<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/NCT03296501</url>
  </required_header>
  <id_info>
    <org_study_id>DPO/014/33/09/2015</org_study_id>
    <nct_id>NCT03296501</nct_id>
  </id_info>
  <brief_title>Intraspinal Transplantation of Autologous ADRC in ALS Patients</brief_title>
  <acronym>ADIPOSTEM</acronym>
  <official_title>Safety and Efficacy of Intraspinal Transplantation of Autologous ADRC in ALS Patients</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Mossakowski Medical Research Centre Polish Academy of Sciences</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Medical University of Warsaw</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Mossakowski Medical Research Centre Polish Academy of Sciences</source>
  <oversight_info>
    <has_dmc>Yes</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>
      The goal of our nonrandomized, open label study is to investigate the safety and efficacy of&#xD;
      autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the&#xD;
      individuals with diagnosed amyotrophic lateral sclerosis (ALS). All enrolled patients will&#xD;
      have a documented at least 3-months clinical and electrophisiological observation of ALS&#xD;
      disease course prior to study enrollment. Each patient will recive 3 injections of ADRC every&#xD;
      3 months: an intraspinal injection followed by 2 subsequent intrathecal infusions. Safety,&#xD;
      adverse events and efficacy will be confirmed by clinical, elecrophisiological ( EMG, MUNIX),&#xD;
      neuroimmaging and spirometry together with functional (ALSFRS-R) and objective motor&#xD;
      assesment (MRC and dynamometer).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Amyotrophic Lateral Sclerosis (ALS) is an incurable disease of unknown etiology that in a&#xD;
      short time leads to significant impairment of motor functions and death. The frequency of ALS&#xD;
      is 4-8/100 000. Mostly it affects people between 40 and 70 years old, but it can occur at a&#xD;
      younger age. Since the symptom onset is most frequently in the fifth or sixth decade, ALS is&#xD;
      a huge economic burden for the society. There are many studies conducted to treat the disease&#xD;
      and prevent it, but currently the medicine offers only one drug that can slow the appearance&#xD;
      of disease symptoms but could not stop the progression. Yet, improvements in medical&#xD;
      management, including nutrition and breathing, regularly increase patient survival - 50% of&#xD;
      affected patients live at least 3 or more years after diagnosis; 20% live 5 years or more;&#xD;
      and up to 10% will survive more than 10 years. The stem-cell-based therapies could be&#xD;
      therefore a new waited strategy for ALS clinical treatment.&#xD;
&#xD;
      Since the clinical course of ALS may vary substantially between patients, we are planning to&#xD;
      qualify them with a primarily established clinical course based on a detailed anamnesis and&#xD;
      clinical assessment. The goal of our nonrandomized, open label study is to investigate the&#xD;
      safety and efficacy of autologous adipose derived mesenchymal regenerative cells (ADRC)&#xD;
      transplantation into the individuals with diagnosed amyotrophic lateral sclerosis. In order&#xD;
      to select a group of 30 ALS patients for the ADRC treatment, approximately 50 ALS patients&#xD;
      will be examined. All patients enrolled will have a documented at least 3-months clinical and&#xD;
      electrophisiological observation of ALS disease course prior to study enrollment. Each&#xD;
      patient will recive 3 injections of ADRC every 3 months: an intraspinal injection followed by&#xD;
      2 subsequent intrathecal infusions. Safety, adverse events and efficacy will be confirmed by&#xD;
      clinical, elecrophisiological ( EMG, MUNIX), neuroimmaging and spirometry together with&#xD;
      functional (ALSFRS-R) and objective motor assesment (MRC and dynamometer).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Active, not recruiting</last_known_status>
  <start_date type="Actual">October 13, 2015</start_date>
  <completion_date type="Anticipated">October 31, 2022</completion_date>
  <primary_completion_date type="Anticipated">October 31, 2020</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Functional progression rate</measure>
    <time_frame>3 months</time_frame>
    <description>Delta ALS Functional Rating Scale (ALSFRS-R)/month</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Electrophisiological progression rate</measure>
    <time_frame>3 months</time_frame>
