rapid eye movement sleep behavior disorder questionnaire (RBDQ), Epworth Sleepiness
           Scale (ESS), Morningness-Eveningness Questionnaire (MEQ), Insomnia Severity Index (ISI),
           Beck's Scale for Suicide Ideation (BSSI), Scales for Outcomes in Parkinson's
           Disease-Autonomic questionnaire (SCOPA-AUT), Hospital Anxiety and Depression Scale
           (HADS), Patient Health Questionnaire (PHQ9), lifestyle and life history, and occupation
           history.

        2. Blood sampling Blood taking would be carried out at Prince of Wales Hospital and will be
           processed and transported to the laboratory according to standard procedure.

           Venous blood samples are collected into 6 EDTA tubes and 1 Heparin tube. The volume of
           total blood samples will not exceed 23ml. Serum is obtained within 1 hour by
           centrifugation at 3,000 rpm for 10 min and stored at -70°C until laboratory evaluation
           for proteomics, SERS and other biochemical and genetics studies.

        3. Sub-studies Selected participants in the cohort groups, especially those with early
           disease onset and/or familial cases, would proceed to sub-studies which include brain
           MRI, brain PET, lumbar puncture and/or skin biopsy. Subjects are voluntary to join one
           or more sub-studies.
observational model: Cohort
time perspective: Prospective
measure: the score change in Unified Parkinson's Disease Rating Scale (UPDRS)
time frame: baseline visit, 2 years after baseline visit, 4 years after baseline visit
description: The Unified Parkinson's Disease Rating Scale (UPDRS) would be administered to assess disease severity for iPD, PSP and SVD patients with parkinsonism features
measure: the score change in Unified MSA Rating Scale (UMSARS)
time frame: baseline visit, 2 years after baseline visit, 4 years after baseline visit
description: The Unified MSA Rating Scale (UMSARS) would be administered to assess disease severity for patients with MSA
measure: the score change in Montreal Cognitive Assessment Hong Kong version (HK-MoCA)
time frame: baseline visit, 2 years after baseline visit, 4 years after baseline visit
description: Montreal Cognitive Assessment Hong Kong version (HK-MoCA) is used to assess participants' cognitive functions for all groups
arm group label: Early idiopathic Parkinson's Disease
description: 200 patients with iPD based on Movement Disorder Society clinical diagnostic criteria for Parkinson's disease with disease onset less than 5 years
arm group label: Non-early idiopathic Parkinson's Disease
description: 200 patients with iPD based on Movement Disorder Society clinical diagnostic criteria for Parkinson's disease with disease onset more than 5 years
arm group label: Multiple System Atrophy
description: 100 patients with Multiple System Atrophy based on Second consensus statement on the diagnosis of MSA
arm group label: Progressive Supranuclear Palsy
description: 100 patients with Progressive Supranuclear Palsy based on Clinical research criteria for diagnosis of PSP
arm group label: Alzheimer's Disease
description: 100 patients with Alzheimer's Disease by Diagnostic and Statistical Manual of Mental disorder, Fifth edition (DSM-5) criteria
arm group label: Motor Neuron Disease
description: 200 patients with Motor Neuron Diseases by revised El Escorial criteria or Awaji ALS criteria
arm group label: Small Vessel Disease
description: 200 patients with cerebral Small Vessel Diseases
arm group label: Frontotemporal Dementia
description: 100 patients with Frontotemporal Dementia by International consensus criteria for behavioral variant FTD (FTDC) or Primary Progressive Aphasia by Gorno-Tempini
arm group label: Healthy Control
description: 200 age and sex matched healthy controls
Plasma, blood cells, cerebrospinal fluid
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: 80 Years
healthy volunteers: Accepts Healthy Volunteers
last name: Vincent Mok, PhD
role: Principal Investigator
affiliation: Chinese University of Hong Kong
last name: Elyia Han, Bachelor
phone: +852 2697 5027
email: elyiahan@cuhk.edu.hk
last name: Pauline Kwan, Master
phone: +852 2635 2160
email: paulinekwan@cuhk.edu.hk
facility: 
status: Recruiting
contact: 
country: Hong Kong
responsible party type: Sponsor-Investigator
investigator affiliation: Chinese University of Hong Kong
investigator full name: Vincent Mok
investigator title: Professor
mesh term: Parkinson Disease
mesh term: Neurodegenerative Diseases
mesh term: Multiple System Atrophy
mesh term: Shy-Drager Syndrome
mesh term: Supranuclear Palsy, Progressive