<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/NCT04454892</url>
  </required_header>
  <id_info>
    <org_study_id>PUTH2019388</org_study_id>
    <nct_id>NCT04454892</nct_id>
  </id_info>
  <brief_title>Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS)</brief_title>
  <official_title>A Study on the Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS) in Mainland China</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Peking University Third Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Peking University Third 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>
      1. Describe the distribution of ALS in mainland China, to explore the differences in the&#xD;
           number of ALS in different times, regions and populations in order to further explore&#xD;
           the causes affecting the distribution of ALS;&#xD;
&#xD;
        2. To investigate the cause of ALS in mainland China in the crowd disease development&#xD;
           process and the corresponding characteristics change ;&#xD;
&#xD;
        3. To explore the effect of prognosis of ALS;&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      With the development of supportive measures, the natural history of ALS has changed.&#xD;
      Researchers compared the natural history of ALS patients from 1999-2004 and 1984-1998 and&#xD;
      found that the median survival time was significantly longer in the former than in the latter&#xD;
      (4.32 years vs. 3.22 years) and that the disease progression was slower in the former, even&#xD;
      after adjusting for other confounding factors. Although previous studies have provided&#xD;
      reference for the diagnosis and treatment of ALS, the etiology of ALS is still unknown, and&#xD;
      the relevant clinical features and natural history of ALS still lack the verification of&#xD;
      large samples. Therefore, the research on the natural history of ALS is of great significance&#xD;
      to further increase the understanding of ALS and provide new evidence for the diagnosis and&#xD;
      treatment of ALS.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">November 1, 2019</start_date>
  <completion_date type="Anticipated">December 1, 2030</completion_date>
  <primary_completion_date type="Anticipated">December 1, 2030</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>Overall Survival</measure>
    <time_frame>5 years to 10 years after recruitment</time_frame>
    <description>Time interval from disease diagnosis to death</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Indicators of disease progression</measure>
    <time_frame>1 year to 10 years after recruitment</time_frame>
    <description>1) in the diagnosis of disease progression (⊿ FS) = (diagnosis of FRS - R score - when follow-up FRS - R)/diagnosis and follow-up time 2）Weight loss =(weight at diagnosis - weight at follow-up)/time to follow-up 3) BMI decreased = (BMI) at the time of diagnosis of BMI - follow-up/diagnosis and follow-up time 4) FVC decreased = (FVC - during the diagnosis to follow-up FVC)/diagnosis and follow-up time 5) between the above indexes can also be in all follow-up multiple numerical calculation, observing the trend of the change over time</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">3000</enrollment>
  <condition>Amyotrophic Lateral Sclerosis</condition>
  <arm_group>
    <arm_group_label>Amyotrophic lateral sclerosis patients</arm_group_label>
    <description>Although previous studies have provided reference for the diagnosis and treatment of ALS, the etiology of ALS is still unknown, and the relevant clinical features and natural history of ALS still lack the verification of large samples. Therefore, the research on the natural history of ALS is of great significance to further increase the understanding of ALS and provide new evidence for the diagnosis and treatment of ALS</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Amyotrophic lateral sclerosis</intervention_name>
    <description>AMYOTROPHIC lateral sclerosis (ALS) is a degenerative disorder of motor neurons in the cortex, brainstem and spinal cord1,2. Its cause is unknown and it is uniformly fatal, typically within five years3</description>
    <arm_group_label>Amyotrophic lateral sclerosis patients</arm_group_label>
  </intervention>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Serum and plasma&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        ALS patients in mainland China.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        - Patients were diagnosed with ALS recruited from all participant clinical centers.The&#xD;
        diagnosis of ALS was made using the revised El Escorial criteria for definite, probable,&#xD;
