<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/NCT04454840</url>
  </required_header>
  <id_info>
    <org_study_id>PUTH2017118</org_study_id>
    <nct_id>NCT04454840</nct_id>
  </id_info>
  <brief_title>Treatment of Intravenous Infusion Plasma in Amyotrophic Lateral Sclerosis</brief_title>
  <official_title>A Clinical Study on Safety and Effectiveness of Intravenous Infusion Plasma From Healthy Young People to Treat Amyotrophic Lateral Sclerosis</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>
      To evaluate the safety and effectiveness of intravenous infusion of plasma from healthy young&#xD;
      people for the treatment of amyotrophic lateral sclerosis.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a single-center, open-label clinical study to evaluate the safety and effectiveness&#xD;
      of intravenous infusion of plasma from healthy young people for the treatment of amyotrophic&#xD;
      lateral sclerosis. The main outcome indicators are the record of adverse reactions and the&#xD;
      rate of change of amyotrophic lateral sclerosisFunctional Rating Scale score. The secondary&#xD;
      outcome indicators include survival time-time to the end event (death, tracheotomy,&#xD;
      continuous ventilator dependence), forced vital capacity (FVC), recognition Knowledge&#xD;
      function evaluation (ECAS score).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">May 1, 2016</start_date>
  <completion_date type="Actual">May 1, 2019</completion_date>
  <primary_completion_date type="Actual">May 1, 2019</primary_completion_date>
  <phase>Early Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
    <masking_description>Open Lable</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Amyotrophic lateral sclerosis Functional Rating Scale scores</measure>
    <time_frame>22 months after intervention</time_frame>
    <description>Rate of amyotrophic lateral sclerosis Functional Rating Scale scores changes</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Survival time</measure>
    <time_frame>22 months after intervention</time_frame>
    <description>The time of the end event (death, tracheotomy, continuous ventilator dependence);</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Forced vital capacity (FVC)</measure>
    <time_frame>22 months after intervention</time_frame>
    <description>The change from baseline to the end of follow-up;</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Cognitive function evaluation (ECAS score)</measure>
    <time_frame>22 months after intervention</time_frame>
    <description>The change from baseline to the end of follow-up;</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">20</enrollment>
  <condition>Amyotrophic Lateral Sclerosis</condition>
  <arm_group>
    <arm_group_label>Biological+Riluzole</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Plasma from healthy young people treatment + Riluzole</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Riluzole</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Riluzole</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Plasma from healthy young people treatment + Riluzole</intervention_name>
    <description>Intravenous injection: the subjects received 400ml of intravenous plasma infusion every 2 weeks, with a continuous course of 2 treatments and a course of treatment every 3 months. The course of treatment lasted 10 months, with a total of 3200ml of plasma infusion.</description>
    <arm_group_label>Biological+Riluzole</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Riluzole</intervention_name>
    <description>The basic treatment is Riluzole 25~50mg twice daily</description>
    <arm_group_label>Riluzole</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Meet the following diagnosis standard: confirmed, proposed and laboratory supported&#xD;
             diagnosis;&#xD;
&#xD;
          -  Age 50-70 years old ;&#xD;
&#xD;
          -  3-18 months course of disease;&#xD;
&#xD;
          -  Forced vital capacity (FVC) ≥70% predicted value;&#xD;
&#xD;
          -  Total amyotrophic lateral sclerosis Functional Rating Scale score ≥36, scores of&#xD;
             respiratory related items ≥10;&#xD;
&#xD;
          -  Take Riluzole regularly before participate in this trial (25~50mg twice a day for at&#xD;
             least 30 days continuously) without obvious side effects and can continue to take for&#xD;
             22 months;&#xD;
&#xD;
          -  Participants of childbearing age take reasonable and effective contraceptive measures&#xD;
             from the time of enrollment to the end of follow-up;&#xD;
&#xD;
          -  Signed informed consent.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Familial amyotrophic lateral sclerosis;&#xD;
&#xD;
          -  Female during pregnancy and lactation;&#xD;
&#xD;
          -  Positive hepatitis B, hepatitis C or HIV in screening&#xD;
&#xD;
          -  History of cytomegalovirus and malaria infection;&#xD;
&#xD;
          -  After tracheotomy and ventilator-dependent state (daily use of non-invasive ventilator&#xD;
             ≥ 22 hours for 7 consecutive days);&#xD;
&#xD;
          -  After percutaneous gastrostomy (PEG) operation;&#xD;
&#xD;
          -  Has had allergic reactions and other adverse reactions during blood transfusion;&#xD;
&#xD;
          -  Have diseases of the blood system (including Immunoglobulin A deficiency);&#xD;
&#xD;
          -  alanine transaminase, Aspartate transaminase≥ 3 times the upper limit of normal;&#xD;
&#xD;
          -  Abnormal renal function (Cr, BUN);&#xD;
&#xD;
          -  History of malignant tumors;&#xD;
&#xD;
          -  Combining severe cardiopulmonary diseases, autoimmune diseases, mental diseases,&#xD;
             substance abuse history, etc;&#xD;
&#xD;
          -  Currently participating in other clinical studies or using other drugs in researching.&#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>
  <location>
    <facility>
      <name>Peking University Third Hospital</name>
      <address>
        <city>Beijing</city>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <verification_date>July 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>July 1, 2020</last_update_submitted>
  <last_update_submitted_qc>July 1, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">July 7, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Amyotrophic lateral sclerosis; Plasma infusion</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>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Riluzole</mesh_term>
  </intervention_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/NCT04454840
org study id: PUTH2017118
nct id: NCT04454840
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
To evaluate the safety and effectiveness of intravenous infusion of plasma from healthy young
      people for the treatment of amyotrophic lateral sclerosis.
This is a single-center, open-label clinical study to evaluate the safety and effectiveness
      of intravenous infusion of plasma from healthy young people for the treatment of amyotrophic
      lateral sclerosis. The main outcome indicators are the record of adverse reactions and the
      rate of change of amyotrophic lateral sclerosisFunctional Rating Scale score. The secondary
      outcome indicators include survival time-time to the end event (death, tracheotomy,
      continuous ventilator dependence), forced vital capacity (FVC), recognition Knowledge
      function evaluation (ECAS score).
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
masking description: Open Lable
measure: Amyotrophic lateral sclerosis Functional Rating Scale scores
time frame: 22 months after intervention
description: Rate of amyotrophic lateral sclerosis Functional Rating Scale scores changes
measure: Survival time
time frame: 22 months after intervention
description: The time of the end event (death, tracheotomy, continuous ventilator dependence);
measure: Forced vital capacity (FVC)
time frame: 22 months after intervention
description: The change from baseline to the end of follow-up;
measure: Cognitive function evaluation (ECAS score)
time frame: 22 months after intervention
description: The change from baseline to the end of follow-up;
arm group label: Biological+Riluzole
arm group type: Experimental
description: Plasma from healthy young people treatment + Riluzole
arm group label: Riluzole
arm group type: Active Comparator
description: Riluzole
intervention type: Biological
intervention name: Plasma from healthy young people treatment + Riluzole
description: Intravenous injection: the subjects received 400ml of intravenous plasma infusion every 2 weeks, with a continuous course of 2 treatments and a course of treatment every 3 months. The course of treatment lasted 10 months, with a total of 3200ml of plasma infusion.
arm group label: Biological+Riluzole
intervention type: Drug
intervention name: Riluzole
description: The basic treatment is Riluzole 25~50mg twice daily
arm group label: Riluzole
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
facility: 
country: China
responsible party type: Sponsor
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
mesh term: Sclerosis
mesh term: Riluzole
sharing ipd: No