<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/NCT02193893</url>
  </required_header>
  <id_info>
    <org_study_id>ZPO 02</org_study_id>
    <secondary_id>ZPO 02, ALS-BMSC #01</secondary_id>
    <nct_id>NCT02193893</nct_id>
  </id_info>
  <brief_title>Biological Treatment of Amyotrophic Lateral Sclerosis</brief_title>
  <acronym>NeuStem-ALS</acronym>
  <official_title>Safety/Efficacy Study for the Biological Treatment of Amyotrophic Lateral Sclerosis With Autologous Stem/Progenitor Cells</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Pomeranian Medical University Szczecin</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Pomeranian Medical University Szczecin</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to test the safety and effectiveness of an autologous bone&#xD;
      marrow-derived stem/progenitor cells infusion in the subjects with diagnosed amyotrophic&#xD;
      lateral sclerosis.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Amyotrophic lateral sclerosis (ALS) is a universally fatal neurodegenerative condition that&#xD;
      causes weakness leading to paralysis and death. Life expectancy is less that 5 years. The&#xD;
      cause is generally unknown and there is no effective treatment. Patients with ALS typically&#xD;
      exhibit a progressive paralysis due to the continued loss of motoneurons. Intraspinal&#xD;
      injections of bone marrow mononuclear cells have been able to ameliorate the course of ALS in&#xD;
      murine models. The purpose of this prospective, nonrandomized, open label, pilot study is to&#xD;
      conduct the investigation of the safety and efficacy of infusion of autologous bone&#xD;
      marrow-isolated stem/progenitor cells with different selected phenotypes into the subjects&#xD;
      with diagnosed ALS. Especially, this clinical trial is designated to test the therapeutic&#xD;
      (pro-regenerative and neuro-protective) functions of different stem/progenitor cell&#xD;
      populations able to secrete bioactive neurotrophic factors. All patients enrolled will have a&#xD;
      documented history of ALS disease prior to study enrollment. Patients diagnosed as early&#xD;
      stage of ALS with the duration of disease less than 6 months and patients diagnosed with&#xD;
      advanced stage of ALS disease with duration of 6-12 months will be recruited and allocated&#xD;
      based on their disease severity to two treatment groups: Group I - patients of early ALS&#xD;
      disease stage and Group II - patients of advanced ALS disease. Next, autologous bone&#xD;
      marrow-isolated stem/progenitor cells administration to the cerebrospinal fluid at the site&#xD;
      of the spinal cord will be performed. Finally, treatment safety, adverse events and&#xD;
      exploratory parameters, including electromyographic (EMG) studies, forced vital capacity&#xD;
      (FVC), functional rating scale (FRS) and maximum voluntary isometric contraction-arm&#xD;
      (MVIC-arm) evaluation, to establish ALS progression rate will be recorded throughout the&#xD;
      duration of the post-treatment follow up period.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Enrolling by invitation</last_known_status>
  <start_date>January 2010</start_date>
  <completion_date type="Anticipated">December 2017</completion_date>
  <primary_completion_date type="Anticipated">July 2017</primary_completion_date>
  <phase>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>
  </study_design_info>
  <primary_outcome>
    <measure>Safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients</measure>
    <time_frame>1 year</time_frame>
    <description>Confirm the safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients by repeated follow-up over one year with clinical and laboratory evaluations.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients.</measure>
    <time_frame>1 year</time_frame>
    <description>Confirm the efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients by repeated follow-up over one year with clinical and laboratory evaluation tests for neurodegeneration processes analysis and general health outcomes.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">50</enrollment>
  <condition>Amyotrophic Lateral Sclerosis</condition>
  <arm_group>
    <arm_group_label>Stem/progenitor cells transplantation.</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Intervention: Biological: Cell-based therapeutics Autologous bone marrow-derived stem/progenitor cells will be transplanted intrathecally (via a standard lumbar puncture) into early vs. progressive ALS subjects.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Standard treatment of ALS</arm_group_label>
    <arm_group_type>Sham Comparator</arm_group_type>
    <description>Symptomatic treatment of ALS without biologic cell-based treatment</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Biological: Cell-based therapeutics</intervention_name>
    <description>Human autologous bone marrow-derived stem/progenitor cell transplantation in ALS patients.</description>
    <arm_group_label>Stem/progenitor cells transplantation.</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Symptomatic treatment of ALS</intervention_name>
    <description>Symptomatic neurological treatment of ALS</description>
    <arm_group_label>Standard treatment of ALS</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  diagnosis of the ALS disease before the cell transplantation (diagnose established&#xD;
             following the El Escorial criteria for definite ALS)&#xD;
&#xD;
          -  good understanding of the protocol and willingness to consent&#xD;
&#xD;
          -  patient is mentally intact and psychologically stable&#xD;
&#xD;
          -  signed informed consent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        Concomitant of other systemic disease or diseases:&#xD;
&#xD;
          -  inflammation (high protein or lymphocytosis in the CSF), active infections.&#xD;
&#xD;
          -  diabetes,&#xD;
&#xD;
          -  cardio-vascular disorders,&#xD;
&#xD;
          -  cancer,&#xD;
&#xD;
          -  autoimmune diseases&#xD;
&#xD;
          -  renal failure,&#xD;
&#xD;
          -  impaired hepatic function.&#xD;
&#xD;
          -  subject is a respiratory dependent.&#xD;
&#xD;
          -  subject unwilling or unable to comply with the requirements of the protocol.&#xD;
&#xD;