    <description>electromyography (EMG)/ Motor unit number index (MUNIX)</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Respiratory insufficiency estimation</measure>
    <time_frame>3 months</time_frame>
    <description>Forced Vital Capacity (FVC)</description>
  </other_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">30</enrollment>
  <condition>Amyotrophic Lateral Sclerosis</condition>
  <arm_group>
    <arm_group_label>Autologous ADRC injection</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients</intervention_name>
    <description>3 injections of ADRC: 1 intraspinal and 2 intrathecal</description>
    <arm_group_label>Autologous ADRC injection</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  clinically definite or probable ALS according to El Escorial criteria&#xD;
&#xD;
          -  life expectancy of more than 1 year&#xD;
&#xD;
          -  INR ≤2 before liposuction&#xD;
&#xD;
          -  lack of treatment with immune-suppressants and/or corticosteroids within min. 20 days&#xD;
             prior to recruitment&#xD;
&#xD;
          -  constant riluzole treatment (50 mg/bid) throughout the study period&#xD;
&#xD;
          -  compliance with treatment regimen e.g. will and possibility to attend check-up visits&#xD;
&#xD;
          -  Polish citizens&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  primary haematological disease, including hypercoagulable states&#xD;
&#xD;
          -  Presence of comorbidity that would stand in the way of neurosurgical treatment-&#xD;
&#xD;
          -  previous history of a spinal-cord surgery at the clinically affected level&#xD;
&#xD;
          -  previous/current history of neoplasm or comorbidity that could impact upon patient's&#xD;
             survival&#xD;
&#xD;
          -  PEG&#xD;
&#xD;
          -  pregnancy /lactation&#xD;
&#xD;
          -  noninvasive/invasive mechanical ventilation at time of recruitment&#xD;
&#xD;
          -  alcohol abuse, cocaine amphetamine, etc.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>65 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Medical University of Warsaw</name>
      <address>
        <city>Warsaw</city>
        <zip>02-097</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Poland</country>
  </location_countries>
  <results_reference>
    <citation>Kuzma-Kozakiewicz M, Marchel A, Kaminska A, Gawel M, Sznajder J, Figiel-Dabrowska A, Nowak A, Maj E, Krzesniak NE, Noszczyk BH, Domanska-Janik K, Sarnowska A. Intraspinal Transplantation of the Adipose Tissue-Derived Regenerative Cells in Amyotrophic Lateral Sclerosis in Accordance with the Current Experts' Recommendations: Choosing Optimal Monitoring Tools. Stem Cells Int. 2018 Aug 12;2018:4392017. doi: 10.1155/2018/4392017. eCollection 2018.</citation>
    <PMID>30158984</PMID>
  </results_reference>
  <verification_date>April 2020</verification_date>
  <study_first_submitted>September 21, 2017</study_first_submitted>
  <study_first_submitted_qc>September 23, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 28, 2017</study_first_posted>
  <last_update_submitted>April 14, 2020</last_update_submitted>
  <last_update_submitted_qc>April 14, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">April 15, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Mossakowski Medical Research Centre Polish Academy of Sciences</investigator_affiliation>
    <investigator_full_name>Anna Sarnowska</investigator_full_name>
    <investigator_title>M.D., Ph.D.</investigator_title>
  </responsible_party>
  <keyword>ALS</keyword>
  <keyword>ADRC</keyword>
  <keyword>adipose derived regenerative cells</keyword>
  <keyword>intraspinal transplantation</keyword>
  <keyword>mesenchymal cells</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>
  </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/NCT03296501
org study id: DPO/014/33/09/2015
nct id: NCT03296501
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
The goal of our nonrandomized, open label study is to investigate the safety and efficacy of
      autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the
      individuals with diagnosed amyotrophic lateral sclerosis (ALS). All enrolled patients will
      have a documented at least 3-months clinical and electrophisiological observation of ALS
      disease course prior to study enrollment. Each patient will recive 3 injections of ADRC every
      3 months: an intraspinal injection followed by 2 subsequent intrathecal infusions. Safety,
      adverse events and efficacy will be confirmed by clinical, elecrophisiological ( EMG, MUNIX),
      neuroimmaging and spirometry together with functional (ALSFRS-R) and objective motor
      assesment (MRC and dynamometer).