        lab-supported, and possible.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Decline to follow-up.&#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>Dongsheng Fan, MD.PHD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Peking University Third Hospital</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Lu Tang, Msc</last_name>
    <phone>13811854649</phone>
    <email>tanglu@bjmu.edu.cn</email>
  </overall_contact>
  <location>
    <facility>
      <name>Peking University Third Hospital</name>
      <address>
        <city>Beijing</city>
        <country>China</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Dongsheng Fan, PHD. MD</last_name>
    </contact>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <verification_date>June 2020</verification_date>
  <study_first_submitted>June 29, 2020</study_first_submitted>
  <study_first_submitted_qc>June 29, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 2, 2020</study_first_posted>
  <last_update_submitted>June 29, 2020</last_update_submitted>
  <last_update_submitted_qc>June 29, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">July 2, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>amyotrophic lateral sclerosis; Epidemiology</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Motor Neuron Disease</mesh_term>
    <mesh_term>Amyotrophic Lateral Sclerosis</mesh_term>
    <mesh_term>Sclerosis</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</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/NCT04454892
org study id: PUTH2019388
nct id: NCT04454892
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
1. Describe the distribution of ALS in mainland China, to explore the differences in the
           number of ALS in different times, regions and populations in order to further explore
           the causes affecting the distribution of ALS;

        2. To investigate the cause of ALS in mainland China in the crowd disease development
           process and the corresponding characteristics change ;

        3. To explore the effect of prognosis of ALS;
With the development of supportive measures, the natural history of ALS has changed.
      Researchers compared the natural history of ALS patients from 1999-2004 and 1984-1998 and
      found that the median survival time was significantly longer in the former than in the latter
      (4.32 years vs. 3.22 years) and that the disease progression was slower in the former, even
      after adjusting for other confounding factors. Although previous studies have provided
      reference for the diagnosis and treatment of ALS, the etiology of ALS is still unknown, and
      the relevant clinical features and natural history of ALS still lack the verification of
      large samples. Therefore, the research on the natural history of ALS is of great significance
      to further increase the understanding of ALS and provide new evidence for the diagnosis and
      treatment of ALS.
observational model: Cohort
time perspective: Prospective
measure: Overall Survival
time frame: 5 years to 10 years after recruitment
description: Time interval from disease diagnosis to death
measure: Indicators of disease progression
time frame: 1 year to 10 years after recruitment
description: 1) in the diagnosis of disease progression (⊿ FS) = (diagnosis of FRS - R score - when follow-up FRS - R)/diagnosis and follow-up time 2）Weight loss =(weight at diagnosis - weight at follow-up)/time to follow-up 3) BMI decreased = (BMI) at the time of diagnosis of BMI - follow-up/diagnosis and follow-up time 4) FVC decreased = (FVC - during the diagnosis to follow-up FVC)/diagnosis and follow-up time 5) between the above indexes can also be in all follow-up multiple numerical calculation, observing the trend of the change over time
arm group label: Amyotrophic lateral sclerosis patients
description: Although previous studies have provided reference for the diagnosis and treatment of ALS, the etiology of ALS is still unknown, and the relevant clinical features and natural history of ALS still lack the verification of large samples. Therefore, the research on the natural history of ALS is of great significance to further increase the understanding of ALS and provide new evidence for the diagnosis and treatment of ALS
intervention type: Other
intervention name: Amyotrophic lateral sclerosis
description: AMYOTROPHIC lateral sclerosis (ALS) is a degenerative disorder of motor neurons in the cortex, brainstem and spinal cord1,2. Its cause is unknown and it is uniformly fatal, typically within five years3
arm group label: Amyotrophic lateral sclerosis patients
Serum and plasma
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Dongsheng Fan, MD.PHD
role: Principal Investigator
affiliation: Peking University Third Hospital
last name: Lu Tang, Msc
phone: 13811854649
email: tanglu@bjmu.edu.cn
facility: 
status: Recruiting
contact: 
country: China
responsible party type: Sponsor
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
mesh term: Sclerosis
sharing ipd: No