          -  patient has been treated previously with any cellular therapy.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>65 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Boguslaw Machalinski, MD, PhD</last_name>
    <role>Study Director</role>
    <affiliation>Pomeranian Medical University Szczecin</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Przemyslaw Nowacki, MD, PhD</last_name>
    <role>Study Chair</role>
    <affiliation>Pomeranian Medical University Szczecin</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Department of Neurology of Pomeranian Medical University in Szczecin</name>
      <address>
        <city>Szczecin</city>
        <zip>71-252</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Poland</country>
  </location_countries>
  <verification_date>July 2014</verification_date>
  <study_first_submitted>July 16, 2014</study_first_submitted>
  <study_first_submitted_qc>July 17, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">July 18, 2014</study_first_posted>
  <last_update_submitted>July 17, 2014</last_update_submitted>
  <last_update_submitted_qc>July 17, 2014</last_update_submitted_qc>
  <last_update_posted type="Estimate">July 18, 2014</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Pomeranian Medical University Szczecin</investigator_affiliation>
    <investigator_full_name>Milosz Kawa</investigator_full_name>
    <investigator_title>Principal Investigator: Boguslaw Machalinski, Professor and Chief, Department of General Pathology, Pomeranian Medical University, Pomeranian Medical University Szczecin</investigator_title>
  </responsible_party>
  <keyword>cell-based therapy</keyword>
  <keyword>autologous bone marrow-derived stem/progenitor cells</keyword>
  <keyword>stem cell infusion, stem cell transplantation</keyword>
  <keyword>biological therapy with neurotrophic factors</keyword>
  <keyword>bioactive neurotrophic factors</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>Coal Tar</mesh_term>
  </intervention_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/NCT02193893
org study id: ZPO 02
secondary id: ZPO 02, ALS-BMSC #01
nct id: NCT02193893
lead sponsor: 
has dmc: No
The purpose of this study is to test the safety and effectiveness of an autologous bone
      marrow-derived stem/progenitor cells infusion in the subjects with diagnosed amyotrophic
      lateral sclerosis.
Amyotrophic lateral sclerosis (ALS) is a universally fatal neurodegenerative condition that
      causes weakness leading to paralysis and death. Life expectancy is less that 5 years. The
      cause is generally unknown and there is no effective treatment. Patients with ALS typically
      exhibit a progressive paralysis due to the continued loss of motoneurons. Intraspinal
      injections of bone marrow mononuclear cells have been able to ameliorate the course of ALS in
      murine models. The purpose of this prospective, nonrandomized, open label, pilot study is to
      conduct the investigation of the safety and efficacy of infusion of autologous bone
      marrow-isolated stem/progenitor cells with different selected phenotypes into the subjects
      with diagnosed ALS. Especially, this clinical trial is designated to test the therapeutic
      (pro-regenerative and neuro-protective) functions of different stem/progenitor cell
      populations able to secrete bioactive neurotrophic factors. All patients enrolled will have a
      documented history of ALS disease prior to study enrollment. Patients diagnosed as early
      stage of ALS with the duration of disease less than 6 months and patients diagnosed with
      advanced stage of ALS disease with duration of 6-12 months will be recruited and allocated
      based on their disease severity to two treatment groups: Group I - patients of early ALS
      disease stage and Group II - patients of advanced ALS disease. Next, autologous bone
      marrow-isolated stem/progenitor cells administration to the cerebrospinal fluid at the site
      of the spinal cord will be performed. Finally, treatment safety, adverse events and
      exploratory parameters, including electromyographic (EMG) studies, forced vital capacity
      (FVC), functional rating scale (FRS) and maximum voluntary isometric contraction-arm
      (MVIC-arm) evaluation, to establish ALS progression rate will be recorded throughout the
      duration of the post-treatment follow up period.
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients
time frame: 1 year
description: Confirm the safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients by repeated follow-up over one year with clinical and laboratory evaluations.
measure: Efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients.
time frame: 1 year
description: Confirm the efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients by repeated follow-up over one year with clinical and laboratory evaluation tests for neurodegeneration processes analysis and general health outcomes.
arm group label: Stem/progenitor cells transplantation.
arm group type: Active Comparator
description: Intervention: Biological: Cell-based therapeutics Autologous bone marrow-derived stem/progenitor cells will be transplanted intrathecally (via a standard lumbar puncture) into early vs. progressive ALS subjects.
arm group label: Standard treatment of ALS
arm group type: Sham Comparator
description: Symptomatic treatment of ALS without biologic cell-based treatment
intervention type: Other
intervention name: Biological: Cell-based therapeutics
description: Human autologous bone marrow-derived stem/progenitor cell transplantation in ALS patients.
arm group label: Stem/progenitor cells transplantation.
intervention type: Other
intervention name: Symptomatic treatment of ALS
description: Symptomatic neurological treatment of ALS
arm group label: Standard treatment of ALS
criteria: 
gender: All
minimum age: 18 Years
maximum age: 65 Years
healthy volunteers: No
last name: Boguslaw Machalinski, MD, PhD
role: Study Director
affiliation: Pomeranian Medical University Szczecin
last name: Przemyslaw Nowacki, MD, PhD
role: Study Chair
affiliation: Pomeranian Medical University Szczecin
facility: 
country: Poland
responsible party type: Principal Investigator
investigator affiliation: Pomeranian Medical University Szczecin
investigator full name: Milosz Kawa
investigator title: Principal Investigator: Boguslaw Machalinski, Professor and Chief, Department of General Pathology, Pomeranian Medical University, Pomeranian Medical University Szczecin
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
mesh term: Sclerosis
mesh term: Coal Tar