Amyotrophic Lateral Sclerosis (ALS) is an incurable disease of unknown etiology that in a
      short time leads to significant impairment of motor functions and death. The frequency of ALS
      is 4-8/100 000. Mostly it affects people between 40 and 70 years old, but it can occur at a
      younger age. Since the symptom onset is most frequently in the fifth or sixth decade, ALS is
      a huge economic burden for the society. There are many studies conducted to treat the disease
      and prevent it, but currently the medicine offers only one drug that can slow the appearance
      of disease symptoms but could not stop the progression. Yet, improvements in medical
      management, including nutrition and breathing, regularly increase patient survival - 50% of
      affected patients live at least 3 or more years after diagnosis; 20% live 5 years or more;
      and up to 10% will survive more than 10 years. The stem-cell-based therapies could be
      therefore a new waited strategy for ALS clinical treatment.

      Since the clinical course of ALS may vary substantially between patients, we are planning to
      qualify them with a primarily established clinical course based on a detailed anamnesis and
      clinical assessment. The goal of our nonrandomized, open label study is to investigate the
      safety and efficacy of autologous adipose derived mesenchymal regenerative cells (ADRC)
      transplantation into the individuals with diagnosed amyotrophic lateral sclerosis. In order
      to select a group of 30 ALS patients for the ADRC treatment, approximately 50 ALS patients
      will be examined. All patients enrolled will have a documented at least 3-months clinical and
      electrophisiological observation of ALS disease course prior to study enrollment. Each
      patient will recive 3 injections of ADRC every 3 months: an intraspinal injection followed by
      2 subsequent intrathecal infusions. Safety, adverse events and efficacy will be confirmed by
      clinical, elecrophisiological ( EMG, MUNIX), neuroimmaging and spirometry together with
      functional (ALSFRS-R) and objective motor assesment (MRC and dynamometer).
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Functional progression rate
time frame: 3 months
description: Delta ALS Functional Rating Scale (ALSFRS-R)/month
measure: Electrophisiological progression rate
time frame: 3 months
description: electromyography (EMG)/ Motor unit number index (MUNIX)
measure: Respiratory insufficiency estimation
time frame: 3 months
description: Forced Vital Capacity (FVC)
arm group label: Autologous ADRC injection
arm group type: Experimental
intervention type: Biological
intervention name: Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients
description: 3 injections of ADRC: 1 intraspinal and 2 intrathecal
arm group label: Autologous ADRC injection
criteria: 
gender: All
minimum age: 18 Years
maximum age: 65 Years
healthy volunteers: No
facility: 
country: Poland
citation: Kuzma-Kozakiewicz M, Marchel A, Kaminska A, Gawel M, Sznajder J, Figiel-Dabrowska A, Nowak A, Maj E, Krzesniak NE, Noszczyk BH, Domanska-Janik K, Sarnowska A. Intraspinal Transplantation of the Adipose Tissue-Derived Regenerative Cells in Amyotrophic Lateral Sclerosis in Accordance with the Current Experts' Recommendations: Choosing Optimal Monitoring Tools. Stem Cells Int. 2018 Aug 12;2018:4392017. doi: 10.1155/2018/4392017. eCollection 2018.
PMID: 30158984
responsible party type: Principal Investigator
investigator affiliation: Mossakowski Medical Research Centre Polish Academy of Sciences
investigator full name: Anna Sarnowska
investigator title: M.D., Ph.D.
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
sharing ipd: No